Cell Therapy News Volume 12.44 | Nov 7 2011

Dopamine Neurons Derived from Human Embryonic Stem Cells Efficiently Engraft in Animal Models of Parkinson’s Disease
Here scientists present a novel floor-plate-based strategy for the derivation of human dopamine neurons that efficiently engraft in vivo, suggesting that past failures were due to incomplete specification rather than a specific vulnerability of the cells. [Nature] Abstract | Press Release

Long-Term Outcomes Among Older Patients Following Nonmyeloablative Conditioning and Allogeneic Hematopoietic Cell Transplantation for Advanced Hematologic Malignancies
The objective of this study is to describe outcomes of patients 60 years or older after receiving minimally toxic nonmyeloablative allogeneic hematopoietic cell transplantation. [JAMA] Abstract | Press Release

Cancer Cell-Selective In Vivo Near Infrared Photoimmunotherapy Targeting Specific Membrane Molecules
In this study, researchers have developed a new type of molecular-targeted cancer therapy, photoimmunotherapy, that uses a target-specific photosensitizer based on a near-infrared phthalocyanine dye, IR700, conjugated to monoclonal antibodies targeting epidermal growth factor receptors. [Nat Med] Abstract | Press Release

Rejuvenating Senescent and Centenarian Human Cells by Reprogramming through the Pluripotent State
Investigators demonstrate, using an optimized protocol, that cellular senescence is not a limit to reprogramming and that age-related cellular physiology is reversible. [Genes Dev] Abstract | Press Release

Engineered Cell Homing
Using a platform approach that preserves the mesenchymal stem cell (MSC) phenotype and does not require genetic manipulation, researchers modified the surface of MSCs with a nanometer-scale polymer construct containing sialyl Lewis^x that is found on the surface of leukocytes and mediates cell rolling within inflamed tissue. [Blood] Abstract | Press Release

Long-Term RNA Interference Gene Therapy in a Dominant Retinitis Pigmentosa Mouse Model
Investigators used self-complementary AAV2/8 vector to develop an RNA interference-based gene therapy in a dominant retinal degeneration mouse model expressing bovine GCAP1(Y99C). [Proc Natl Acad Sci USA] Abstract

Adventitial Delivery of an Allogeneic Bone Marrow–Derived Adherent Stem Cell in Acute Myocardial Infarction
In this phase I clinical trial in patients with first ST-elevation–myocardial infarction, investigators combine first-in-man delivery of MultiStem with a first-in-coronary adventitial delivery system to determine the effects of this system on left ventricular function at 4 months after acute myocardial infarction. [Circ Res] Abstract

Precipitation of Nanohydroxyapatite on PLLA/PBLG/Collagen Nanofibrous Structures for the Differentiation of Adipose Derived Stem Cells to Osteogenic Lineage
Results proved that the Poly(l-lactic acid)/Poly-benzyl-l-glutamate/Collagen/nanohydroxyapatite (PLLA/PBLG/Col/n-HA) scaffolds promoted greater osteogenic differentiation of adipose derived stem cells as evident from the enzyme activity and mineralization profiles for bone tissue engineering. [Biomaterials] Abstract

Dedifferentiation-Reprogrammed Mesenchymal Stem Cells with Improved Therapeutic Potential
Here scientists show that, after in vitro induction of neuronal differentiation and dedifferentiation, upon withdrawal of extrinsic factors, mesenchymal stem cells derived from bone marrow, which have already committed to neuronal lineage, revert to a primitive cell population retaining stem cell characteristics but exhibiting a reprogrammed phenotype distinct from their original counterparts. [Stem Cells] Abstract | Press Release

Functionally Enhanced siRNA Targeting TNFα Attenuates Dextran Sulphate Sodium-Induced Colitis and Toll-Like Receptor-Mediated Immunostimulation in Mice
In this study, native and chemically modified small interfering RNA (siRNA) against tumor necrosis factor α (TNFα) was developed and characterized using a murine model of inflammatory bowel disease. [Mol Ther] Abstract

Restoration of Normal BMP Signaling Levels and Osteogenic Differentiation in FOP Mesenchymal Progenitor Cells by Mutant Allele-Specific Targeting
Investigators generated allele-specific siRNA (ASP-RNAi) duplexes capable of specifically suppressing the expression of the mutant c.617A allele in mesenchymal progenitor cells from fibrodysplasia ossificans progressiva (FOP) patients and showed that this ASP-RNAi approach decreased the elevated bone morphogenetic protein (BMP) signaling that is characteristic of patient cells to levels similar to control cells and restored enhanced osteogenic differentiation to control levels. [Gene Ther] Abstract | Press Release

