Category Archives: Genetic Engineering

PUBLIC RELEASE DATE:

21-Aug-2014

Contact: Kathryn Ryan kryan@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, August 21, 2014--Mesenchymal stem cells (MSCs) are present in virtually every type of human tissue and may help in organ regeneration after injury. But the theory that MSCs are released from the bone marrow into the blood stream following organ damage, and migrate to the site of injury, has long been debated. M.J. Hoogduijn and colleagues provide conclusive evidence to resolve the controversy over the mobilization and migration of MSCs in humans in a new study published in Stem Cells and Development, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available on the Stem Cells and Development website.

In "No Evidence for Circulating Mesenchymal Stem Cells in Patients with Organ Injury," Hoogduijn and coauthors from Erasmus University Medical Center (Rotterdam, The Netherlands), describe the results of studies to detect MSCs in the blood of healthy individuals, of patients with end-stage renal disease, of patients with end-stage liver disease, and of heart transplant patients with organ rejection. Whereas they did not find MSCs in the circulation of these individuals, they did report the presence of MSCs in the blood of a patient suffering from severe trauma with multiple fractures. In the trauma patient, the circulating MSCs likely derived from disruption of the bone marrow caused by the fractures.

"We can add the simple but elegant work of Martin Hoogduijn to the pantheon of studies in stem cell research that skewer a long treasured tenet of faith and consign it to mythology," says Editor-in-Chief Graham C. Parker, PhD, The Carman and Ann Adams Department of Pediatrics, Wayne State University School of Medicine, Detroit, MI.

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About the Journal

Stem Cells and Development is an authoritative peer-reviewed journal published 24 times per year in print and online. The Journal is dedicated to communication and objective analysis of developments in the biology, characteristics, and therapeutic utility of stem cells, especially those of the hematopoietic system. A complete table of contents and free sample issue may be viewed on the Stem Cells and Development website.

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Conclusive evidence on role of circulating mesenchymal stem cells in organ injury

PUBLIC RELEASE DATE:

24-Jul-2014

Contact: Kathryn Ruehle kruehle@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, July 24, 2014Malcolm K. Brenner, MD, PhD, Baylor College of Medicine (Houston, TX) has devoted his career in basic and clinical research toward understanding how tumors are able to escape detection by the body's immune defense system, and developing genetically modified T cells that can effectively target tumors. In recognition of his scientific achievements and leadership in the field, Dr. Brenner is the recipient of a Pioneer Award from Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. Human Gene Therapy is commemorating its 25th anniversary by bestowing this honor on the leading 12 Pioneers in the field of cell and gene therapy selected by a blue ribbon panel* and publishing a Pioneer Perspective by each of the award recipients. The Perspective by Dr. Brenner is available on the Human Gene Therapy website.

In "Gene Modified Cells for Stem Cell Transplantation and Cancer Therapy", Dr. Brenner recounts the highlights of his career to date. He describes the evolution of his research, which has contributed significantly to advancing the field of gene transfer using retroviral vectors in the development of both autologous (AUTO) and allogeneic (ALLO) hematopoietic stem cell transplantation (HSCT) approaches to cancer immunotherapy, and the strategy of using chimeric antigen receptors (CARs) to modify T cells stimulating their activation, proliferation, and anti-tumor activity.

Dr. Brenner received a PhD in immunology and early in his career sought to understand how B cells interact with T cells to produce antibodies. After pursuing the development of cellular therapies to treat immune disorders, Dr. Brenner shifted the focus of his research to bone marrow transplantation, or what is now called HSCT. Together with colleagues he developed and tested an approach to improve patients' immune recovery after their T cells are depleted in preparation for a transplant. As Dr. Brenner explains, "This work was the forerunner of our later efforts to improve antiviral and antitumor immunity by adoptive transfer of T cells."

"Malcolm has been driving the field of cell-based gene therapy forward since its infancy. His contributions have been truly seminal," says James M. Wilson, MD, PhD, Editor-in-Chief of Human Gene Therapy and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia.

*The blue ribbon panel of leaders in cell and gene therapy, led by Chair Mary Collins, PhD, MRC Centre for Medical Molecular Virology, University College London selected the Pioneer Award recipients. The Award Selection Committee selected scientists that had devoted much of their careers to cell and gene therapy research and had made a seminal contribution to the field--defined as a basic science or clinical advance that greatly influenced progress in translational research.

