Cell Therapy News Volume 13.29 | Jul 30 2012

Restoration of Hearing in the VGLUT3 Knockout Mouse Using Virally Mediated Gene Therapy
Mice lacking the vesicular glutamate transporter-3 (VGLUT3) are congenitally deaf due to loss of glutamate release at the inner hair cell afferent synapse. Cochlear delivery of VGLUT3 using adeno-associated virus type 1 (AAV1) leads to transgene expression in only inner hair cells, despite broader viral uptake. Within two weeks of AAV1-VGLUT3 delivery, auditory brainstem response thresholds normalize, along with partial rescue of the startle response. [Neuron] Abstract | Press Release

Human Cardiac Progenitor Cells Engineered with Pim-I Kinase Enhance Myocardial Repair
The authors demonstrated that efficacy of human cardiac progenitor cells from the failing myocardium can be safely and significantly enhanced through expression of Pim-1 kinase. [J Am Coll Cardiol]
Full Text | Press Release

Redirected T Cells that Target Pancreatic Adenocarcinoma Antigens Eliminate Tumors and Metastases in Mice
Scientists investigated the effects of adoptive transfer of tumor-specific T cells that express antibody-based chimeric receptors to mice with primary and metastatic pancreatic adenocarcinoma xenografts. [Gastroenterology] Abstract

Gene Therapy/Bone Marrow Transplant in ADA-Deficient Mice: Roles of Enzyme Replacement Therapy and Cytoreduction
To determine the contributions of conditioning and discontinuation of enzyme replacement therapy to the therapeutic effects, researchers analyzed these factors in adenosine deaminase-deficient (Ada) gene knock-out mice. [Blood] Abstract

An Oncolytic Adenovirus Enhanced for Toll-Like Receptor 9 Stimulation Increases Antitumor Immune Responses and Tumor Clearance
Researchers generated the first genetically modified oncolytic adenovirus backbone able to enhance Toll-like receptor 9-stimulation for increased antitumor activity. [Mol Ther] Abstract

Transcription Activator-Like Effector Proteins Induce the Expression of the Frataxin Gene
Researchers aimed to develop a potential treatment for Friedreich ataxia by increasing the expression of the frataxin gene. They engineered 12 transcription activator-like effector (TALE) genes (TALE_Frat) encoding TALE_Frat proteins, each specifically targeting different 14-bp DNA sequences within the proximal region of the human frataxin promoter. [Hum Gene Ther] Abstract | Press Release

Tolerance to MHC Class II Disparate Allografts through Genetic Modification of Bone Marrow
Investigators demonstrated for the first time that expression of an allogeneic MHC class II gene in autologous bone marrow cells, resulting in a state of molecular chimerism, induces tolerance to MHC class II mismatched skin grafts, a stringent test of transplant tolerance. [Gene Ther] Abstract

Microwave Sintering and In Vitro Study of Defect-Free Stable Porous Multilayered HAp-ZrO₂ Artificial Bone Scaffold
Results confirmed that hydroxyapatite (HAp)-ZrO₂ performs as an artificial bone, showing excellent cell growth, attachment and proliferation behavior using osteoblast-like MG63 cells. [Sci Technol Adv Mat]
Abstract | Press Release

Adult Stem Cells from Liposuction Used to Create Blood Vessels in the Lab
Adult stem cells derived from fat were turned into smooth muscle cells in the laboratory, and then “seeded” onto a very thin collagen membrane. As the stem cells multiplied, the researchers rolled them into tubes matching the diameter of small blood vessels. In three to four weeks, they grew into usable blood vessels. [Press release from the American Heart Association, Inc. discussing research presented at the American Heart Association’s Basic Cardiovascular Sciences 2012 Scientific Sessions, New Orleans] Press Release

Bone Marrow Transplant Eliminates Signs of HIV Infection
Two men with longstanding HIV infections no longer have detectable HIV in their blood cells following bone marrow transplants. The virus was easily detected in blood lymphocytes of both men prior to their transplants but became undetectable by eight months post-transplant. [Press release from EurekAlert! discussing research presented at the XIX International AIDS Conference, Washington] Press Release

