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Category Archives: Stem Cells

Tokyo’s Heartseed reaps $26M to test its stem cell injections for heart failure – FierceBiotech

Posted: November 25, 2019 at 8:45 pm

Tokyo-based Heartseed has raised 2.8 billion yen, or about $26 million U.S., to help develop its stem cell-based treatment for heart failure.

The company has its eyes on two clinical trials, set to start in the next year: an investigator-initiated study through its research partner Keio University in dilated cardiomyopathy, followed by a phase 1/2 trial in late 2020 for heart failure with reduced left ventricular ejection fraction.

Heartseeds treatment differs in approach from other, similar therapies using induced pluripotent stem cells (iPSCs), where sheets of cells are grafted onto the surface of the heart to improve vascularization and blood flow.

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Instead, the company forms spherical clusters of heart cells from purified, allogeneic iPSCs, which are injected directly into the heart muscle wall. As they grow and electrically sync with other cells within the myocardium, Heartseed expects to see improvements in the strength of contractions.

The companys series B round included new investors SBI Investment, JMDC, Gene Techno Science, Nissay Capital and SMBC Capital as well as Astellas Venture Management, returning from Heartseeds series A raise. The latest proceeds bring its total funding to 3.8 billion yen, or about $35 million.

RELATED: Growing transplantable arteries from stem cells

Heartseed was founded in 2015 through the research of its CEO, Keiichi Fukuda, a professor in the department of cardiology at Keio University, and his teams work in regenerative medicine and cell production, purification and delivery.

We are grateful for the support of our investors, which I believe is a reflection of their expectation and confidence that our lead pipeline HS-001 can be a curative therapy for severe HF, with the mechanism that transplanted ventricular-specific highly-purified cardiomyocytes engraft to patients heart and retain for a long-term, Fukuda said in a statement.

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Global Stem Cell Banking Market by Source, Application, Service Type & Region – Forecast to 2025 – ResearchAndMarkets.com – Business Wire

Posted: November 25, 2019 at 8:45 pm

DUBLIN--(BUSINESS WIRE)--The "Stem Cell Banking Market by Source, by Application, and by Service Type: Global Industry Perspective, Comprehensive Analysis, and Forecast, 2018-2025" report has been added to ResearchAndMarkets.com's offering.

The report covers a forecast and an analysis of the stem cell banking market on a global and regional level. The study provides historical data from 2016 to 2018 along with a forecast from 2019 to 2025 based on revenue (USD Billion).

The study includes the drivers and restraints of the stem cell banking market along with their impact on the demand over the forecast period. Additionally, the report includes the study of opportunities available in the stem cell banking market on a global level.

In order to give the users of this report a comprehensive view of the stem cell banking market, we have included a competitive landscape and an analysis of Porter's Five Forces model for the market. The study encompasses a market attractiveness analysis, wherein all the segments are benchmarked based on their market size, growth rate, and general attractiveness.

The report provides company market share analysis to give a broader overview of the key players in the market. In addition, the report also covers key strategic developments of the market including acquisitions & mergers, new launch, agreements, partnerships, collaborations & joint ventures, research & development, and regional expansion of major participants involved in the market.

The study provides a decisive view of the stem cell banking market by segmenting it on the basis of source, application, service type, and region. All the segments have been analyzed based on present and future trends and the market is estimated from 2019 to 2025. The regional segment includes the current and forecast demand for North America, Europe, Asia Pacific, Latin America, and the Middle East and Africa.

Some major players of the global stem cell banking market are Cord Blood Registry (CBR) Systems, Cordlife Group Limited, Cryo-Cell International, ViaCord, Cryo-Save, LifeCell International, StemCyte, Global Cord Blood Corporation, Vita34, Smart Cells International, and CryoHoldco.