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Malcolm K. Brenner receives Pioneer Award for advances in gene-modified T cells targeting cancer

PUBLIC RELEASE DATE:

23-Jul-2014

Contact: Kathryn Ruehle kruehle@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY -- The potential for clinical use of induced pluripotent stem cell (iPSC) technology for transplant-based therapeutic strategies has previously been hindered by the risk of dysregulated cell growth, specifically the development of tumors. The ability to use etoposide treatment to halt teratoma formation in iPSCs for the treatment of heart disease, specifically acute myocardial infarction, is demonstrated in an article in Stem Cells and Development, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available on the Stem Cells and Development website.

In the article 'Inhibition of DNA topoisomerase II selectively reduces the threat of tumorigenicity following induced pluripotent stem cell-based myocardial therapy' Saranya Wyles, Andre Terzic, Timothy Nelson, and coauthors, Mayo Clinic (Rochester, MN), discovered a strategy that alone or in conjunction with other methods could significantly reduce the risk of a tumorigenic event occurring. Their work demonstrates how pretreatment with genotoxic etoposide significantly lowered the threat of abnormal growths by removing the contaminated pluripotent cells and establishing an adjunctive therapy to further harness the clinical value of iPSC-derived cardiac regeneration.

"For anyone seeking to exploit iPSC technology in a clinical setting, the Mayo Clinic has described a strategy that significantly mitigates the risk of tumor development. Furthermore, the paper provides benchmark strategies for assessing the localization and persistence of cell-based treatments in a preclinical model," says Editor-in-Chief Graham C. Parker, PhD, The Carman and Ann Adams Department of Pediatrics, Wayne State University School of Medicine, Detroit, MI.

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About the Journal

Stem Cells and Development is an authoritative peer-reviewed journal published 24 times per year in print and online. The Journal is dedicated to communication and objective analysis of developments in the biology, characteristics, and therapeutic utility of stem cells, especially those of the hematopoietic system. Complete tables of content and a free sample issue may be viewed on the Stem Cells and Development website.

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New method for reducing tumorigenicity in induced pluripotent stem-cell based therapies

PUBLIC RELEASE DATE:

22-Jul-2014

Contact: Kathryn Ruehle kruehle@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY -- Stem cells offer much promise for treating damaged organs and tissues, but with current transplantation approaches stem cell survival is poor, limiting their effectiveness. New methods are being developed and tested to improve the survival and optimize their therapeutic function after transplantation, as described in a Review article in BioResearch Open Access, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the BioResearch Open Access website.

In the article 'Preconditioning Stem Cells for In Vivo Delivery,' Sbastien Sart, Ecole Polytechnique (Palaiseau, France) and Teng Ma and Yan Li, Florida State University (Tallahassee) examine the leading strategies for preconditioning stem cells prior to transplantation to prepare them for the environment often found in damaged tissue. Preconditioning methods might include exposing stem cells to microenvironments characterized by reduced oxygen levels, heat shock, and oxidative stress, creating three-dimensional stem cell aggregates or microtissues, and using hydrogels in which to embed or encapsulate the cells.

"This article provides an extensive review of the current methods of stem cell preconditioning for transplantation," says BioResearch Open Access Editor Jane Taylor, PhD, MRC Centre for Regenerative Medicine, University of Edinburgh, Scotland. "It also highlights the cutting edge technologies employed to do this."

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About the Journal

BioResearch Open Access is a bimonthly peer-reviewed open access journal led by Editor-in-Chief Robert Lanza, MD, Chief Scientific Officer, Advanced Cell Technology, Inc. and Editor Jane Taylor, PhD. The Journal provides a new rapid-publication forum for a broad range of scientific topics including molecular and cellular biology, tissue engineering and biomaterials, bioengineering, regenerative medicine, stem cells, gene therapy, systems biology, genetics, biochemistry, virology, microbiology, and neuroscience. All articles are published within 4 weeks of acceptance and are fully open access and posted on PubMedCentral. All journal content is available on the BioResearch Open Access website.

