Volume 12.29 | Jul 25

Cell Therapy News 12.29, July 25, 2011
     In this issue: Science | Policy | Business | NIH | CBER | Regulatory | Events
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Clinical Trial of Molecular Therapy for Muscular Dystrophy Yields Significant Positive Results
A molecular technique has taken one step closer to becoming a treatment for the devastating genetic disease Duchenne muscular dystrophy. [Press release from the University of North Carolina at Chapel Hill discussing online prepublication in The Lancet]


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Cancer Cells and Stem Cells Share Same Origin, Researchers Say
Scientists have proven that oncogenes also can change normal cells into stem-like cells, paving the way to a safer and more practical approach to treating diseases such as multiple sclerosis and cancer with stem cell therapy. [Press release from the University of Southern California discussing online prepublication in the Proceedings of the National Academy of Sciences, United States]

Alnylam and MIT Collaborators Discover Novel “Core-Shell” Nanoparticles for Systemic Delivery of RNAi Therapeutics
Alnylam Pharmaceuticals, Inc. together with collaborators at the Massachusetts Institute of Technology (MIT), announced the discovery of “core-shell” nanoparticles for systemic delivery of RNAi therapeutics. [Press release from Business Wire discussing online prepublication in the Proceedings of the National Academy of Sciences, United States]

Researchers Improve Method to Create Induced Pluripotent Stem Cells
Researchers have developed a new strategy to improve the development of induced pluripotent stem cells. [Press release from the University of Minnesota discussing online prepublication in Stem Cells]

Gene Therapy Delivered Once to Blood Vessel Wall Protects Against Atherosclerosis in Rabbit Studies
A one-dose method for delivering gene therapy into an arterial wall effectively protects the artery from developing atherosclerosis despite ongoing high blood cholesterol. [Press release from the University of Washington discussing online prepublication in Molecular Therapy]

Alnylam and MIT Collaborators Publish Data on Novel Lipid Nanoparticles for Systemic Delivery of RNAi Therapeutics
Alnylam Pharmaceuticals, Inc. together with collaborators at the Massachusetts Institute of Technology (MIT), announced new data describing a novel approach for systemic delivery of RNAi therapeutics using synergistic combinations of novel lipid-like materials called “lipidoids.” [Press release from Alnylam Pharmaceuticals, Inc. discussing online prepublication in Molecular Therapy]

Protein Switch Controls How Stem Cells Turn into New Heart Tissue
Researchers have identified a protein that can direct stem cells to become either new heart muscle or blood vessels. [Press release from the University of Oxford discussing online prepublication in Development]

Survival of Stage IV Breast Cancer Patients Improves with Stem Cell Treatment, Study Finds
Researchers found that a greater proportion of patients who received the aggressive high-dose chemotherapy treatment 12 to 14 years ago, followed by a rescue with their own, specially purified blood stem cells that had been purged of cancer, survived compared with those who were rescued with unmanipulated blood grafts. [Press release from Stanford School of Medicine discussing online prepublication in the Biology of Blood and Marrow Transplantation]

New Gene Therapy to Reverse Heart Failure Ready for Clinical Trials
A promising gene therapy developed to prevent and reverse congestive heart failure is on the verge of clinical trials, after years of proving itself highly effective in the lab and a large animal study. [Press release from Thomas Jefferson University Hospitals discussing online prepublication in Science Translational Medicine]

Genetically Engineered Cardiac Stem Cells Repaired Damaged Mouse Heart
Genetically engineered human cardiac stem cells helped repair damaged heart tissue and improved function after a heart attack, in a new animal study. [Press release from the American Heart Association discussing research presented at the Basic Cardiovascular Sciences Annual Conference 2011 in New Orleans]

Researchers Create Reprogrammed Stem Cells for Disease Studies
The University of Michigan’s Consortium for Stem Cell Therapies has achieved another of its primary goals: reprogramming adult skin cells so they behave like embryonic stem cells. [University of Michigan Press Release]

ON168-HIV NatureWebcast_645x110

(Ranked by Impact Factor of the Journal)
Exon Skipping and Dystrophin Restoration in Patients with Duchenne Muscular Dystrophy after Systemic Phosphorodiamidate Morpholino Oligomer Treatment: An Open-Label, Phase II, Dose-Escalation Study
Scientists report clinical safety and biochemical efficacy from a dose-ranging study of intravenously administered AVI-4658 phosphorodiamidate morpholino oligomer in patients with Duchenne muscular dystrophy. [Lancet]

Generation of Healthy Mice from Gene-Corrected Disease-Specific Induced Pluripotent Stem Cells
Using the murine model of tyrosinemia type 1 (fumarylacetoacetate hydrolase [FAH] deficiency; FAH−/− mice) as a paradigm for orphan disorders researchers evaluated fibroblast-derived FAH−/−-induced pluripotent stem cells as targets for gene correction in combination with the tetraploid embryo complementation method. [PLoS Biol]