Key Topics Covered

Chapter 1. Introduction

1.1. Report Description & Scope

1.2. Research Scope

1.3. Research Methodology

Chapter 2. Executive Summary

2.1. Global Stem Cell Banking Market, 2016-2025 (USD Billion)

2.2. Global Stem Cell Banking Market: Snapshot

Chapter 3. Stem Cell Banking Market - Industry Dynamics

3.1. Introduction

3.2. Market Drivers

3.3. Market Restraints

3.4. Opportunities

3.5. Porter's Five Forces Analysis

3.6. Market attractiveness analysis

Chapter 4. Global Stem Cell Banking Market - Competitive Landscape

4.1. Company Market Share Analysis

4.2. Strategic Development

Chapter 5. Global Stem Cell Banking Market - Source Analysis

5.1. Global Stem Cell Banking Market: Source Overview

5.2. PSCs

5.3. hESCs

5.4. BMSCs

5.5. ADSCs

5.6. Others

Chapter 6. Global Stem Cell Banking Market - Application Analysis

6.1. Global Stem Cell Banking Market: Application Overview

6.2. Personalized Banking

6.3. Clinical

6.4. Research

Chapter 7. Global Stem Cell Banking Market - Service Type Analysis

7.1. Global Stem Cell Banking Market: Service Type Overview

7.2. Sample Collection & Transportation

7.3. Sample Processing

7.4. Sample Analysis

7.5. Sample Preservation & Storage

Chapter 8. Global Stem Cell Banking Market - Regional Analysis

8.1. Global Stem Cell Banking Market: Regional Overview

8.2. North America

8.3. Europe

8.4. Asia-Pacific

8.5. Latin America

8.6. The Middle East & Africa

Chapter 9. Company Profiles

9.1. Cord Blood Registry (CBR) Systems

9.2. Cordlife Group Limited

9.3. Cryo-Cell International

9.4. ViaCord

9.5. Cryo-Save

9.6. LifeCell International

9.7. StemCyte

9.8. Global Cord Blood Corporation

9.9. Smart Cells International

9.10. Vita34

9.11. CryoHoldco

For more information about this report visit https://www.researchandmarkets.com/r/t0fyz4

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Global Stem Cell Banking Market by Source, Application, Service Type & Region - Forecast to 2025 - ResearchAndMarkets.com - Business Wire

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Applying AI and CRISPR to stem cells to improve regenerative medicine – FierceBiotech

Posted: November 25, 2019 at 8:45 pm

Human-induced pluripotent stem cells (hiPSCs) generated from a persons own adult cells can grow into complex organs that help scientists test drugs or even transplant into patients. However, directing stem cells into forming desired, functional organs in the lab remains challenging.

Now, in a study published in the journal Cell Systems, researchers from Gladstone Institutes in collaboration with Boston University (BU) described using machine learning to better understand how to use CRISPR-Cas9 gene-editing tools to control iPSC organization.

By coaxing these stem cells into forming specific arrangements, the researchers believe they could create functional organs for research or therapeutic purposes.

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While researchers have managed to develop iPSCs into many different cell types but not necessarily functional 3D organs, mainly because they have struggled to manipulate the spatial patterns of stem cells, which define the tissues they eventually grow into. Some have resorted to 3D printing, but it isnt always successful, as cells often migrate away from their printed locations.

Despite the importance of organization for functioning tissues, we as scientists have had difficulty creating tissues in a dish with stem cells, Ashley Libby, a co-first author of the new study, said in a statement. Instead of an organized tissue, we often get a disorganized mix of different cell types.

The researchers previously showed that knocking down two genes, ROCK1 and CDH1, affected the layout of iPSCs in lab dishes. The proteins they encode help regulate interactions between cells, making them ideal candidates to alter the cellular organization of an iPSC group.

But there are so many variables to considerincluding the timing and level of each gene knockdown, the duration and the proportion of cells to work onthat make testing all the combinations by human almost impossible. So, they turned to machine learning for help.

RELATED:Growing transplantable arteries from stem cells

They used a CRISPR-Cas9 gene-editing system that could be triggered by adding the antibiotic doxycycline. To help link changes to specific arrangements of the iPSCs, the cells were also engineered to fluoresce in different colors when they lost ROCK1 or CDH1.