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Novel methods may help stem cells survive transplantation into damaged tissues

PUBLIC RELEASE DATE:

30-Jun-2014

Contact: Kathryn Ruehle kruehle@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, June 30, 2014Zebrafish, a model organism that plays an important role in biological research and the discovery and development of new drugs and cell-based therapies, can form embryonic stem cells (ESCs). For the first time, researchers report the ability to maintain zebrafish-derived ESCs for more than 2 years without the need to grow them on a feeder cell layer, in a study published in Zebrafish, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the Zebrafish website.

Ho Sing Yee and coauthors from the Malaysian Ministry of Science, Technology and Innovation (Pulau Pinang), Universiti Sains Malaysia (Penang), and National University of Singapore describe the approach they used to be able to maintain zebrafish stem cells in culture and in an undifferentiated state for long periods of time. The ability to establish and grow the zebrafish ESCs without having a feeder layer of cells to support them simplifies their use and could expand their utility. In the article "Derivation and Long-Term Culture of an Embryonic Stem Cell-Like Line from Zebrafish Blastomeres Under Feeder-Free Condition", the authors show that the ESCs retain the morphology, properties, and ability to differentiate into a variety of cell types that is characteristic of ESCs, and were used to generate offspring after transmission through the germline.

"By addressing a major technical bottleneck in the field, this new culture system enables an array of exciting cellular and molecular genetic manipulations for the zebrafish," says Stephen Ekker, PhD, Editor-in-Chief of Zebrafish and Professor of Medicine at Mayo Clinic, Rochester, MN.

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About the Journal

Zebrafish is an authoritative peer-reviewed journal published bimonthly in print and online. It is the only peer-reviewed journal to focus on the zebrafish and other aquarium fish species as models for the study of vertebrate development, evolution, toxicology, and human disease. Tables of content and a sample issue may be viewed on the Zebrafish website.

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New method to grow zebrafish embryonic stem cells can regenerate whole fish

PUBLIC RELEASE DATE:

20-Jun-2014

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 x2156 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, June 20, 2014Spiral ganglion cells are essential for hearing and their irreversible degeneration in the inner ear is common in most types of hearing loss. Adult spiral ganglion cells are not able to regenerate. However, new evidence in a mouse model shows that spiral ganglion stem cells present in the inner ear are capable of self-renewal and can be grown and induced to differentiate into mature spiral ganglion cells as well as neurons and glial cells, as described in an article in BioResearch Open Access, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the BioResearch Open Access website.

Marc Diensthuber and coauthors from Goethe-University (Frankfurt, Germany), Justus-Liebig University (Giessen, Germany), Harvard Medical School and Massachusetts Eye and Ear Infirmary (Boston, MA), and Harvard University and MIT (Cambridge, MA), conclude that the self-renewing properties demonstrated by spiral ganglion stem cells make them a promising source of replacement cells for therapies designed to regenerate the neural structures of the inner ear in the article "Spiral Ganglion Stem Cells Can Be Propagated and Differentiated Into Neurons and Glia."

"These findings are particularly interesting as they show that spiral ganglion stem cells can be propagated in vitro," says BioResearch Open Access Editor Jane Taylor, PhD, MRC Centre for Regenerative Medicine, University of Edinburgh, Scotland. "These cells are normally poorly regenerated in the mammalian ear."

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About the Journal

BioResearch Open Access is a bimonthly peer-reviewed open access journal led by Editor-in-Chief Robert Lanza, MD, Chief Scientific Officer, Advanced Cell Technology, Inc. and Editor Jane Taylor, PhD. The Journal provides a new rapid-publication forum for a broad range of scientific topics including molecular and cellular biology, tissue engineering and biomaterials, bioengineering, regenerative medicine, stem cells, gene therapy, systems biology, genetics, biochemistry, virology, microbiology, and neuroscience. All articles are published within 4 weeks of acceptance and are fully open access and posted on PubMed Central. All journal content is available on the BioResearch Open Access website.

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Inner ear stem cells hold promise for restoring hearing

Take a hot new method that's opened up a new era of genetic engineering, apply it to the wonder stem cells that in 2012 won their discoverer a Nobel prize, and you might just have a tool to cure HIV infection.

That's the hope of researchers led by Yuet Kan of the University of California, San Francisco and they have proved the basic principle, altering the genome of induced pluripotent stem cells (iPSCs) to give them a rare natural mutation that allows some people to resist HIV.

