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CRISPR: A game-changing genetic engineering technique …

Posted: March 10, 2019 at 3:42 pm

Have you heard? A revolution has seized the scientific community. Within only a few years, research labs worldwide have adopted a new technology that facilitates making specific changes in the DNA of humans, other animals, and plants. Compared to previous techniques for modifying DNA, this new approach is much faster and easier. This technology is referred to as CRISPR, and it has changed not only the way basic research is conducted, but also the way we can now think about treating diseases [1,2].

CRISPR is an acronym for Clustered Regularly Interspaced Short Palindromic Repeat. This name refers to the unique organization of short, partially palindromic repeated DNA sequences found in the genomes of bacteria and other microorganisms. While seemingly innocuous, CRISPR sequences are a crucial component of the immune systems [3] of these simple life forms. The immune system is responsible for protecting an organisms health and well-being. Just like us, bacterial cells can be invaded by viruses, which are small, infectious agents. If a viral infection threatens a bacterial cell, the CRISPR immune system can thwart the attack by destroying the genome of the invading virus [4]. The genome of the virus includes genetic material that is necessary for the virus to continue replicating. Thus, by destroying the viral genome, the CRISPR immune system protects bacteria from ongoing viral infection.

Figure 1 ~ The steps of CRISPR-mediated immunity. CRISPRs are regions in the bacterial genome that help defend against invading viruses. These regions are composed of short DNA repeats (black diamonds) and spacers (colored boxes). When a previously unseen virus infects a bacterium, a new spacer derived from the virus is incorporated amongst existing spacers. The CRISPR sequence is transcribed and processed to generate short CRISPR RNA molecules. The CRISPR RNA associates with and guides bacterial molecular machinery to a matching target sequence in the invading virus. The molecular machinery cuts up and destroys the invading viral genome. Figure adapted from Molecular Cell 54, April 24, 2014 [5].

Interspersed between the short DNA repeats of bacterial CRISPRs are similarly short variable sequences called spacers (FIGURE 1). These spacers are derived from DNA of viruses that have previously attacked the host bacterium [3]. Hence, spacers serve as a genetic memory of previous infections. If another infection by the same virus should occur, the CRISPR defense system will cut up any viral DNA sequence matching the spacer sequence and thus protect the bacterium from viral attack. If a previously unseen virus attacks, a new spacer is made and added to the chain of spacers and repeats.

The CRISPR immune system works to protect bacteria from repeated viral attack via three basic steps [5]:

Step 1) Adaptation DNA from an invading virus is processed into short segments that are inserted into the CRISPR sequence as new spacers.

Step 2) Production of CRISPR RNA CRISPR repeats and spacers in the bacterial DNA undergo transcription, the process of copying DNA into RNA (ribonucleic acid). Unlike the double-chain helix structure of DNA, the resulting RNA is a single-chain molecule. This RNA chain is cut into short pieces called CRISPR RNAs.

Step 3) Targeting CRISPR RNAs guide bacterial molecular machinery to destroy the viral material. Because CRISPR RNA sequences are copied from the viral DNA sequences acquired during adaptation, they are exact matches to the viral genome and thus serve as excellent guides.

The specificity of CRISPR-based immunity in recognizing and destroying invading viruses is not just useful for bacteria. Creative applications of this primitive yet elegant defense system have emerged in disciplines as diverse as industry, basic research, and medicine.

In Industry

The inherent functions of the CRISPR system are advantageous for industrial processes that utilize bacterial cultures. CRISPR-based immunity can be employed to make these cultures more resistant to viral attack, which would otherwise impede productivity. In fact, the original discovery of CRISPR immunity came from researchers at Danisco, a company in the food production industry [2,3]. Danisco scientists were studying a bacterium called Streptococcus thermophilus, which is used to make yogurts and cheeses. Certain viruses can infect this bacterium and damage the quality or quantity of the food. It was discovered that CRISPR sequences equipped S. thermophilus with immunity against such viral attack. Expanding beyond S. thermophilus to other useful bacteria, manufacturers can apply the same principles to improve culture sustainability and lifespan.

In the Lab

Beyond applications encompassing bacterial immune defenses, scientists have learned how to harness CRISPR technology in the lab [6] to make precise changes in the genes of organisms as diverse as fruit flies, fish, mice, plants and even human cells. Genes are defined by their specific sequences, which provide instructions on how to build and maintain an organisms cells. A change in the sequence of even one gene can significantly affect the biology of the cell and in turn may affect the health of an organism. CRISPR techniques allow scientists to modify specific genes while sparing all others, thus clarifying the association between a given gene and its consequence to the organism.