Researchers at Gladstone tested different doses and timing of gene blockade. How changes in cell subpopulations affected the observed pattern was captured, and the BU computational scientists fed the results to a machine learning algorithm, which was hence trained to classify patterns according to their similarity and infer ways of how ROCK1 and CDH1 affect iPSC organization.

Our machine-learning model allows us to predict new ways that stem cells can organize themselves, and produces instructions for how to recreate these predictions in the lab, the studys co-first author Demarcus Briers said in a statement.

The model simulated specific experimental conditionssuch as when, where and how to add drugs to the iPSCsthat could yield unique patterns in silico. Then, the team put those suggested conditions to test.

It was successful. The researchers were able to generate concentric circles to two layers of stem cell populations in a bulls-eye pattern, they reported.

We've shown how we can leverage the intrinsic ability of stem cells to organize, Todd McDevitt, the studys senior author, said in a statement. This gives us a new way of engineering tissues, rather than a printing approach where you try to physically force cells into a specific configuration.

RELATED:Nose drop with adult stem cells restores sense of smell in mice

Stem cells are a key venue for regenerative research, either for studying disease and potential treatment or for transplant. Last year, scientists from the University of Edinburgh used 3D scaffolds made of polycaprolactone to carry embryonic stem cells and iPSCs, and successfully generated functional liver tissues that help diseased mice break down the amino acid tyrosine. A research team at the Morgridge Institute for Research recently used a drug called RepSox to help iPSCs form better smooth muscle cells as building blocks for functional arteries.

For the Gladstone-BU team, the researchers are planning to expand the model to include other genes to get an even wider pool of possible cell configurations. On top of that, rather than just making flat patterns as in this study, their goal is to design 3D shapes or organs.

We're now on the path to truly engineering multicellular organization, which is the precursor to engineering organs, said McDevitt. When we can create human organs in the lab, we can use them to study aspects of biology and disease that we wouldn't otherwise be able to.

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ALS Stem Cell Therapy Developer Seeks Amendment to its AstroRx Trial – ALS News Today

Posted: November 25, 2019 at 8:45 pm

Kadimastem plans to submit an amendment to the protocol of its ongoing Phase 1/2a trial in Israel assessing the use of AstroRx, an off-the-shelf stem cell therapy, for the treatment of amyotrophic lateral sclerosis (ALS).

The company wants to change the planned dosage in one of the studys cohorts and test repeated injections of a low dose of AstroRx, instead of the medium dose that was originally planned.

The request is based on positive interim data from the first set of five patients given a single intrathecal (into the spinal canal) injection of the lowest dose (100 x 106 cells) of AstroRx. At this dose, the therapy was found to be safe with no serious side effects or dose-limiting toxicities identified, according to the data, which was released in September.

Moreover, AstroRx significantly reduced disease progression after three months of treatment, compared to the start (baseline) of the trial. The ALS Functional Rating Scale revised (ALSFRS-R) score decreased on average by 0.87 per month during the three months before treatment; however, it started to increase again (on average by 0.26 per month) in the three months after treatment. The ALSFRS-R score is a validated assessment of disability progression, with lower scores indicating greater motor impairment.

Evidence suggests that poorly working astrocytes (cells which support and protect neurons) are involved in the progression of ALS. AstroRx is composed of healthy functional astrocytes, which have been derived from human embryonic stem cells. The treatment, injected into a patients spinal fluid, is thought to compensate for the diseased astrocytes and prevent the death of motor neurons, thereby slowing disease progression.

AstroRx was granted orphan drug status by the U.S. Food and Drug Administration in November 2018 for the treatment of ALS.

Preclinical (in the lab) studies have shown that AstroRx was safe, delayed disease onset, maintained muscle function, and increased survival in rodent models of ALS.

The ongoing open-label Phase 1/2a clinical trial (NCT03482050) is testing the safety and effectiveness of AstroRx in ALS patients. The trial is being conducted at Hadassah Ein-Kerem Medical Center in Israel where it recruited 21 patients withearly stagedisease.

The trials original protocol included four doses of AstroRx delivered into the spinal canal: a low (100 x 106 cells), medium (250 x 106 cells), or high (500 x 106 cells) dose.