Rheumatoid Arthritis New Major Clinical Target after Mesoblast Obtains Positive Results in Inflammatory Arthritis
Mesoblast Limited announced positive results in a large animal model of rheumatoid arthritis following a single intravenous injection of its proprietary allogeneic immunomodulatory adult mesenchymal precursor cells. [Mesoblast Limited] Press Release

Seventeenth Patient Dosed in Neuralstem ALS Stem Cell Trial
Neuralstem, Inc. announced that the seventeenth patient was treated in the ongoing Phase I trial of its spinal cord neural stem cells for the treatment of amyotrophic lateral sclerosis (ALS). This patient is also the second to return to the trial for additional injections. [Neuralstem, Inc.] Press Release

Cuts Loom for US Science
Hanging over the effective decrease in support proposed by the House of Representatives is the ‘sequester’, a pre-programmed budget cut that research advocates say would starve US science-funding agencies. [House of Representatives, United States] Press Release

U.S. Federal Court Says Stem Cell Treatments Are Drugs
A U.S. federal court has found that a stem cell therapy offered by a Colorado clinic meets the definition of a regulated drug. The ruling could spur a U.S. Food and Drug Administration crackdown on other clinics offering untested adult stem cell treatments in the United States. [U.S. District Court for the District of Columbia, United States] Press Release

Stem Cell Agency Commits $150 Million to Develop New Therapies
California’s stem cell agency, the California Institute for Regenerative Medicine approved $150 million in new funding to help move promising stem cell-based therapies from the laboratory research phase to clinical trials in people. [California Institute For Regenerative Medicine] Press Release

U.S. National Institutes of Health to Support Development of Pluristem’s PLX-RAD Cells for Treatment of Acute Radiation Syndrome
Pluristem Therapeutics, Inc. announced that it has received an invitation from the U.S. National Institute of Allergy and Infectious Diseases to submit its PLacental eXpanded (PLX) cells to the agency for evaluation in models of the acute radiation syndrome. [Pluristem Therapeutics, Inc.] Press Release

Medical College of Wisconsin Researcher to Study New Treatment for Chronic Pain
The Medical College of Wisconsin received a four-year, $1.1 million grant from the National Institutes of Health’s National Institute of Neurological Disorders and Stroke to study the use of genetically modified adult stem cells to treat neuropathic pain. [Medical College of Wisconsin] Press Release

UTMB Scientists Awarded NIH Grant for Lab-Grown Lung Tissue Project
University of Texas Medical Branch at Galveston (UTMB) researchers have been awarded a two-year, $1.25 million National Institutes of Health (NIH) grant to develop a method of custom-growing human lung tissue to make a three-dimensional model for biomedical studies. [EurekAlert!] Press Release

Fraundhofer IZI Receives Official Certification for Manufacturing of Northwest Bio’s DCVax®-L Product
Northwest Biotherapeutics, Inc. announced that its partner in Germany, Fraunhofer IZI, has received the official approval and certification from the regional and national regulatory agencies in Germany for the manufacture of Northwest Bio’s DCVax®-L immune therapy for human patients with Glioblastoma multiforme brain cancer. [Northwest Biotherapeutics, Inc.] Press Release

Histogenics Closes $49 Million Series A Fundraising to Support Commercial Development of Transformational Cartilage Repair Products
Histogenics Corporation announced the completion of a $49 million round of financing. Proceeds will be used to complete the ongoing Phase III clinical program for lead product candidate NeoCart^®. Funds will also support efforts to obtain regulatory clearance in the European Union for product candidate VeriCart™, a cell-free collagen scaffold uniquely designed to be used in conjunction with the patient’s own stem cells, to repair small cartilage defects. [Histogenics Corporation] Press Release