Rather than relying on bacteria to generate CRISPR RNAs, scientists first design and synthesize short RNA molecules that match a specific DNA sequencefor example, in a human cell. Then, like in the targeting step of the bacterial system, this guide RNA shuttles molecular machinery to the intended DNA target. Once localized to the DNA region of interest, the molecular machinery can silence a gene or even change the sequence of a gene (Figure 2)! This type of gene editing can be likened to editing a sentence with a word processor to delete words or correct spelling mistakes. One important application of such technology is to facilitate making animal models with precise genetic changes to study the progress and treatment of human diseases.

Figure 2 ~ Gene silencing and editing with CRISPR. Guide RNA designed to match the DNA region of interest directs molecular machinery to cut both strands of the targeted DNA. During gene silencing, the cell attempts to repair the broken DNA, but often does so with errors that disrupt the geneeffectively silencing it. For gene editing, a repair template with a specified change in sequence is added to the cell and incorporated into the DNA during the repair process. The targeted DNA is now altered to carry this new sequence.

In Medicine

With early successes in the lab, many are looking toward medical applications of CRISPR technology. One application is for the treatment of genetic diseases. The first evidence that CRISPR can be used to correct a mutant gene and reverse disease symptoms in a living animal was published earlier this year [7]. By replacing the mutant form of a gene with its correct sequence in adult mice, researchers demonstrated a cure for a rare liver disorder that could be achieved with a single treatment. In addition to treating heritable diseases, CRISPR can be used in the realm of infectious diseases, possibly providing a way to make more specific antibiotics that target only disease-causing bacterial strains while sparing beneficial bacteria [8]. A recent SITN Waves article discusses how this technique was also used to make white blood cells resistant to HIV infection [9].

Of course, any new technology takes some time to understand and perfect. It will be important to verify that a particular guide RNA is specific for its target gene, so that the CRISPR system does not mistakenly attack other genes. It will also be important to find a way to deliver CRISPR therapies into the body before they can become widely used in medicine. Although a lot remains to be discovered, there is no doubt that CRISPR has become a valuable tool in research. In fact, there is enough excitement in the field to warrant the launch of several Biotech start-ups that hope to use CRISPR-inspired technology to treat human diseases [8].

Ekaterina Pak is a Ph.D. student in the Biological and Biomedical Sciences program at Harvard Medical School.

1. Palca, J. A CRISPR way to fix faulty genes. (26 June 2014) NPR < http://www.npr.org/blogs/health/2014/06/26/325213397/a-crispr-way-to-fix-faulty-genes> [29 June 2014]

2. Pennisi, E. The CRISPR Craze. (2013) Science, 341 (6148): 833-836.

3. Barrangou, R., Fremaux, C., Deveau, H., Richards, M., Boyaval, P., Moineau, S., Romero, D.A., and Horvath, P. (2007). CRISPR provides acquired resistance against viruses in prokaryotes. Science 315, 17091712.

4. Brouns, S.J., Jore, M.M., Lundgren, M., Westra, E.R., Slijkhuis, R.J., Snijders, A.P., Dickman, M.J., Makarova, K.S., Koonin, E.V., and van der Oost, J. (2008). Small CRISPR RNAs guide antiviral defense in prokaryotes. Science 321, 960964.

5. Barrangou, R. and Marraffini, L. CRISPR-Cas Systems: Prokaryotes Upgrade to Adaptive Immunity (2014). Molecular Cell 54, 234-244.

6. Jinkek, M. et al. A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. (2012) 337(6096):816-21.

7. CRISPR reverses disease symptoms in living animals for first time. (31 March 2014). Genetic Engineering and Biotechnology News. <http://www.genengnews.com/gen-news-highlights/crispr-reverses-disease-symptoms-in-living-animals-for-first-time/81249682/> [27 July 2014]

8. Pollack, A. A powerful new way to edit DNA. (3 March 2014). NYTimes < http://www.nytimes.com/2014/03/04/health/a-powerful-new-way-to-edit-dna.html?_r=0> [16 July 2014]

9. Gene editing technique allows for HIV resistance? <http://sitn.hms.harvard.edu/flash/waves/2014/gene-editing-technique-allows-for-hiv-resistance/> [13 June 2014]

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How A "Full Body Stem Cell Makeover" Works – Ben …

Posted: March 9, 2019 at 6:53 am

Note from Ben regarding the Full Body Stem Cell Makeover: I mentioned 30K as total cost on the podcast.That actually is the cost for all the stem cell and exosome injectionsinto the spine and joints.The *cosmetic* and *sexual* add-ons tack 12-15K onto the total cost of this procedure. Just a heads up!

What you are about to discover in this podcast is one of the most advanced, fringe, cutting-edge biohacks and forays into self-experimentation I've ever done

a full body stem-cell makeover at Docere Clinics in Park City, Utahincluding a little-known type of compound called an exosome.

