Volume 15.30 | Aug 4

Issue 15.30 August 4, 2014
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Key to Aging Immune System Is Discovered
Through cycles of cell division hematopoietic stem cells preserve their own numbers and generate the daughter cells that give rise to replacement blood and immune cells. Especially vulnerable to the breakdown, researchers discovered in their new study of old mice, are transplanted, aging, blood-forming stem cells, which lack the ability to make B cells of the immune system. [Press release from UCSF discussing online prepublication in Nature] Press Release | Abstract
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PUBLICATIONS (Ranked by impact factor of the journal)
In Utero Gene Therapy Rescues Microcephaly Caused by Pqbp1-Hypofunction in Neural Stem Progenitor Cells
Researchers demonstrated that in utero gene therapy for conditional Pqbp1-knockout (cKO) mice by intraperitoneal injection of the PQBP1-AAV vector at E10 successfully rescued microcephaly with preserved cortical structures and improved behavioral abnormalities in Pqbp1-cKO mice. [Mol Psych] Full Article

MicroRNA-21 Coordinates Human Multipotent Cardiovascular Progenitors Therapeutic Potential
Injection of only 104 human embryonic stem cells-derived SSEA-1+/MesP1+ cells, or their progeny obtained after treatment with VEGF-A or PDGF-BB, was effective enough to enhance post-ischemic revascularization in immunodeficient mice with critical limb ischemia. [Stem Cells] Abstract

WNT3A Promotes Myogenesis of Human Embryonic Stem Cells and Enhances In Vivo Engraftment
Scientists describe the use of WNT3A protein to promote in vitro myogenic commitment of human embryonic stem cells-derived cells and their subsequent in vivo function. In vivo transplantation of these committed cells into cardiotoxin-injured skeletal muscles of NOD/SCID mice reveals survival and engraftment of the donor cells. [Sci Rep] Full Article

HLA and KIR Genotyping Correlates with Relapse after T-Cell-Replete Haploidentical Transplantation in Chronic Myeloid Leukemia Patients
Scientists prospectively analyzed the human leukocyte antigen (HLA) typing of donor-recipient pairs and the KIR typing of the donors in 97 CML patients to address the predictive roles of natural killer cells in relapse undergoing T-cell-replete haploidentical transplantation. [Br J Cancer] Abstract

Re-Engineered p53 Activates Apoptosis In Vivo and Causes Primary Tumor Regression in a Dominant Negative Breast Cancer Xenograft Model
Researchers demonstrate the superior tumor suppressor activity of chimeric superactive p53 and its ability to cause tumor regression of the MDA-MB-468 aggressive p53-dominant negative breast cancer tumor model in vivo. [Gene Ther] Abstract

Combining Enriched Environment and Induced Pluripotent Stem Cell Therapy Results in Improved Cognitive and Motor Function following Traumatic Brain Injury
Scientists evaluated enriched environment (EE) and induced pluripotent stem cells (iPSC) as a polytherapy for remediating cognitive deficits following medial frontal cortex controlled cortical impact (CCI) injury. Sixty adult male rats received a midline mFC CCI or sham injury and were randomly placed in either EE or standard environment. Seven days post-injury rats received bilateral transplantation of iPSCs or media. [Restor Neurol Neurosci] Abstract

Therapeutic Effects of Mesenchymal Stem Cell-Derived Microvesicles on Pulmonary Arterial Hypertension in Rats
Scientists investigated the therapeutic effects of mesenchymal stem cell derived microvesicles on pulmonary artery hypertension in rats. [Acta Pharmacol Sin] Abstract

Ex Vivo Expanded Autologous Polyclonal Regulatory T Cells Suppress Inhibitor Formation in Hemophilia
Adoptive cell therapy utilizing ex vivo expanded polyclonal CD4+CD25+FOXP3+ regulatory T cells is in use in clinical trials for the treatment of type 1 diabetes and prevention of graft versus host disease in bone marrow transplantation. Scientists evaluated this approach in the treatment of inherited protein deficiencies, i.e., hemophilia, which is often complicated by antibody formation against the therapeutic protein. [Mol Ther Methods Clin Dev] Full Article