The primary outcome of the trial is to assess the safety and tolerability of AstroRx. Secondary outcome measures include changes in patients ALSFRS-R scores, respiratory muscle strength, hand grip strength, limb muscle strength, and quality of life.

In cohort A, participants received a single low dose of the therapy. In cohort B, participants received a single medium dose of the therapy. Results from cohort A are expected to be reported by the end of 2019, and cohort B results are expected in 2020.

Based on the positive interim results from cohort A, Kadimastem is seeking to amend the therapy regime being assessed in cohort C and D, so that cohort C will receive two injections of the low dose (instead of the originally planned medium dose), with the injections being separated by two to three months. Results from this section of the trial are expected to be reported in the first half of 2021. Under the amendment, cohort D participants will receive the regimen originally planned for cohort C (repeated administration of the medium dose, 250 x 106). Cohort D dosing will be dependent on the results of the previous cohorts.

We are the first to treat ALS patients with astrocyte cells. Following our positive interim results, we look forward to achieving a prolonged therapeutic effect in the repeated low dose administration, bringing new hope for patients with this incurable disease Rami Epstein, CEO of Kadimastem, said in a press release.

Kadimastem expects to submit an investigational new drug application to the U.S. Food and Drug Administration by mid-2021 with the aim of testing AstroRx in a multi-center clinical trial, which will compare the current and frozen version of the therapy.

Patricia holds a Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She has also served as a PhD student research assistant at the Department of Microbiology & Immunology, Columbia University, New York.

Total Posts: 279

Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Tcnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.

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2 Small-Cap Biotechs That Soared Last Week – Motley Fool

Posted: November 25, 2019 at 8:45 pm

The stocks of Magenta Therapeutics (NASDAQ:MGTA) and Molecular Templates (NASDAQ:MTEM) bolted skywards last week, to the tune of 39% and 28% respectively.

Cutting-edge gene-editing therapies, chimeric antigen receptor T-cell (CAR-T) treatments, and stem cell transplants all require priming or conditioning regimens. Doctors today utilize older chemotherapy drugs or radiation, which often lead to infection or hospitalization. Magenta Therapeutics and Molecular Templates are among the companies seeking to develop less toxic, non-chemotherapy options for patients.

Image source: Getty Images.

On Nov. 18, Molecular Templates and Vertex Pharmaceuticals (NASDAQ:VRTX) forged a discovery and development collaboration to create novel targeted conditioning regimens applicable to gene-editing, CAR-T, and stem cell transplants. Vertex shelled out $38 million of up-front cash and an equity investment in Molecular Templates. The stock barely flinched, losing $0.03 from the prior day's closing price.

The next day, Nov. 19, Vertex and its collaborator CRISPR Therapeutics announced positive safety and efficacy data for the gene-editing therapy CTX001 in its first two patients. One patient had severe sickle cell disease; the other had beta thalassemia. These interventions edit a patient's genome, potentially allowing for a one-time curative treatment. Both patients received the chemotherapy busulfan prior to CTX001.

Revisiting the prior day's collaboration announcement, biotech investors focused on comments made by Vertex about how Molecular Templates could benefit the CTX001 program.

Vertex's Chief Scientific Officer David Altshuler said,

"We believe that gene editing holds significant promise in the treatment of severe hemoglobinopathies such as sickle cell disease and beta thalassemia, and Molecular Templates' unique technology platform could play an important role in creating a targeted conditioning regimen that could replace chemotherapy currently required in conditioning regimens and thus enhance the overall future treatment experience for patients."

Investors jumped on the message from Vertex, one of the biotech industry's stalwarts: Non-chemotherapy conditioning approaches are the future for gene and cell therapies.

In response, the stocks of other companies focused on achieving that goal (like Magenta) shot up. In fact, Magenta's nearly 40% gain in share price came during a week when it didn't release any news.

Magenta plans to present data on Dec. 6 at the American Society of Hematology's Annual Meeting for its lead program CD117-ADC. Targeting a protein on hematopoietic stem cells called CD117, the treatment eliminated mutated cells without the need for chemotherapy or radiation. Magenta believes CD117-ADC can potentially be used for genetic diseases like sickle cell disease, prior to either gene therapy or hematopoietic stem cell transplantation (HSCT).