Dr. Harry Adelson, my firstguest on today's podcast,was one of the early adopters of stem cell therapy for the treatment of pain. He began his training in regenerative injection therapy (prolotherapy) in 1998 while in his final year at The National College of Naturopathic Medicine, in Portland, Oregon after having been cured of a rock-climbing injury with prolotherapy. During his residency program in Integrative Medicine at the Yale/Griffin Hospital in Derby, Connecticut, he volunteered after hours in a large homeless shelter in Bridgeport, Connecticut, providing regenerative injection therapies to the medically underserved while gaining valuable experience. He opened Docere Clinics in Park City in 2002 and from day one, his practice has been 100% regenerative injection therapies for the treatment of musculoskeletal pain conditions. In 2006 he incorporated platelet-rich plasma and ultrasound-guided injection into his armamentarium, in 2010, bone marrow aspirate concentrate and adipose-derived stem cells, and in 2013, fluoroscopic-guided injection (motion X-ray).

Since February of 2010, Dr. Adelson has performed over 5,000 bone marrow and adipose-derived adult stem cell procedures and has injected stem cells into over 500 intervertebral discs, placing him among those most experienced in the world with the use of autologous stem cells for the treatment of musculoskeletal pain conditions.

Dr. Amy Killen is also a guest on today's show. She joined Docere Medical after moving to Utah from Portland, Oregon, where she was the Medical Director of an anti-aging and regenerative medicine specialty practice. Dr. Killens medical training began in Dallas, where she attended medical school at UT Southwestern and received her M.D. degree, graduating in the top 10% of her class. She completed a residency in Emergency Medicine at the University of Arizona and served as Chief Resident during her final year. After working as a board-certified emergency physician for more than 7 years, Dr. Killen began studying and training in anti-aging, regenerative, and aesthetic medicine in hopes of approaching patients wellness and beauty from a different perspective and with a new toolbox of innovative and effective treatment options.

Advanced training has included completion of the fellowship in Anti-Aging and Regenerative Medicine through the American Association of Anti-Aging Medicine and certification training in Age Management Medicine. Dr. Killen is certified by the National Institute of Medical Aesthetics in both basic and advanced cosmetic injections and trained with Dr. Lisbeth Roy to learn the O-Shot and P-Shot PRP procedures. Further PRP injection training was provided by PRP Life Lift and she completed the Hair Coach certification program with Dr. Alan Bauman in Florida, which taught a comprehensive approach to non-surgical hair loss. Dr. Killen learned how to extract and process stem cells as part of the U.S. Stem Cell Training Course taught by leading stem cell scientist Dr. Kristin Comella. Dr. Amy Killen has also spent many hours in the operating suite learning directly from Harry Adelson N.D.at Docere Clinics.

Dr. Killen has spoken nationally about PRP and stem cell therapies and teaches a physician training course for ApexBiologix outlining current best practices for using regenerative therapies in aesthetics and sexual optimization.

During my discussion with Harry and Amy, you'll discover:

-How Harry got started with prolotherapy and PRP for pain management in farmers and construction workers14:45

-The Stem Cell Theory of Aging, which states that biologic aging is a result of loss function and population of stem cells in musculoskeletal tissues24:00

-How Harry is harvesting stem cells from bone marrow, supplementing them with exosomes (the currency of stem cells, the inter-cellular communication blocks), and then injecting them into all major moving parts as well as skin of face, scalp, and penis28:30

-Why umbilical and amniotic stem cells could potentially be dangerous due to exposing the body to foreign DNA32:30

-What MSC's are, why are they different and better than other stem cell therapies, and how they differentiate into musculoskeletal tissues and have been called medical signaling cells because they trigger the healing of damaged tissues42:00

-Why somebody would undergo a full body stem cell treatment, and whether is it going to rejuvenate all of the cells in my body49:30

-What to expect after a full body stem cell makeover57:00

Interview with Dr. Amy Killen:

-WhatPRP therapy is, and how it differs from stem cells1:07:00

-What happens when you inject stem cells into a mans unit, and why Amy injected Ben1:11:30

-Why Amy injects stem cells and PRP into vaginas, and how the O-Shot improves the power and duration of female orgasms1:15:20

-The secrets of the vampire breast lift and other cosmetic procedures she has done1:18:15

-What causes hair loss, and how to fix it with stem cells1:22:15

-What V-cells are and how they work1:27:00

-Post-treatment care and biohacks to enhance recovery and efficacy of the treatment1:29:00

-And much more!

Click here for the full written transcript of this podcast episode.

Resources from this episode:

Docere Clinics in Park City, Utah

-Video:What Are Exosomes? Ben Greenfield Interviews Dr. Harry Adelson On Full Body Stem Cell Makeover

-Video:Ben Greenfield Interviews Dr. Amy Killen About Stem Cells, PRP, Exosomes & P-Shot

-Video:Ben Greenfield Full Body Stem Cell Makeover With Exosomes: Part 1

-Video:Ben Greenfield Full Body Stem Cell Makeover With Exosomes: Part 2

-All the recovery biohacks I implemented to recover from the stem cell procedures and enhance stem cell production:

Flexpulse PEMFPulsecenters PEMFBiomat InfraredTrusii Hydrogen Rich WaterJoovv Infrared LightNanoVi DNA RepairExogenous HVMN KetonesOmega-3 DHA Superessential Fish OilVielight PhotobiomodulationKion FlexClearlight Sauna

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Do you have questions, thoughts or feedback for Dr. Harry, Amy or me? Leave your comments below and one of us will reply!