Induced Neural Stem Cells Achieve Long-Term Survival and Functional Integration in the Adult Mouse Brain
Researchers show an in vivo long-term analysis of transplanted induced neural stem cells (iNSCs) in the adult mouse brain. iNSCs showed sound in vivo long-term survival rates without graft overgrowths. [Stem Cell Rep]
Full Article | Graphical Abstract | Press Release

Culture and Characterize Human Mammary Epithelial Progenitors with Serum-Free EpiCult-B (Human) - Free Protocols
Genomic Instability in Human Stem Cells: Current Status and Future Challenges
Genomic instability is recognized as one of the most important hurdles in the expanding field of stem cell-based therapies. Researchers address the current status and emerging trends of the field, ultimately underscoring the need to implement new regulatory standards that are able to streamline the route to therapeutic applications. [Stem Cells] Abstract

Caveats of Mesenchymal Stem Cell Therapy in Solid Organ Transplantation
The authors address the potential pitfalls for the use of mesenchymal stem cells (MSCs) as a therapeutic agent to control alloimmune effects. The aim of this review is not to discourage investigators from clinical studies, but to raise awareness of the intrinsic risks of MSC therapy. [Transpl Int] Abstract

Improved Mouse Models to Assess Tumor Immunity and irAEs after Combination Cancer Immunotherapies
The authors discuss the immune-related adverse events (irAEs) observed in patients after checkpoint blockade and discuss which mouse models of cancer can be appropriate to assess the development of tumour immunity and irAEs following combination cancer immunotherapies. [Clin Transl Immunol] Full Article

Visit our reviews page to see a complete list of reviews in the cell, gene and immunotherapy research field.

Your Free Copy of the Nature Neuroscience Poster
Cytonet’s Liver Cell Therapy May Be Effective in Stabilizing Children with Severe Urea Cycle Disorders Awaiting Liver Transplant
Cytonet announced that new research on its investigational liver cell therapy (LCT) was presented. The presentation demonstrated how investigational liver cell therapy may help temporarily stabilize pediatric patients with urea cycle disorders as they await liver transplantation. [Press release from Cytonet (Business Wire) discussing research presented at the 2014 World Transplant Congress (WTC), San Francisco] Press Release

Fraunhofer Life Science Symposium
Three Win Inaugural Blavatnik Prizes
Harvard University chemical biologist Adam Cohen uses light to answer some of neuroscience’s most pressing questions. He and his colleagues have found a promising way to optically measure brain activity down to the level of the individual neuron by engineering these nerve cells with proteins that glow whenever the cells fire. [ScienceInsider] Editorial

Spain Needs ‘Major Cultural Change’ to Do Better in Science, International Panel Says
More cash and many profound structural changes – that, according to a panel of European experts, is what Spain’s national science and innovation system needs to become more competitive. [ScienceInsider] Editorial

Transparency Promised for Vilified Impact Factor
The most misused metric in science is getting a makeover – although many researchers would like it to disappear altogether. Information firm Thomson Reuters says that it will become more transparent over how it calculates impact factors, an annual ranking of more than 10,900 scientific journals that it published on 29 July, along with the names of 39 journals that it is barring from the list. [Nature News] Editorial

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Cynata Contracts University of Wisconsin – Madison to Advance Cymerus™ Stem Cell Product Development
Cynata Therapeutics Ltd. announced that it has signed an agreement with the University of Wisconsin – Madison to develop a novel approach for preserving cell therapy products to enhance their shelf life and convenience. [Cynata Therapeutics Ltd.] Press Release

SALSI Postdoc Winners Announced
The San Antonio Life Sciences Institute, a joint venture between The University of Texas at San Antonio (UTSA) and The University of Texas Health Science Center at San Antonio (HSC), has selected the winners of the Postdoc Initiative Pilot Program. The winners, Emily Boice from UTSA and Lei Huang from HSC, will receive $25,000 for their project titled, “Novel engineered ferritins for tracking and protecting neural stem cells in post-ischemic environment.” [San Antonio Life Sciences Institute] Press Release

TargaZyme, Inc. Awarded SBIR Grant from National Eye Institute
TargaZyme, Inc. a clinical- stage biopharmaceutical company developing enzyme technologies and products to improve efficacy outcomes for cancer immunotherapy and cell therapy, announced the receipt of an SBIR award to fund its program for a novel cell-based restorative approach to treat diabetes- related retinopathy. [Targazyme, Inc.] Press Release

