Volume 15.27 | Jul 14

Issue 15.27 July 14, 2014
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Kit Regulates HSC Engraftment across the Human-Mouse Species Barrier
To generate an improved model for human HSC transplantation and analysis, scientists developed immune-deficient mouse strains containing Kit mutations. They found that mutation of the Kit receptor enables robust, uniform, sustained, and serially transplantable engraftment of human HSCs in adult mice without a requirement for irradiation conditioning. [Cell Stem Cell] Abstract | Press Release | Graphical Abstract
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PUBLICATIONS (Ranked by impact factor of the journal)
Transplant Restoration of Spinal Cord Inhibitory Controls Ameliorates Neuropathic Itch
Researchers tested the hypothesis that the neuropathic itch in BHLHB5-deficient animals can be treated by restoring inhibitory controls through spinal cord transplantation and integration of precursors of cortical inhibitory interneurons derived from the embryonic medial ganglionic eminence. [J Clin Invest] Full Article

ESCRT Mediated Uptake and Degradation of Brain Targeted α-Synuclein-Single Chain Antibody Attenuates Neuronal Degeneration In Vivo
Scientists investigated a single chain antibodies (scFV) directed against oligomeric α-syn fused to the LDL receptor-binding domain from apoB. The modified scFV showed enhanced brain penetration and was imported into neuronal cells through the ESCRT pathway leading to lysosomal degradation of α-syn aggregates. [Mol Ther] Abstract | Full Article

Diabetic Mesenchymal Stem Cells Are Ineffective for Improving Limb Ischemia due to Their Impaired Angiogenic Capability
Researchers investigated the effects of diabetes on mesenchymal stem cells in terms of their angiogenic and therapeutic potential for repairing tissue ischemia. [Cell Transplant] Abstract

Antitumor Effects of Baculovirus-Infected Dendritic Cells against Human Pancreatic Carcinoma
Researchers examined the antitumor effect of baculovirus (BV)-infected dendritic cells-(DCs) on human pancreatic cancer cells (AsPC-1). After treatment with BV-infected bone marrow-derived dendritic cells (BMDCs), human pancreatic tumors caused by AsPC-1 cells in a nude mouse model were significantly reduced in size, and the survival of the mice was improved compared with that of non-immature BMDC- and BV-DC-immunized mice. [Gene Ther] Abstract

Outcomes after Hematopoietic Stem Cell Transplant for Children with I-Cell Disease
Although hematopoietic stem cell transplant (HSCT) has been used to successfully treat some lysosomal storage diseases, there have been only two case reports in the use of HSCT to treat mucolipidosis type II (MLII) or I-Cell Disease. Scientists describe the combined international experience in the use of HSCT for MLII in 22 patients. [Biol Blood Marrow Transplant] Abstract

Outcome of Patients with HTLV-1-Associated Adult T-Cell Leukemia/Lymphoma after SCT: A Retrospective Study by the EBMT LWP
Experience with allo-SCT for adult T-cell leukemia/lymphoma (ATL) appears encouraging but is limited to Japanese series. Researchers indicate that allo-SCT but not auto-SCT may salvage a subset of ATL patients, supporting the existence of graft vs ATL effect also in non-Japanese patients. [Bone Marrow Transplant] Abstract

Autograft-Derived Spinal Cord Mass following Olfactory Mucosal Cell Transplantation in a Spinal Cord Injury Patient
The authors present the case of an 18-year-old woman who sustained a complete spinal cord injury at T10-11. Three years after injury, she remained paraplegic and underwent olfactory mucosal cell implantation at the site of injury. She developed back pain 8 years later, and imaging revealed an intramedullary spinal cord mass at the site of cell implantation, which required resection. [J Neurosurg] Abstract | Press Release