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Marshfield Clinic Research Institute – Welcome to The …

Posted: March 9, 2019 at 6:51 am

The Center for Precision Medicine Research (CPMR), formerly the Center for Human Genetics,wasestablished in 2004 and focuses its research on discovering the structure of the human genome and the hunt for genes that influence human health disorders. The discoveryof short tandem repeat polymorphisms in 1989revolutionized the study of human genetics, and theMarshfield mapsare among the most reliable and widely used maps of the human genome in the world.Thecentercomprises oftwo of Marshfield Clinics internationally known research assets: the Center for Medical Genetics, founded in 1994, and the Personalized Medicine Research Center, started in 2001.Scientists and CPMR staffnow focus on multiple areas of study including Personalized Medicine Research Project (PMRP) study, Precision Medicine Initiative (PMI) study and All of Us (AoU) Research Program.

In 2002,CPMRlaunchedthe largest population-based genetic research project in the United States, involving more than 20,000 central Wisconsin residents, the Personalized Medicine Research Projector PMRP. By understanding which genes and environmental factors are involved in disease and cancer, doctors might be able to better target the biological pathways involved. This information may also enable doctors to predict disease risk and prescribe preventative measures.

The Marshfield Clinic isone of four partners in the Wisconsin Genomics Initiative, a historic collaboration, whose vision is to be able to predict, for individual patients in a clinical setting, the risks of disease susceptibility and treatment response using the combined power of cutting edge genetic, phenotypic, and environmental analysis. Each of the four partners is a leader in one or more areas needed for a successful effort.

A team of investigators and staff in the Center for Precision Medicine Research, led by Scott Hebbring, PhD, are conducting a pilot study that applies genetic testing results to improve and personalize care for 2,000 Marshfield Clinic patients. It is estimated that up to 3% of patients will carry a genetic variant that is clinically actionable. Clinically actionable variants are those that increase disease risk for conditions where early detection may improve outcomes such as cancer and heart disease. In addition to disease risk variants, it is expected that 95% of patients will carry one or more pharmacogenetic variants. Pharmacogenetic variants are those that influence how people respond to specific medications. This genetic data are being integrated into Marshfield Clinics EHR and decision support tools are being developed so our patients can receive the right drug, at the right time, and at the right dose. This project is supported by generous patient donations, financial support from Security Health Plan, and grant awards from National Institute of Health.

Marshfield Clinic Research Institute is the lead site in Wisconsin for thenationalAll of Us Research Program with collaborators at the Universityof WisconsinSchool of Medicine& Public Health, Froedtert &the Medical College of Wisconsin, and the Versiti Wisconsin, Inc. This study is a momentous effort to advance individualized prevention, treatment and care for people of all backgrounds. Murray Brilliant, PhD, Interim CPMR Director, is leading the state-wide recruitment efforts. The catchment area of the Wisconsin consortium covers 80% of the state thereby reflecting the true diversity of the state including both rural and urban populations.For additional information, including how to participate, visit JoinAllofUs.org, email allofus@marshfieldresearch.org, or call (888) 633-9987.

The mission of the Center forPrecision Medicine Researchis to conduct translational research in medical genetics that substantially improves patient care. Dr. Murray Brilliant affirms that the Center is committed to advancing scientific knowledge through humangenetics research and to translating that knowledge into practical applications that will foster improved health.

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The Marino Center for Integrative Health – Cambridge – Mount …

Posted: March 9, 2019 at 6:49 am

At the Marino Center for Integrative Health, we're honored that you have put your trust in us to deliver the best in comprehensive healthcare services. Here, we offer a unique, integrative model of healthcare, featuring both conventional primary care and a full array of complementary services - conveniently brought together under one roof - to promote your health and the pursuit of a healthy lifestyle.

Integrative Health - its in our name, but you may be wondering exactly what it means. Well, for us at the Marino Center, Integrative Health, or Integrative Medicine, means a unique model of healthcare focused on treatingyou as anindividual, not the disease. Here we aim to restore the focus on health and healing through a strong patient/practitioner relationship. For us, it also means taking more time to understand you and your needs, making sureyou feel listened to, and building an individualized plan for living better and staying better.

With a broader approach to healing and wellness, Integrative Medicine takes into account all aspects of what makes a person whole - mind body and spirit and addresses them all at the same time. Through an active practitioner and patient partnership, a treatment plan including the use of both traditional (sometimes also referred to as conventional or western medicine) and alternative (also called CAM and complementary) therapies is developed to promote health AND prevent illness and disease.