Roche and AREVA Med Join Forces in the Fight Against Cancer and Create a Novel Radio-Immunotherapy
Roche and AREVA Med announced that they have entered into a strategic collaboration to create a novel, advanced alpha radio-immunotherapy platform to target and kill cancer cells. [AREVA] Press Release

ACT Issued Broad Patent for Human RPE Cells Derived from All Types of Pluripotent Stem Cells
Advanced Cell Technology, Inc. (ACT) announced that it has been issued a patent in Australia, patent number 2005325753, “Improved modalities for the treatment of degenerative diseases of the retina.” The patent broadly covers the use of human retinal pigment epithelial (RPE) cells generated from pluripotent stem cells in the manufacture of pharmaceutical preparations of RPE cells, and the use of those preparations to treat patients with degenerative diseases of the retina. [Advanced Cell Technology, Inc.] Press Release

Neuralstem Receives Notice of Issuance for Patent Covering Human Neural Cell Transplantation for Neurodegenerative Conditions
Neuralstem, Inc. announced that it received a notice of issuance for patent number 12/710,097 titled: “Transplantation of Human Neural Cells for Treatment of Neurodegenerative Conditions.” This patent covers both the culturing of central nervous system cells as well as their transplantation into spinal cord tissue to treat neurodegenerative conditions. [Neuralstem, Inc.] Press Release

Notice of Intent to Publish a Funding Opportunity Announcement for Opportunities for Collaborative Research at the NIH Clinical Center (U01) (NOT-HD-12-025)

NCI National Clinical Trials Network – Network Group Operations Centers (U10) (RFA-CA-12-010)

NCI National Clinical Trials Network – Network Group Integrated Translational Science Centers (U10) (RFA-CA-12-012)

NCI National Clinical Trials Network – Canadian Collaborating Clinical Trials Network (Limited Competition U10) (RFA-CA-12-504)

Instrument Development for Biomedical Applications (R21) (RFA-GM-13-010)

National Institute of Diabetes and Digestive and Kidney Diseases; Notice of Closed Meeting (FR Doc No: 2012-18067)

European Medicines Agency (European Union)

European Medicines Agency Sees Benefits of Interaction with Japanese Regulators
The European Medicines Agency has increased its level of interaction and cooperation with medicines regulatory authorities in Japan over the past three years, according to the report on interactions. Press Release

Food and Drug Administration (United States)

Agency Information Collection Activities; Submission for Office of Management and Budget Review; Comment Request; Financial Disclosure by Clinical Investigators (FR Doc No: 2012-18235)

NEW The Jackson Laboratory: Educational Webinar on the Mouse Phenome Database
August 9, 1:00PM (ET in US)
Webinar

NEW 8^th World Stem Cell Summit
December 3-5, 2012
West Palm Beach, United States

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.

Scientist – Induced Pluripotent Stem Cells (STEMCELL Technologies, Inc.)

Scientist – Epithelial Cell Research (STEMCELL Technologies, Inc.)

Scientist – hPSC Bioengineering (STEMCELL Technologies, Inc.)

Scientist – Mesenchymal Cell Research (STEMCELL Technologies, Inc.)

Research Associate – Chemistry (STEMCELL Technologies, Inc.)

Research Associate – Particle Chemistry (STEMCELL Technologies, Inc.)

Scientific Communications & Publishing Coordinator (STEMCELL Technologies, Inc.)

Quality Control Operations Coordinator (STEMCELL Technologies, Inc.)

Research Technologist – Research and Development (STEMCELL Technologies, Inc.)

Research Technologist – Contract Assay Services (STEMCELL Technologies, Inc.)

Research Technologist – Media Development (STEMCELL Technologies, Inc.)

Clinical Coordinator (New York Obesity Nutrition Research Center)

Postdoctoral Research Position (University Lille 2 France)

Postdoctoral Position – Stem Cells (Children’s Hospital of Chicago Research Center)

Postdoctoral Position – Induced Pluripotent Stem Cell Research (University of Washington)


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