Regeneration of Rodent Small Intestine Tissue Following Implantation of Scaffolds Seeded with a Novel Source of Smooth Muscle Cells
The aim of this study is to apply an organ regeneration platform technology of autologous smooth muscle cell/biomaterial combination products, previously demonstrated to be successful for urinary tissue regeneration, to the regeneration of the small intestine. [Regen Med] Abstract | Press Release

Phenotypic Correction of Hemophilia A in Sheep by Postnatal Intraperitoneal Transplantation of FVIII-Expressing MSC
Here, investigators tested a novel, nonablative transplantation therapy in two pediatric hemophilia A animals. Paternal mesenchymal stem cells (MSC) were transduced with a porcine FVIII-encoding lentivector and transplanted via the intraperitoneal route without preconditioning. [Exp Hematol] Abstract | Press Release

Novel Three-Dimensional Long-Term Bone Marrow Culture System Using Polymer Particles with Grafted Epoxy-Polymer-Chains Supports the Proliferation and Differentiation of Hematopoietic Stem Cells
Scientists examined the hematopoietic supportive ability of stromal cells in a three-dimensional culture system using polymer particles with grafted epoxy polymer chains. [Exp Biol Med] Abstract | Press Release

Marina Biotech Demonstrates That Its Novel UsiRNA Oligonucleotide Abrogates Cytotoxicity Resulting from MicroRNA-Like Off-Target Activity
Marina Biotech, Inc. reported significant reduction of microRNA-like cytotoxic effect by incorporating a single unlocked nucleobase analog moiety in the guide strand of an siRNA (UsiRNA) when compared to an unmodified siRNA of the same sequence. [Marina Biotech, Inc.] Press Release

Researchers Design a Viral Vector to Treat a Genetic Form of Blindness
Researchers have developed a viral vector designed to deliver a gene into the eyes of people born with an inherited, progressive form of blindness that affects mainly males. [The Ohio State University Medical Center] Press Release

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Spanish Institute Faces Cash Crisis
The Prince Felipe Research Centre announced an emergency financial plan on 19 October that would see about 100 research staff lose their jobs, and roughly halve salaries for those who remain. [Prince Felipe Research Centre, Spain] Press Release

U.S. Official Defends Science-Technology Exchanges with China
President Barack Obama’s top science official said U.S.-China science cooperation benefits the United States, rejecting moves to curb the exchanges by lawmakers who say it strengthens China’s military. [Office of Science and Technology, United States] Press Release

Texas Delays Action on Stem Cell Therapy Rules
The Texas State Medical Board voted to delay until next year final approval of new stem cell therapy rules that could restrict – or even block – procedures such as the one Gov. Rick Perry recently underwent on his aching back. [Texas State Medical Board, United States] Press Release

Oxford BioMedica Announces Research and Development Collaboration for Glaucoma
Oxford BioMedica plc announces that it has entered into a research and development collaboration with Mayo Clinic, Rochester to develop a novel gene therapy for the treatment of chronic glaucoma. [Oxford BioMedica plc] Press Release

Technology to Collect Stem Cells from Fat Tissue May Help Soldiers Recover from Traumatic Injuries
IKOTECH LLC and Techshot Inc. are working to develop equipment called Quadrasep™ that improves the collection of stem cells from adult adipose tissue. The U.S. Army Medical Research and Materiel Command selected the companies for a $730,000 contract to develop the technology. [IKOTECH LLC] Press Release

Stratatech Awarded $4.6-Million Clinical Trial Grant for ExpressGraft^® Antimicrobial Skin Substitute
Stratatech Corp. announced that the company has been awarded a $4.6-million grant by the National Institute of Diabetes and Digestive and Kidney Diseases to support the first human clinical trial of Stratatech’s ExpressGraft^® antimicrobial skin substitute. [Stratatech Corporation] Press Release

Foundation Fighting Blindness Invests $8.25 Million in Six Gene Therapy Research Projects Poised for Clinical Trials
The Foundation Fighting Blindness announces an $8.25 million investment in six new gene therapy research projects that are targeted to have treatments ready for clinical trials within three years. [The Foundation Fighting Blindness] Press Release

Osteoarthritis Gene Therapy Being Developed at UF Could Help Both People, Animals
University of Florida (UF) researchers are developing a gene therapy technique that could help both humans and horses fight osteoarthritis. The research team received a highly competitive one-year, $900,000 grant from the National Institute of Health’s National Institute of Arthritis, Musculoskeletal and Skin Disease to fund the work. [University of Florida] Press Release