Relying on the best of scientifically-validated conventional and alternative therapies, Integrative Medicine is a practical strategy that puts the whole patient at the center of care. In addition to physical symptoms, it examines the psychological, social and spiritual nature of the individual. An integrative approach educates and empowers the patient to play an active and responsible role in his or her own health.

Our mission is to integrate scientifically and empirically demonstrated conventional and complementary healing traditions to improve the health of those we serve and to extend our knowledge to others through health education, training, and research.

Patient Care

Providing healthcare services through a collaborative team of compassionate, innovative practitioners who provide preventive health care, and work with patients with acute and chronic conditions

Education and Training

Providing each person seeking our services with scientific explanations and informed choices regarding the professional care and self-care of body, mind, and spirit

Sponsoring and participating in public and professional presentations, seminars, and symposia along with publications and other media to educate the general public, medical, and insurance institutions about the unique potential of integrative medicine

Educating medical students through clinical rotations, internships, residencies, and fellowships

Research

Examining the applied clinical aspects of integrating complementary and alternative therapies to determine efficacy and safety

Participating in research studies on specific medical practices and our integrative model with leading research institutions

Performing critical analyses of the service delivery system and examine the clinical and administrative structure of integrative medicine

Lelio 'Les' Marino was the visionary, founder and generous benefactor of what was then known as the Marino Foundation for Integrative Medicine, the sponsor of The Marino Centers for Progressive Health. Because of his vision and generosity, the Center has grown into one of the region's premier providers of integrative medicine and healthcare services.

Our origins date back to 1993 when Mr. Marino acquired a small alternative medical practice in Cambridge, MA. From this humble beginning, he envisioned a model of care that would integrate mind, body, and spirit, and genuinely blend traditional and complementary healing practices. In 2008, we integrated our two names and became the Marino Center for Integrative Health. Throughout the years, the Marino Center has been guided by Mr. Marino's vision and its mission of providing patient care, education, and research - and continues to be today.

After a decade operating centers in Cambridge and Wellesley, in 2013, the Marino Foundation for Integrative Medicine decided to turn its focus to supporting new initiatives in Integrative Medicine, and the Cambridge Marino Center for Integrative Health was acquired by Mount Auburn Hospital, a Harvard-affiliated teaching hospital with a long history of support for integrative, alternative and prevention-based medical approaches.

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Stem Cell Lake Havasu City Arizona 86406

Posted: March 8, 2019 at 6:44 am

Stem cell therapy has actually ended up being a popular dispute in the global medical scene. This highly questionable therapy has received combined opinions from numerous stakeholders in the healthcare industry and has also attracted the attention of political leaders, religious leaders and the basic population at large. Stem cell treatment is considered a revolutionary treatment for people struggling with a vast array of degenerative conditions. Some typical questions concerning this therapy are responded to listed below.

Are you a stem cell therapy specialist near Lake Havasu City AZ 86406? Contact us for more information about joining our website.

Stem cells can be described as blank state or non-specialized cells that have the capability to become specialized cells in the body such as bone, muscle, nerve or organ cells. This implies that these special cells can be utilized to regrow or develop a wide range of damaged cells and tissues in the body. Stem cell therapy is for that reason a treatment that aims at attaining tissue regrowth and can be used to treat health conditions and illnesses such as osteoarthritis, degenerative disc illness, spinal cord injury, muscular degeneration, motor neuron illness, ALS, Parkinsons, heart disease and many more.

Being a treatment that is still under research study, stem cell treatment has actually not been fully accepted as a viable treatment choice for the above discussed health conditions and illnesses. A lot of research study is presently being performed by scientists and medical specialists in numerous parts of the world to make this treatment practical and reliable. There are however numerous restrictions enforced by governments on research involving embryonic stem cells.

Presently, there havent been numerous case studies performed for this form of treatment. Nevertheless, with the few case studies that have actually been carried out, among the significant issues that has actually been raised is the increase in a patients risk of developing cancer. Cancer is brought on by the fast multiplication of cells that tend not to pass away so easily. Stem cells have actually been associated with similar growth elements that may lead to formation of tumors and other malignant cells in clients.

Contact us for more information about stem cell provider in Lake Havasu City AZ 86406

Stem cells can be extracted from a young embryo after conception. These stem cells are commonly described as embryonic stem cells. After the stem cells are extracted from the embryo, the embryo is ended. This is basically among the major reasons for debate in the field of stem cell research. Many individuals suggest that termination of an embryo is unethical and undesirable.

Stem cells can still be acquired through other means as they can be found in the blood, bone marrow and umbilical cables of adult humans. Typical body cells can likewise be reverse-engineered to become stem cells that have limited capabilities.