Kan's work relies on "genome editing" snipping out a particular DNA sequence and replacing it with another. It's much more precise than traditional forms of genetic engineering, in which sequences are added to the genome at random locations.

To alter the stem cells, Kan's team turned to the CRISPR-Cas9 system, a super-efficient method of genome editing based on an ancient bacterial "immune system". In bacteria, the system takes fragments of DNA from invading viruses and splices them into the cell's own DNA, where they act like "wanted" posters, allowing the viruses to be recognised and attacked in future.

About 1 per cent of people of European descent are resistant to HIV, because they carry two copies of a mutation in the gene for a protein called CCR5. The virus must lock onto this protein before it can invade white blood cells, and the mutations prevent it from doing so.

Using a bone marrow transplant from a naturally HIV-resistant person, Timothy Ray Brown was famously "cured" of HIV infection. Kan's goal is to achieve the same result without the need to find compatible HIV-resistant bone marrow donors who are in vanishingly short supply.

It's fairly easy to make iPSCs from a person's cells, which then have the potential to grow into any type of cell in the body. So if iPSCs could be given two copies of the protective mutation, it should be possible to make personalised versions of the therapy that cleared HIV from Brown's body. Kan's team has now shown that CRISPR-Cas9 can efficiently make the necessary genome edit. As expected, white blood cells grown from these altered stem cells were resistant to HIV upon testing.

"It's a really fantastic application of the tool," says Philip Gregory, chief scientific officer with Sangamo BioSciences of Richmond, California. However, he warns that there is a long way to go before it can be turned into a practical therapy.

Kan has not yet grown the iPSCs into the specific type of white blood cells called CD4+ T cells that are ravaged by HIV. What he instead plans to do is turn the iPSCs into blood-forming stem cells, which when transplanted into the body would give rise to all of the cell types found in the blood. "One of the problems is converting iPSCs into a type of cell that is transplantable," says Kan. "It is a big hurdle."

Regulators will also need to be convinced that cells that have been subjected to extensive genetic manipulation both to create the iPSCs, and to give them the protective mutation are safe.

Read more:
Gene editing tool can write HIV out of the picture

TIME Health Heart Disease

Doctors, and especially doctors who do research, dont like to use the words cure or eradicate. They know how dangerous that can be, since the human body is so unpredictable. But Dr. Kiran Musunuru is showing some uncharacteristic swagger about his latest success in lowering heart attack risk among some lucky mice.

Taking advantage of advances in genetic engineering, a team lead by Musunuru, who holds positions at Harvard Universitys Department of Stem Cell and Regenerative Biology and Brigham and Womens Hospital, have edited the genomes of mice and successfully protected them from heart disease. The results, published in the journal Circulation Research, hint at an entirely new way of avoiding the leading killer of Americans by possibly cutting heart attack risk by up to 90%. What has me excited as a cardiologist is that my goal is eradicating disease, says Musunuru. There is no bolder way I can put it. I want to eradicate the disease and this offers one potential way to do it.

MORE: Experimental Cholesterol-Lowering Drug Shows Promise

He admits that it may be 10 years or more before the technique is ready for testing in people, but these first results are enough to justify the research that could make that happen. This approach in general will be a game changer, says Dr. Deepak Srivastava, director of cardiovascular disease and stem cell biology and regenerative medicine at the Gladstone Institutes, who was not affiliated with the study.

Heres how they did it. In 2003, genetic information was gleaned from a French family that carried a genetic mutation giving them low LDL cholesterol, the kind that, when its high, can lead to heart disease. Using a new genetic engineering technique that allows scientists to splice more efficiently into specific locations on a genome, Musunuru was able to essentially bestow the genetic advantage from the French family onto his mice, slowing down production of a protein that normally keeps LDL circulating in the blood. With less of the protein around, less LDL remains in the blood; those with the PCSK9 mutation showed as much as an 88% lower risk of heart disease compared to people without the genetic change.

The genetic monkeying was accomplished with the help of a virus, which has a remarkable ability to get into cells. The virus was injected, along with the DNA-disrupting machinery, into the liver of the mice. Within days, more than half of the liver cells had been genetically edited and the mice showed 35% to 40% less cholesterol in the blood.