Magenta and Molecular Templates are not the only players in the field. Forty Seven and bluebird bio paired up earlier this month to develop antibody-based conditioning regimens for HSCT. According to the World Health Organization, 50,000 HSCT procedures are performed annually worldwide.

Furthermore, recently approved CAR-T for cancer, such as Kymriah from Novartis or Yescarta from Gilead Sciences, require three days of cyclophosphamide and fludarabine. Developers of these and next-generation CAR-T treatments also seek to eliminate chemotherapy or radiation.

Patients greatly need less toxic methods to prepare them for gene- and cell-based therapies, or stem cell and bone marrow transplants. Many patients, particularly the elderly, are deemed ineligible for these interventions because the toxicity could be too severe. Any success could have broad implications for the treatment of cancers and genetic diseases.

While a variety of successful approaches may ultimately emerge, Magenta has taken an early lead with CD117-ADC. Molecular Templates, with Vertex as a seasoned partner by its side, may soon leap onto the scene with a targeted approach derived from its "engineered toxin bodies" platform.

The investor takeaway is clear: New treatment modalities will be dependent on non-chemotherapy conditioning. Investors in biotech companies that can figure out that piece of the puzzle should be richly rewarded.

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Join the stem cell registry – Berea Mail

Posted: November 25, 2019 at 8:45 pm

Nicolene Moonsamy. Sheena Dhunlal and Stephanie Berry encourage people to attend the blood stem cell donor drive.

THE number of patients diagnosed with blood diseases is on the rise, with 75 per cent of those diagnosed under the age of 25 years.

For most of these patients, a blood stem cell transplant is their only hope of cure.

Lenmed Ethekwini Hospital and Heart Centre is partnering with The Sunflower Fund to host a blood stem cell donor drive on 27 November at 9.30am to recruit well informed and committed donors onto The Sunflower Fund Registry, The drive will take place in the auditorium on the fourth floor until 3pm.

Leukaemia, aplastic anaemia and immune deficiencies are among the few blood conditions that we treat in our practise and can be managed with a blood stem cell transplant. Stem cells make up the component that your immune system needs to function. During the transplant your body is rescued with an infusion of healthy blood stem cells, explained Dr Keshnie Moodley, treating specialist paediatrician and haematologist at KIDZCAN based at Lenmed Ethekwini Hospital and Heart Centre.

ALSO READ: Girls celebrate with The Sunflower Fund

The chances of finding this match is 1:100 000 and the best chance of a match is within your same ethnic background. There is only a 25 per cent chance that a sibling will be a match. The remaining 75 per cent chance depends on an unrelated matching donor being found. Patients of colour and mixed ethnicity around the world are at a disadvantage due to the under-representation of Black, Coloured, Indian and Asian donors in the global donor pool.

As such The Sunflower Fund proactively creates awareness and education about blood diseases as well as the process involved in becoming a blood stem cell donor. We remain a registry of donors that is representative of the people groups in our rainbow nation, who are willing to help anyone for whom they might be a match, said Stephanie Berry from The Sunflower Fund.

To attend the drive on 27 November, contact Xolani Hlongwane on 079 261 2664 or email: [emailprotected]

To become a blood stem cell donor, visit: http://www.sunflowerfund.org.

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Cell Isolation Market To Reach USD 18.26 Billion By 2026 | Reports And Data – GlobeNewswire

Posted: November 25, 2019 at 8:45 pm

New York, Nov. 25, 2019 (GLOBE NEWSWIRE) -- In the recent years, interest of biotechnology companies and scientists in cancer and stem cell research has increased. Advanced cell isolation products deliver improved separation of biological molecules including proteins, nucleic acids, chromatin and protein complexes for further analysis. In addition to this, there has been an increase in demand for personalized medicine. This has further helped this market grow.