New studio has nevertheless revealed promise as researchers target at establishing stem cells that do not form into tumors in later treatment phases. These stem cells can therefore efficiently change into other kinds of specialized cells. This treatment is for that reason worth investigating into as numerous patients can benefit from this revolutionary treatment.

The best stem cell provider near Lake Havasu City AZ 86406

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Main address:Lake Havasu City, Arizona, 86406

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Bone Marrow & Stem Cell Transplant | Weill Cornell Medicine

Posted: March 8, 2019 at 6:43 am

Bone Marrow & Stem Cell Transplant

The Bone Marrow and Stem Cell Transplant Program at Weill Cornell Medicine was established with the mission of providing the best care and most innovative research in a compassionate and comfortable environment.

We take a multidisciplinary approach to care for patients with cancer and blood diseases who need stem cell transplants, providing world-class clinical care in collaboration with experts in leukemia, lymphoma, myeloma and other blood disorders. Based at NewYork-Presbyterian/Weill Cornell Medical Center, one of the top ten general hospitals in the nation, the expertise of our consulting team is unsurpassed.

Our patients and families cope with life-threatening illness; as such, sensitivity and compassion are a priority for our team. We view each patient as an individual, and our approach ensures that each treatment regimen is narrowly tailored to meet the unique, changing needs of our patients and their families before, during and after transplant.

As New Yorks premier healthcare institution, Weill Cornell Medicine is at the forefront of scientific research and clinical trials, enabling us to provide a full range of diagnostic and treatment protocols, including the latest breakthroughs in medicine.

Our Team

Our team of internationally-recognized bone marrow transplant and stem cell surgery specialists is known for advanced work and published research in:

Treating patients with aggressive leukemia and myelodysplastic syndromes

Bridge protocols for patients with refractory lymphoma and leukemia

Novel strategies to mobilize stem cells and improve transplantation for patients with multiple myeloma, leukemia and lymphoma

Transplants for solid tumors, severe auto-immune disorders, and AIDS

Treatment

We pride ourselves on exceptional outcomes and offer patients the most advanced diagnostic methods and treatment therapies to improve quality of life, including:

Umbilical cord blood transplant

Outpatient transplant

Autologous stem cell transplant; uses stem cells extracted from the bone marrow or peripheral blood of the patients own blood

Allogeneic stem cell transplant; uses stem cells extracted from the bone marrow or peripheral blood of a matching donor

Hematopoietic stem cell transplant; used to treat certain cancers of the blood/bone marrow, including leukemia and myeloma

Matched unrelated donor stem cell transplantation through the National Donor Matching Program

Non-ablative "mini" transplants

Haplo-Cord Transplant, allowing us to find donors for all patients, regardless of age or ethnic background

Bendamustine, a therapy that is well-tolerated and has excellent response rates in patients with myeloma

Novel forms of transplant, offering hope and success to older patients with leukemia

Clinical Trials

Clinical trials are important to improve outcomes and offer new treatment options. At Weill Cornell Medicine, we conduct more studies in blood cancers than any of our regional peers, allowing us to provide our patients with access to many multi-phase clinical trials. As active members of the international cancer research community, our oncologists also collaborate with other research centers to offer patients the most promising treatments available.

Second Opinions

In concert with your referring physician, we are always available to offer a second opinion in the form of a consultation with one of our specialists.

Why Choose Us?

Our collaborative approach means our patients receive supportive, comprehensive care and the most cutting-edge stem cell therapy and treatments. This enables patients to receive the best possible transplant outcomes. Additionally, we offer more allogeneic stem cell transplants for older adults than any other center in New York City and the entire tri-state area.

For more information or to schedule an appointment, call us at 212-746-2119 or 212-746-2646.

Located in New York City, Weill Cornell Medical College is ranked among the nations best by U.S. News & World Report year after year.

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OMIM Entry – * 104311 – PRESENILIN 1; PSEN1

Posted: March 8, 2019 at 6:43 am

Alzheimer's Disease Collaborative Group. The structure of the presenilin 1 (S182) gene and identification of six novel mutations in early onset AD families. Nature Genet. 11: 219-222, 1995. [PubMed: 7550356] [Full Text: https://dx.doi.org/10.1038/ng1095-219%5D

Ataka, S., Tomiyama, T., Takuma, H., Yamashita, T., Shimada, H., Tsutada, T., Kawabata, K., Mori, H., Miki, T. A novel presenilin-1 mutation (leu85pro) in early-onset Alzheimer disease with spastic paraparesis. Arch. Neurol. 61: 1773-1776, 2004. [PubMed: 15534188] [Full Text: https://jamanetwork.com/journals/jamaneurology/fullarticle/10.1001/archneur.61.11.1773%5D