American Heart Association Donates $660,000 to Support University of Louisville Heart Research
The American Heart Association has awarded four research grants totaling $660,000 to the University of Louisville to aid in the development of new cardiovascular treatments. [University of Louisville] Press Release

Patent Issued for OHSU Stem Cell Cloning
Oregon Health & Science University (OHSU)’s unique method of transforming a person’s own skin cells into stem cells has officially been patented. [Oregon Health & Science University] Press Release

Dicerna Pharmaceuticals Awarded Notice of Allowance by U.S. Patent and Trademark Office for Patent Claims Broadly Covering Dicer Substrate siRNA Molecules
Dicerna Pharmaceuticals, Inc. announced that the U.S. Patent and Trademark Office has issued a Notice of Allowance in the “Rossi” patent family, which broadly covers Dicer Substrate RNA-containing therapeutics. [Dicerna Pharmaceuticals, Inc.] Press Release

UCSF Training Program Attracts Best Minds in Developmental, Stem Cell Biology
Fittingly enough for a University where embryonic stem cells were first discovered in mice and named, the University of California, San Francisco (UCSF) has launched a pioneering program in Developmental and Stem Cell Biology. [University of California, San Francisco] Press Release

National Institute of Diabetes and Digestive and Kidney Diseases; Notice of Closed Meeting (FR Doc No: 2011-28378)

Public Workshop: Quarantine Release Errors in Blood Establishments Presentations

September 22, 2011: Cellular, Tissue and Gene Therapies Advisory Committee Meeting: Transcript

September 22-23, 2011: Cellular, Tissue and Gene Therapies Advisory Committee Meeting: Final Agenda

September 23, 2011: Cellular, Tissue and Gene Therapies Advisory Committee Meeting: Transcript

Food and Drug Administration (United States)

Draft Guidance for Industry and Food and Drug Administration Staff; De Novo Classification Process (Evaluation of Automatic Class III Designation); Availability; Extension of Comment Period [Docket No. FDA-2011-D-0689]

Guidance for Industry: Clinical Considerations for Therapeutic Cancer Vaccines; Availability [Docket No. FDA-2009-D-0427]

European Medicines Agency (European Union)

European Medicines Agency Meetings to Be Held Offsite During 2012 Olympics
The meetings of the European Medicines Agency’s committees and working parties will be held at the offices of other European regulatory authorities, held virtually or cancelled during late June and July 2012, prior to the Olympic Games in London. Press Release

Medicines and Healthcare Regulatory Agency (United Kingdom)

Good Clinical Practice Forum Launched
The Medicines and Healthcare Regulatory Agency announced that a new Agency discussion forum for Good Clinical Practice has been launched. Press Release

NEW Mouse Models of Human Diabetes and Obesity for Therapeutic Testing
November 10, 2011
Webinar

NEW Modeling Type 2 Diabetes Complications in the Mouse
November 14, 2011
Webinar

NEW Select Biosciences STEMCELLS 2012
February 2-3, 2012
San Diego, United States

NEW 7th Annual Stem Cell Summit
February 21, 2012
New York, United States

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

Lab Technologist – Tissue Culture (STEMCELL Technologies)

Research and Development Technologist, hPSC Media (STEMCELL Technologies)

Scientist (iPSC) (STEMCELL Technologies)

Lab Technologist – Pluripotent Stem Cells (STEMCELL Technologies)

Scientist – Pluripotent Stem Cells (STEMCELL Technologies)

Postdoctoral Positions, Extracellular Matrix Research (University of Oulu)

PhD Student Positions, Extracellular Matrix Research (University of Oulu)

Postgraduate Students (Masaryk University)

Senior Development Associate (Genzyme Corporation)

Research Associate – Cell Culture (Stemgent, Inc.)

Scientist – Differentiation Segment (Stemgent, Inc.)

Clean Room Associate – Cell Therapy Manufacturing (Cell Medica Limited)

Junior/Senior Research Fellowship in Human Pluripotent Stem Cell Research (Masaryk University)

Scientist II, Innovation – Cell Processing Technologies (Lonza Walkersville, Inc.)

Senior Manager, Innovation – Cell Processing Technologies (Lonza Walkersville, Inc.)

Lab Technologist Position (Michigan State University)

Postdoctoral Fellow – Islet Cell Electrophysiology (Vanderbilt University)


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