According to the current analysis of Reports and Data, The global cell isolation market was valued at USD 4.9 billion in 2018 and is expected to reach USD 18.28 billion by the year 2026, at a CAGR of 17.7%. Cell isolation is the process of extracting a specialized cell from a heterogeneous mixture and then process it to identify its properties and replicate it to develop new therapies. For the determination of appropriate separation technique, an exhaustive analysis of the cell size, cell behavior, density, antigen status, and hydrophobic surface properties are done.

Cell isolation plays a very vital role in the diagnostics and research of chronic diseases. It helps in drug discovery by studying the behaviour of the cells and their response to disease and drugs. This technique of drug discovery helps to generate medicines that can be used for the treatment of various diseases such as cancer, genetic disorders, and autoimmune diseases. With incidents of chronic diseases on the rise across the world, the research, drug development, and clinical trials on various cell-based therapies also need to be increased. Therefore, the demand for cell isolation market will also have a boost.

This will be a significant factor fuelling the growth of the cell isolation market. The cell separation techniques play a vital role in personalized medicines, which are used for early detection of disease, selection of appropriate treatment, and determining the prognosis of the therapy. All these factors have contributed towards a positive dynamic growth curve of this market, and it is expected to keep growing in the coming years.

Request free sample of this research report at: https://www.reportsanddata.com/sample-enquiry-form/2237

Further key findings from the report suggest:

To identify the key trends in the industry, click on the link below: https://www.reportsanddata.com/report-detail/cell-isolation-market

Segments covered in the report:

For the purpose of this study, Reports and Data have segmented the cell isolation market on the basis of product, cell type, technique, application, end use and region:

Product (Revenue in USD Billion, 2018 - 2026)

Consumables

Reagents, kits, media, and sera

Beads

Disposables

Instruments

Centrifuges

Flow cytometers

Filtration systems

Magnetic-activated cell separator systems

Delivery Mode Type (Revenue, USD Million; 20162026)

Human cells

Differentiated Cells

Stem Cells

Animal Cells

Technique (Revenue in USD Billion, 2018 - 2026)

Application (Revenue in USD Billion, 2018 - 2026)

End Use (Revenue in USD Billion, 2018 - 2026)

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Regional Outlook (Revenue in USD Million; 20162026)

North America

o U.S.

o Canada

Europe

o Germany

o France

o UK

o Spain

o Italy

o Rest of the Europe

Asia Pacific

o China

o India

o Japan

o Rest of Asia-Pacific

Middle East & Africa

o Latin America

Brazil

About Reports and Data

Reports and Data is a market research and consulting company that provides syndicated research reports, customized research reports, and consulting services. Our solutions purely focus on your purpose to locate, target and analyze consumer behavior shifts across demographics, across industries and help clients make a smarter business decision. We offer market intelligence studies ensuring relevant and fact-based research across a multiple industries including Healthcare, Technology, Chemicals, Power, and Energy. We consistently update our research offerings to ensure our clients are aware about the latest trends existent in the market. Reports and Data has a strong base of experienced analysts from varied areas of expertise.

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Comparative Study of the Therapeutic Potential of Mesenchymal Stem Cells Derived from Adipose Tissue and Bone Marrow on Acute Myocardial Infarction…

Posted: November 25, 2019 at 8:45 pm

OBJECTIVES:

Stem cell therapy is a promising approach in the treatment of acutemyocardial infarction(AMI). Mesenchymal stem cells (MSC) from bone marrow (BM-MSC) and adipose tissue (AT-MSC) are attractive and feasible for preclinical and clinical trials. In this study, we compared the therapeutic potential of BM-MSC and AT-MSC in repairing the hearts of rats with isoproterenol (ISO)-induced AMI.

Forty-two female rats were assigned into two groups; the optimization and the experimental group. The optimization groups were further subdivided into control group and the AMI induced group (using ISO). The experimental group was subdivided into AMI+cell-free media injected in the tail vein, AMI+BM-MSC, and AMI+AT-MSC groups treated with the intravenous injection of their respective cell types. Twenty-eight days after induction, electrocardiogram (ECG) was performed, and heart tissue samples were collected for histological assessment and cells tracing.