Athan, E. S., Williamson, J., Ciappa, A., Santana, V., Romas, S. N., Lee, J. H., Rondon, H., Lantigua, R. A., Medrano, M., Torres, M., Arawaka, S., Rogaeva, E., and 10 others. A founder mutation in presenilin 1 causing early-onset Alzheimer disease in unrelated Caribbean Hispanic families. JAMA 286: 2257-2263, 2001. [PubMed: 11710891] [Full Text: https://jamanetwork.com/journals/jama/fullarticle/vol/286/pg/2257%5D

Bai, G., Chivatakarn, O., Bonanomi, D., Lettieri, K., Franco, L., Xia, C., Stein, E., Ma, L., Lewcock, J. W., Pfaff, S. L. Presenilin-dependent receptor processing is required for axon guidance. Cell 144: 106-118, 2011. [PubMed: 21215373] [Full Text: https://linkinghub.elsevier.com/retrieve/pii/S0092-8674(10)01375-9%5D

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Lopera, F., Ardilla, A., Martinez, A., Madrigal, L., Arango-Viana, J. C., Lemere, C. A., Arango-Lasprilla, J. C., Hincapie, L., Arcos-Burgos, M., Ossa, J. E., Behrens, I. M., Norton, J., Lendon, C., Goate, A. M., Ruiz-Linares, A., Rosselli, M., Kosik, K. S. Clinical features of early-onset Alzheimer disease in a large kindred with an E280A presenilin-1 mutation. JAMA 277: 793-799, 1997. [PubMed: 9052708] [Full Text: https://jamanetwork.com/journals/jama/fullarticle/vol/277/pg/793%5D

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OMIM Entry - * 104311 - PRESENILIN 1; PSEN1

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Sanford stem cell trial crosses key threshold in offering …

Posted: March 8, 2019 at 6:42 am

Dec. 21, 2018

This paid piece is sponsored by South Dakota Biotech.

Sanford Research has hit a key milestone in its efforts to gain FDA approval for ground-breaking use of stem cells in orthopedics and is already pursuing new trials to broaden its efforts.

Early data has been released showing that the first patients who were part of a clinical trial using adipose-derived stem cells to treat rotator cuff tears had no harmful effects from the treatment.

We know all our patients were safe, but a significant amount of our patients also had pain relief and some function relief, said Tiffany Facile, Sanfords director of regenerative medicine.

That really contributes to the next phase.

The next step a phase two trial that is intended to show the treatments efficacy will include about 200 patients at 10 sites, starting in early 2019.

That study duration is about a year, Facile said.

Sanford has worked in partnership with a hospital in Munich, Germany, for years as the treatment was pioneered and offered to patients there. So while the results in the U.S. are welcomed, theyre not entirely surprising, she said.

We know from patients in Munich that there is relief. This is a confirmation of what we knew, but theres additional excitement because this could provide another treatment option for patients here, and it means a lot to me to be able to tell that to patients.

At the same time, Sanford is working through studies using adipose-derived stem cells to treat osteoarthritis in the wrist and in the back. Like the rotator cuff study, researchers have started by testing for safety and will follow with a trial for efficacy.

Sanford Health is well connected nationally and internationally in the regenerative medicine world, Facile said. I think because we have set the standard and maintained a great relationship with the FDA, we will continue to lead in this space.

Facile and David Pearce, executive vice president of innovation and research, will present in early 2019 at the World Stem Cell Summit in Miami, which features global leaders in the stem cell and regenerative medicine community.

Sanford is really trying to lead and be a good mentor for others to work with the FDA, Facile said. We can do it. We can do it the right way, but you just have to trust in the science and have science in the clinical application. We are sharing our story about how it can be done in the best way, and its an extreme honor.

Sanfords leadership in regenerative medicine is a huge asset in growing South Dakotas visibility within the bioscience industry, saidJoni Johnson, executive director of South Dakota Biotech.

This sort of activity in clinical trials is so exciting for our state, Johnson said. It broadens our relationships, leads to additional collaboration and helps continue to attract the sort of research talent that is lifting up our entire bioscience economy.

Sanfords success in pursuing stem cell clinical trials has bolstered its ability to recruit and collaborate, Facile agreed.

Its exciting. This is a competitive field, and were meeting with scientists nationally who are looking for clinical sites to conduct their studies, and Sanford is that place. Thats Sanford. So it gets me excited, and the fact that were following the FDA and bringing this treatment to patients in the right way is so important to take into consideration. Its the right thing for the patient.

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Clinical Trial for Autism – cordbloodbank.com

Posted: March 8, 2019 at 6:41 am

Sarahs StoryTreating Autism with Stem Cells August 27, 2018 | Cord Blood Banking, Treatments Duke University Clinical Trial

In 2010, Anna and her husband decided to bank their son, Ryans, cord blood with New England Cord Blood Bank, Inc. (NECBB). Two years later, when their daughter Sarah was born, they opted to save her cord blood as well. Later, when Sarah was diagnosed with Autism, Anna thought about the possibility of using cord blood stem cells and began a quest to research medical advancements in stem cell therapies that could potentially benefit her daughters condition.