Incyte Announces Clinical Trial Agreement to Evaluate Combination of Two Novel Cancer Immunotherapies
Incyte Corporation announced that it has entered into a clinical trial agreement with Genentech to evaluate the safety, tolerability and preliminary efficacy of Incyte’s oral indoleamine dioxygenase-1 inhibitor, INCB24360, in combination with Genentech’s PD-L1 immune checkpoint inhibitor, MPDL3280A, in patients with non-small cell lung cancer. [Incyte Corporation] Press Release

Israel’s Ministry of Health Approves Pluristem’s Commercial-Scale 3D Cell Manufacturing Process Used at Its New Haifa Facility
Pluristem Therapeutics Inc. announced that it has completed the approval process and received final clearance from a fifth regulatory agency for its 3D cell therapy manufacturing processes in use at its new facility in Haifa. [Pluristem Therapeutics Inc.] Press Release

Cellular Biomedicine Group Announces Acquisition of Immune Cell Therapy Technology and U.S. Patent
Cellular Biomedicine Group Inc., a biomedicine firm engaged in the development of new treatments for degenerative and cancerous diseases, announced reaching an agreement to acquire Agreen Biotech Co. Ltd. China and its founder’s U.S. patent. [Cellular Biomedicine Group Inc.] Press Release

Startup ImStem Raising Funds for Novel Stem-Cell MS Treatment
Startup biotech company ImStem Biotechnology is looking to raise additional funding for their upcoming clinical trials after publishing promising research on a unique stem-cell treatment used on mice with multiple sclerosis. [BioNews Services, LLC] Press Release

Lion Biotechnologies Enters Clinical Trial Grant Agreement with Moffitt Cancer Center
Lion Biotechnologies, Inc. announced that it has entered into a clinical trial grant agreement with Moffitt Cancer Center to expand an ongoing Phase I study of TILs combined with ipilimumab in patients with metastatic melanoma. [Lion Biotechnologies, Inc.] Press Release

Final Patient Treated in Neuralstem Phase II ALS Stem Cell Trial
Neuralstem, Inc. announced that the final patient was treated in its Phase II trial using NSI-566 spinal cord-derived neural stem cells in the treatment of amyotrophic lateral sclerosis (ALS). [Neuralstem, Inc.] Press Release
Center for Scientific Review; Notice of Closed Meeting (FR Doc. No: 2014-18068)

Center for Scientific Review; Notice of Closed Meetings (FR Doc. No: 2014-18069)

Center for Scientific Review; Notice of Closed Meeting (FR Doc. No: 2014-18072)
Food and Drug Administration (United States)

Cellular, Tissue and Gene Therapies Advisory Committee; Notice of Meeting (FR Doc. No: 2014-17890)

Draft Guidance for Industry on Cell-Based Products for Animal Use; Availability (FR Doc. No: 2014-18134)
NEW Genetic Engineering of Mammalian Stem Cells
February 16-28, 2015
Cambridge, United Kingdom

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Senior PhD Scientist – Oncology Cell Therapeutics (Novartis Institutes for BioMedical Research)

Postdoctoral Research Fellow – In-Situ Gene Therapy (Fred Hutchinson Cancer Research Center)

Technical Account Manager (GlobalStem)

Research Scientist – iPSC Biology and Genome Editing (Stem Cell Theranostics)

Research Assistant – iPSC Biology and Genome Editing (Stem Cell Theranostics)

Postdoctoral Researcher – Generation of Myocardium In Situ (Van Andel Research Institute)

Postdoctoral Fellow – AAV Vectors for Gene Therapy of Neurological and Other Disorders (University of Massachusetts Medical School)

Postdoctoral Scientist – Immune Focused In Vitro Analytics (Celentyx Ltd.)

Scientist – Pluripotent Stem Cell Media Development, High Throughput Screening (STEMCELL Technologies Inc.)

Scientist – Cell Culture Support Products (STEMCELL Technologies Inc.)

Scientist – Liver Cell Biology (STEMCELL Technologies Inc.)

Scientist – Immunology/Cell Separation (STEMCELL Technologies Inc.)

Research Associate – Cell Separation (STEMCELL Technologies Inc.)

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