MSC therapy repaired cardiac functions shown by the restoration of ST segment, QT and QRS intervals in the ECG when compared to the AMI group. Infarct area was significantly decreased, and cardiac tissue regeneration signs were shown on histopathological examination.

Both MSC sources proved to be equally efficient in the assessed parameters.

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Comparative Study of the Therapeutic Potential of Mesenchymal Stem Cells Derived from Adipose Tissue and Bone Marrow on Acute Myocardial Infarction...

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Liberty Science Center’s Inaugural Genius of New Jersey to Honor Innovators Who Make the State a World Leader in Cutting-Edge Applied Science -…

Posted: November 25, 2019 at 8:45 pm

JERSEY CITY, N.J.--(BUSINESS WIRE)--New Jersey is home to some of the worlds most accomplished innovators in applied science. Three of them who are pioneering research and solutions in antibacterial therapies, genetics, human life extension, and food production are being honored by Liberty Science Center at its inaugural The Genius of NJ celebration on Monday, December 2.

The celebration starts at 5:30 pm with cocktails and unique technology demonstrations: a full-body 3D scanner from Lenscloud that can scan a person in half a second with 120 cameras and create a realistic 3D avatar; bomb-disposing robots and an autonomous fighting robot from Picatinny Arsenal; and Flyer, a personal aerial vehicle from Kitty Hawk, headquartered in Mountain View, CA.

The New Jersey honorees are Bonnie Bassler, Chair of Molecular Biology at Princeton University, who is developing novel antimicrobial therapies to render pathogenic bacteria harmless; Dr. Robert J. Hariri, Chairman, Founder & CEO of Celularity, Inc. who is pioneering the use of stem cells to cure disease and slow aging; and David Rosenberg, CEO and Co-Founder of AeroFarms, the worlds leader in mass-scale vertical indoor farming.

Our inaugural Genius of NJ Award Winners represent the best this state and the world have to offer in harnessing science for the betterment of humanity, said Liberty Science Center President and CEO Paul Hoffman. Each is using his or her exceptional intellect and creative abilities to disrupt and innovate both in their respective fields and in their commitment to making the world healthier and safer.

Bonnie Bassler is the Squibb Professor of Molecular Biology and Chair of the Department of Molecular Biology at Princeton University, as well as a Howard Hughes Medical Institute Investigator. Professor Bassler deciphered the chemical language bacteria cells use to communicate by studying a harmless marine bacterium called Vibrio fischeri, known to bioluminesce, or make light, like fireflies do. She is a winner of the MacArthur Genius Grant and is now developing therapies that disrupt communication among harmful bacteria and strengthen communication among helpful bacteria. At a time when an increasing number of bacteria are resistant to traditional kinds of antibiotics, Dr. Bassler offers a promising new approach to antimicrobial therapy.

The Chairman, Founder and CEO of Celularity, Inc., in Warren, NJ, and Co-Founder and Vice Chairman of Human Longevity, Inc., Dr. Robert Hariri is the quintessential renaissance man. Hes a neurosurgeon, a medical researcher, and a serial entrepreneur in two technology sectors: aerospace and biomedicine. Dr. Hariri has advised the Vatican on genetics, and in 2018, Pope Francis bestowed on him the Pontifical Key Award for Innovation. Dr. Hariris path to discovering that the placenta, a temporary organ discarded after birth, was a potent source of stem cells began in the 80s when he viewed a first trimester ultrasound of his oldest daughter and wondered why the placenta was so large. Today Dr. Hariri is working to use placental stem cells to cure disease, slow aging, and augment healthy human lifespan.

Prominent entrepreneur David Rosenberg, CEO and Co-Founder of AeroFarms, set out to reinvent one of the most basic aspects of food production, farming. AeroFarms has grown 800 species of plants indoors and can grow them 365 days a year without sun or soil, achieving yields 130 times greater than conventional farming. His system uses 95 percent less water than field farming and no pesticides, herbicides, or fungicides. Rosenbergs adoption of cutting-edge technology has been a cornerstone of AeroFarms, which set up its first indoor vertical farms in abandoned warehouses in Newark. He employs plant biologists, microbiologists, geneticists, systems engineers, and data scientists. AeroFarms innovations in indoor vertical farming have improved not just plant yields but also taste, texture, nutritional density, and shelf life.