Anna joined Facebook Groups and scoured the internet looking for potential treatments for Sarah. After a year of researching options, Anna discovered a clinical trial run by Duke University. If accepted, Sarah could use her own stem cells to participate in the study. In June 2016, she was accepted into the trial.NECBB coordinated with the family and Duke University Hospital to oversee the logistics of transporting and delivery of the cryopreserved stem cells for Sarahs treatment. With high hopes, mother and daughter journeyed to Durham, North Carolina for the trial therapy.Sarahs first treatment was completed in February 2017. The family did not observe any changes in Sarah at that time. Her speech and language skills remained below grade level, her drawings were simple lines on paper and she remained socially awkward. Since the Duke University Hospital Study does not disclose clinical trial treatment information (placebo or stem cells), Anna speculates that the first treatment was, perhaps, a placebo. She does not know for sure. After Sarahs next session, dramatic improvements were noted. In less than a year, Sarahs language improved from two or three word sentences to 16 words and her scribbles on paper evolved into detailed images of flowers. In addition, her tantrums decreased dramatically and her social proficiency evaluation indicated that her skill set was off the charts.

Since the clinical trial at Duke University Hospital did not report the individual outcome to each participant, they only release a general report of the cumulative results, Sarahs success was evaluated independently. She is still on her Individual Education Plan (IEP) at pre-school, but her mother reports that everything is better. Its not a cure and there are still challenges, but there are improvements across the board. Anna notes that there are still lots of hurdles and developmental delays, yet their daughters progress has been exhilarating for the family. But Sarahs story isnt done yet. Her brother, Ryan, is a partial (50%) HLA match and therefore his cord blood stem cells can be used to extend her treatment. Anna has registered Sarah for an allogeneic (using stem cells from a donor, in this case Ryan) transplant this month as part of Dukes Expanded Access Protocol.

We need to stop throwing out these stem cells as medical waste, they are pure gold. I enrolled in this program at NECBB as an insurance policy for my kids. I hoped that I would never need these stem cells. By using a private bank, we had our own stem cells for our own use. Now, Im so glad we made this decision. NECBBs [staff] was knowledgeable and easy to work with, no stress at all. I read on various social media groups that parents discovered that their stem cell samples were contaminated after they applied for a trial. Other families couldnt get their medical reports or specimens released from their cord blood bank. These other banks put some childrens acceptance in the trial at risk. Anna said.

Anna remains hopeful that there will be a mesenchymal stem cell (MSCs) clinical trial for autism in the near future. MSCs are building block cells that not only can self replicate, but can also differentiate into many different cell types. With that trait they could one day repair or replace a wide variety of tissues, such as bone, cartilage, neural cells, muscle cells, and so on. MSCs can be found in umbilical cord tissue and dental pulp. Anna said she wishes she had stored Sarahs umbilical cord tissue when she preserved her cord blood. (NECBB offers the option of storing umbilical cord tissue, a source of MSCs). Additionally, NECBBs affiliate company, National Dental Pulp Laboratory, Inc. (NDPL) processes and stores MSCs collected from dental pulp or extracted teeth.

Anna added, Im excited about the potential of treatment. I hope that soon the FDA will open the floodgates so people can have access to this therapy. Its safe, so let people benefit from it. NECBB has shipped the siblings specimen to Duke University for Sarahs additional treatment. Anna is hopeful that Sarah will show continued development in combating the symptoms of Autism. She said the results to date have been astonishing; Sarah continues to flourish as a 5-1/2 year old girl. She is thriving in school and enjoying life with her friends.

NOTES: The names have been changed to protect the anonymity of the participants. This reviews the results of a Clinical Trial; the FDA has not approved stem cell transplants as a treatment for Autism. This should not be interpreted as a cure additionally, the story is not intended to imply there is a need for a cure.

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Clinical Trial for Autism - cordbloodbank.com

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Neuroscience Investigator Awards – New York Stem Cell …

Posted: March 8, 2019 at 6:41 am

The 2019 RFA is now closed.

NYSCF is soliciting applications from early career investigators for Innovator awards in neuroscience. The goal of this initiative is to foster truly bold, innovative scientists with the potential to transform the field of neuroscience. Applicants are encouraged in the fundamental areas of developmental, cellular, cognitive, and translational neuroscience, broadly interpreted. Applicants need not be working in areas related to stem cells.

The award provides $1.5M USD over 5 years and is open to researchers based at both national and international accredited academic and nonprofit research institutions.

To be eligible, candidates must:

*PLEASE NOTE ONLINE APPLICATION SUBMISSION PROCESS*All applications MUST be submitted through the online grant management system by February 20, 2019 at 5pm Eastern.

Register & Apply

Applicants must submit an Institutional Report Card for Gender Equality in order for the application to be considered complete. Details and FAQs can be found here.

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