Alteration of Naïve and Memory B-Cell Subset in Chronic Graft-versus-Host Disease Patients after Treatment with Mesenchymal Stromal Cells
Scientists investigated changes in the numbers, phenotypes, and subpopulations of B lymphocytes in chronic graft-versus-host disease patients who showed a complete response, partial response, or no response after mesenchymal stromal cell treatment. [Stem Cells Transl Med] Abstract

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The CD133+ Cell as an Advanced Medicinal Product for Myocardial and Limb Ischemia
The authors summarize available experimental and clinical data on CD133+ cells in the context of myocardial and peripheral ischemia, as well as focus on the development of the CD133+ cell as an anti-ischemic advanced cell product. [Stem Cells Dev] Abstract

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

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CEL-SCI Presents Rheumatoid Arthritis Data
CEL-SCI Corporation announced that Daniel Zimmerman, Ph.D., its Senior Vice President of Research, Cellular Immunology, delivered a scientific presentation. The presentation entitled “The next generation of Rheumatoid Arthritis therapies: What has been learned from therapeutic vaccines for models of Rheumatoid Arthritis. Is IL-17 the real key to new therapies?” discussed the potential role CEL-SCI’s LEAPS vaccines might have on the future treatment of rheumatoid arthritis. [Press release from CEL-SCI Corporation discussing research presented at the 12th Vaccines Research & Development: All Things Considered Conference, Boston] Press Release

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Indian Biologists Welcome Budget Hike
Clean water, biotechnology, and higher education are among the winners in the Indian budget presented by the new government in the lower house of Parliament. [ScienceInsider] Editorial

Corruption Probes Ensnare Chinese Science Powerhouses
The Central Commission for Discipline Inspection announced that it had uncovered fraud in research grants managed by China’s Ministry of Science and Technology and at prestigious Fudan University in Shanghai. [ScienceInsider] Editorial

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New Stem Cell Therapy for Ischemic Heart Disease
An EU-funded project is looking into ‘cardiac stem cells’ to tackle the problem of chronic heart failure in Europe. Whilst previous research has concentrated on cells from the patients themselves, the Cardio repair European multidisciplinary initiative team focuses on cells from various donors. [CORDIS] Press Release

Bristol-Myers Squibb Announces Plans for Third Quarter Submission of a Biologics License Application for Opdivo® (Nivolumab), an Investigational PD-1 Immune Checkpoint Inhibitor, for Previously Treated Advanced Melanoma
Bristol-Myers Squibb Company announced that, following discussions with the U.S. Food and Drug Administration, the company is planning a third quarter submission of a biologics licensing application for Opdivo® for previously treated advanced melanoma. [Bristol-Myers Squibb Company] Press Release

CEL-SCI Receives Regulatory Clearance to Expand CEL-SCI’S Phase III Head and Neck Cancer Trial into Austria
CEL-SCI Corporation announced that it has received regulatory clearance from the Austrian Federal Office for Safety in Health Care to begin patient enrollment in the company’s Phase III head and neck cancer clinical trial of its investigational cancer immunotherapy treatment Multikine. [CEL-SCI Corporation] Press Release

Biotech Company Receives Application Approval from Chinese Food and Drug Administration
Beike Biotechnology Co., Ltd. has officially been accepted into the approval process for the Chinese Food and Drug Administration for the use of human umbilical cord derived mesenchymal stem cells for clinical translation and treatment of systemic lupus erythamatosus. [Beike Biotechnology Co., Ltd. (PR Newswire Association LLC)] Press Release

ViaCyte Achieves Significant Milestone with ISO 13485:2003 Certification for Its Encaptra Drug Delivery System
ViaCyte, Inc., a regenerative medicine company developing a cell therapy for treatment of type I diabetes, announced it has received International Standards Organization (ISO) 13485:2003 certification. [ViaCyte, Inc.] Press Release

Sirnaomics Strengthens Its IP Position for Advancing the Company’s Novel siRNA Therapeutics Programs
Sirnaomics, Inc. announced that the company has received an issue notification from United States Patent and Trademark Office for a patent entitled “Multi-Targeted RNAi Therapeutics for Scarless Wound Healing of Skin”. [Sirnaomics, Inc.] Press Release