So far, says Musunuru, there have been no negative effects of the genetic disruption. But he says more research needs to be done to make sure that introducing the changes wont come with unforeseen consequences. When we go in there we want to make sure we are not introducing new spelling errors in the genome, says Srivastava, who is also using the technique for stem-cell based therapies to treat heart disease. Says Musunuru, I think I can confidently say that with this tool, this technology will work on live, breathing human beings, but we need to figure out the safety; thats the barrier to overcome before we can test these therapies.

MORE: Who Really Needs To Take a Statin?

Drug companies are also working on drug-based ways to interfere with PCSK9, and lower LDL levels, but those therapies are antibodies that bind to the protein that the gene makes and need to be injected, at a doctors office, regularly. The genome editing strategy would be a one-stop therapy that could permanently protect against excessively high cholesterol levels.

Read more here:
A Vaccine for Heart Disease Could Mean No Pills, Lettuce or a Gym

John Sterling | 05/01/2014

The following article, reproduced in full below, was originally published at Genetic Engineering & Biotechnology News.

Its been a hot year for biotech! As G. Steven Burrill, CEO of Burrill & Co., noted in a recent report, life science firms raised $2.9 billion in new equity capital globally from public investors in February. This included $1.1 billion raised by 18 companies that completed initial public offerings and $1.8 billion raised by 23 companies that completed follow-on offerings during the month.

In the U.S., 16 life sciences companies raised $959 million through IPOs and 22 companies raised $1.75 billion through follow-on offerings on U.S. exchanges during February, making the month the biggest for IPOs in terms of the number of completed deals since February 2000!

Why the excitement? Promising new biotherapeutics are emerging from the drug pipeline. Advances in stem cell research and regenerative medicine are occurring at a rapid pace. And OMICS technologies (e.g., genomics, proteomics, metabolomics, transcriptomics, glycomics, and lipomics), originally developed and used in the lab, are now making their way into clinical medicine, truly ready to usher in an era of personalized medicine.

The 2014 BIO International Convention will be held in San Diego this June. As usual, the BIO conference committee did a superb job in putting together a first-class program that covers a wide range of topics with something to offer everyone involved in biotech R&D or commercialization. Its been a tough call this year but here are my picks for the top 10 cant miss sessions at the conference.

To learn more about the program and available registration packages for Convention, please visithere

John Sterling is editor-in-chief of Genetic Engineering & Biotechnology News (GEN).

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GENs Top 10 Session Picks for the 2014 BIO International Convention

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PUBLIC RELEASE DATE:

17-Apr-2014

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 x2156 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, April 17, 2014The development of stem cell therapies to cure a variety of diseases depends on the ability to characterize stem cell populations based on cell surface markers. Researchers from the Finnish Red Cross have discovered a new marker that is highly expressed in a type of stem cells derived from human umbilical cord blood, which they describe in an article in BioResearch Open Access, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the BioResearch Open Access website.

Heli Suila and colleagues, Finnish Red Cross Blood Service, Helsinki, Finland present evidence to show that the glycan O-GLcNAc, is present on the surface of stem cells and is part of a stem cell-specific surface signature. In the article "Extracellular O-Linked N-Acetylglucosamine Is Enriched in Stem Cells Derived from Human Umbilical Cord Blood" the authors suggest that the glycan plays a crucial role in a cell signaling pathway that regulates embryonic development.

"This work is particularly interesting as epidermal growth factor domains are found on the Notch receptors, suggesting that these novel glycans may be involved in Notch receptor signaling pathways in stem cells," says BioResearch Open Access Editor Jane Taylor, PhD, MRC Centre for Regenerative Medicine, University of Edinburgh, Scotland.

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About the Journal

BioResearch Open Access is a bimonthly peer-reviewed open access journal led by Editor-in-Chief Robert Lanza, MD, Chief Scientific Officer, Advanced Cell Technology, Inc. and Editor Jane Taylor, PhD. The Journal provides a new rapid-publication forum for a broad range of scientific topics including molecular and cellular biology, tissue engineering and biomaterials, bioengineering, regenerative medicine, stem cells, gene therapy, systems biology, genetics, biochemistry, virology, microbiology, and neuroscience. All articles are published within 4 weeks of acceptance and are fully open access and posted on PubMedCentral. All journal content is available on the BioResearch Open Access website.

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Novel marker discovered for stem cells derived from human umbilical cord blood