Additionally, LSC will honor non-New Jersian Sebastian Thrun, CEO of Kitty Hawk, a company spun off from a Google moonshot effort to free the world from traffic. Kitty Hawk is developing all-electric, vertical take-off flying machines for everyday use. Known as the godfather of self-driving cars, as a Stanford professor in 2005, Thrun led a team that won the $2-million Defense Department Grand Challenge to build an autonomous vehicle which drove itself unassisted on a 132-mile course across the Mojave Desert. His winning entry, Stanley, is now on display at the Smithsonian in Washington, DC. While at Stanford, in 2011 he and colleague Peter Norvig offered their Introduction to Artificial Intelligence course online to anyone, for free. Over 160,000 students in more than 190 countries enrolled! The MOOC (which stands for Massive Open Online Course) was born, and Thrun founded the online education company Udacity, with the goal of democratizing education. Thrun relinquished his tenured Stanford professorship to join Google and founded the companys semi-secret R&D division called Google X (now called simply X) to develop breakthrough technologies, such as self-driving cars, that make the world a radically better place.

Ticket prices for The Genius of NJ start at $750 per guest with options for table sponsorship from $12,500 to $50,000. For more details, please visit The Genius of NJ online. All proceeds from this event will support LSCs mission to inspire the next generation of scientists and engineers.

About Liberty Science Center

Liberty Science Center (LSC.org) is a 300,000-square-foot nonprofit learning center located in Liberty State Park on the Jersey City bank of the Hudson near the Statue of Liberty. Dedicated to inspiring the next generation of scientists and engineers and bringing the power, promise, and pure fun of science and technology to learners of all ages, Liberty Science Center houses the largest planetarium in the Western Hemisphere, 12 museum exhibition halls, a live animal collection with 110 species, giant aquariums, a 3D theater, live simulcast surgeries, a tornado-force wind simulator, K-12 classrooms and labs, and teacher-development programs. More than 250,000 students visit the Science Center each year, and tens of thousands more participate in the Centers off-site and online programs. Welcoming more than 750,000 visitors annually, LSC is the largest interactive science center in the NYC-NJ metropolitan area.

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Liberty Science Center's Inaugural Genius of New Jersey to Honor Innovators Who Make the State a World Leader in Cutting-Edge Applied Science -...

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Company claims signs of success with CRISPR-edited stem cell transplants for two genetic diseases – Science Magazine

Posted: November 19, 2019 at 10:52 pm

By Jon CohenNov. 19, 2019 , 5:40 PM

Companies and academic groups have begun to use the genome editor CRISPR to try to correct various diseases, but none has so far published, or even announced, evidence that patients have been helpeduntil this week. In an update for investors today, CRISPR Therapeutics of Zug, Switzerland, reported that one patient with sickle cell anemia and another with beta thalassemia appear to have benefited from the same CRISPR-based intervention for up to 9 months, STAT reports. (The company gave STAT an early look at the data but did not allow outside commenters to see the results.) Before the treatment, both patients required multiple infusions each year of red blood cells. CRISPR Therapeutics, collaborating with Vertex Pharmaceuticals, removed blood stem cells from their bodies and modified them with CRISPR to knock out a gene that shuts down production of fetal hemoglobin. When the edited cells were put back in each patients body through a stem cell transplantwhich required a toxic chemotherapy to kill their own stem cellsboth people produced high levels of fetal hemoglobin and no longer needed transfusions. These early findings are a triumph for patients, CRISPR, and genetics, Bruce Conklin, a clinician who focuses on stem cell treatments of cardiovascular disease at the University of California, San Francisco, told Science after reviewing the limited data the companies provided in the call and a press release. They chose patients with severe versions of the disease and the results are truly remarkable. Other companies are working on competing gene therapy and gene-editing technologies for the same disorders.

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Company claims signs of success with CRISPR-edited stem cell transplants for two genetic diseases - Science Magazine

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