BioTime Announces Issuance of 14 Patents in the Fields of Regenerative Medicine, and Cancer Diagnosis and Therapy
BioTime, Inc. announced the issuance of 14 new patents to BioTime and its subsidiary companies. The patents issued in Australia, Canada, China, Japan, and the United States, cover a wide range of core technologies foundational to BioTime’s businesses. [BioTime, Inc.] Press Release

Alberto Chiappori, Julien Sage, and E. Alejandro Sweet-Cordero Receive LUNGevity Foundation 2014 Targeted Therapeutics Awards for Lung Cancer Research
LUNGevity Foundation announced the 2014 awardees of its Targeted Therapeutics for Translational Lung Cancer Research program. Each of the three multi-year awards ranges from $100,000 to $200,000 for the first year and is renewable in that amount, based on research progress, for each subsequent year. [LUNGevity Foundation]
Press Release

Sarepta Therapeutics Reports Long-Term Outcomes through 144 Weeks from Phase IIb Study of Eteplirsen in Duchenne Muscular Dystrophy
Sarepta Therapeutics, Inc., a developer of RNA-based therapeutics, announced data through Week 144 from Study 202, a Phase IIb open-label extension study of eteplirsen in patients with Duchenne muscular dystrophy. [Sarepta Therapeutics, Inc.] Press Release
Prospective Grant of Evaluation Option Exclusive License: Development of Granulysin Immunotherapy (FR Doc. No: 2014-16267)

Prospective Grant of Start-Up Exclusive Evaluation Option License Agreement: AAV Mediated Aquaporin-1 Gene Transfer to Treat Sjögren’s Syndrome (FR Doc. No: 2014-16030)
Food and Drug Administration (United States)

Draft Guidance for Industry: Design and Analysis of Shedding Studies for Virus or Bacteria-Based Gene Therapy and Oncolytic Products; Availability (FR Doc. No: 2014-15991)

Request for Notification From Industry Organizations Interested in Participating in the Selection Process for a Nonvoting Industry Representative and Request for Nominations for Nonvoting Industry Representatives on the Cellular, Tissue, and Gene Therapies Advisory Committee (FR Doc. No: 2014-16038)
NEW Stem Cell Meeting on the Mesa
October 7-9, 2014
La Jolla, United States

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Postdoctoral Researcher – Generation of Myocardium In Situ (Van Andel Research Institute)

Postdoctoral Fellow – Genome Editing in Human Embryonic Stem Cells to Study Hereditary Cancer-Associated Mutations (UConn Health Center)

Production Manager – Cell Banking Program (Medicines and Healthcare Products Regulatory Agency)

Senior Scientist – Stem Cell (Medicines and Healthcare Products Regulatory Agency)

Postdoctoral or Senior Scientist Positions – Molecular Genetics and Gene Therapy (Carl von Ossietzky University Oldenburg)

PhD Studentship – Effects of Aging on MSC Therapy Activity (Loughborough University)

Cell Therapy Lab Assistant Supervisor (Fred Hutchinson Cancer Research Center)

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.)

Assistant or Associate Professor – Stem Cell Transplantation Program (Dana-Farber Cancer Institute)

Postdoctoral Fellow – Prostate Cancer Research (Henry Ford Hospital)

Postdoctoral Position – Neurotoxicity and Epigenetics (Thomas Jefferson University)

Research/Review Scientist – Cell Therapy and Regenerative Medicine (Center for Biologics Evaluation and Research)

Postdoctoral Position – Cell Death and Inflammation (Inflammation Research Center – VIB-UGent)

Postdoctoral Position – Role of Calreticulin in TGF-Beta Signaling in the Kidney Proximal Tubule under Diabetic Conditions (University of Alabama)

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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