Combinatorial Synthesis of Chemically Diverse Core-Shell Nanoparticles for Intracellular Delivery
Using robotic automation, epoxide-functionalized block polymers were combinatorially cross-linked with a diverse library of amines, followed by measurement of molecular weight, diameter, RNA complexation, cellular internalization, and in vitro siRNA and pDNA delivery. [Proc Natl Acad Sci U S A]

Increase Developmental Plasticity of Human Keratinocytes with Gene Suppression
In this study, primary human keratinocytes were successfully induced into a stage of plasticity by transient inactivation of p53, without enforced expression of any of the transcription factors previously used in induced pluripotent stem cell generation. [Proc Natl Acad Sci U S A]

Expression of Apolipoprotein A-I in Rabbit Carotid Endothelium Protects Against Atherosclerosis
Researchers used a helper-dependent adenoviral vector to test whether expression of apolipoprotein A-I in the artery wall could retard the development of atherosclerosis in hyperlipidemic rabbits. [Mol Ther]



Advocates Attempt to Advance Regenerative Medicine with Introduction of HR 1862
The nonprofit Alliance for Regenerative Medicine hopes to accomplish more stem cell-based therapy approvals  in part through the Regenerative Medicine Promotion Act of 2011 (HR 1862), which was introduced in May. [Alliance for Regenerative Medicine, United States]

HHS Unveils Plan to Tighten Human Subjects Rules
After 20 years, it’s time for an overhaul. That was the message from the U.S. Department of Health and Human Services (HHS), which wants to beef up protections for people participating in research. [U.S. Department of Health and Human Services, United States]

Regulations Proposed for Animal–Human Chimeras
Animal-human chimeras might be useful to researchers studying human cognition or human-specific pathogens. But it would be ethically unacceptable and should be banned, argues a government-commissioned report from the UK Academy of Medical Sciences. [UK Academy of Medical Sciences, United Kingdom]


BrainStorm Signs MOU for Collaboration with Massachusetts General Hospital and University of Massachusetts Medical School to Conduct Human Clinical Trials in the United States
BrainStorm Cell Therapeutics Inc. announced that it has entered into a Memorandum of Understating (MOU) with Massachusetts General Hospital and the University of Massachusetts Medical School in anticipation of applying for FDA approval to begin ALS human clinical trials in the United States. [Brainstorm Cell Therapeutics, Inc. Press Release]

University of Pittsburgh Receives $67.3 Million to Translate Science into Therapies
A University of Pittsburgh institute aimed at accelerating the pace of translating science into real-life treatments for patients has received $67.3 million from the National Institutes of Health to expand its work over the next five years. [University of Pittsburgh Medical Center Press Release]

Columbia University Medical Center Receives $38.9 Million to Help Translate Science into Treatment
The Irving Institute for Clinical and Translational Research is among 10 institutes nationwide to receive renewed funding, in recognition of their successes during the first five years of the Clinical and Translational Science Awards program. [Columbia University Medical Center Press Release]

Ten UConn Researchers Receive State Stem Cell Funding
Ten scientists at the University of Connecticut (UConn) and its Health Center received grant awards totaling $5.7 million from the Connecticut Stem Cell Research Advisory Committee, to support their pioneering embryonic stem cell research. [University of Connecticut Press Release]

Ottawa Hospital Researchers Awarded $4.8 Million from Ontario Research Fund to Advance Regenerative Medicine and Patient Safety
Two research teams affiliated with the Ottawa Hospital Research Institute, The Ottawa Hospital and the University of Ottawa have been awarded major new grants through the Ontario Research Fund – Research Excellence competition. [Ottawa Hospital Research Institute Press Release]

BioVeteria Licenses Regenerative Technology for Cartilage and Bone
BioVeteria Life Sciences, LLC announced they have exclusively licensed a patented, orally active cartilage and bone repair technology from the Canadian Arthritis Network. [BioVeteria Life Sciences, LLC Press Release]

Neuralstem Receives Russian Patent for Stem Cell Transplantation to Treat Neurodegenerative Conditions
Neuralstem, Inc. announced that it has received a patent covering the transplantation of human neural cells for the treatment of neurodegenerative conditions from the Russian Federation. [Neuralstem, Inc. Press Release]

Genethon and Children’s Hospital Boston Get FDA Approval for a Wiskott Aldrich Gene Therapy Trial
The US Food & Drug Administration (FDA) approved the launching in the U.S. of a clinical trial for gene therapy for a rare immunodeficiency, Wiskott-Aldrich syndrome. [Children’s Hospital Boston Press Release]

Will NIH Embrace Biomedical Research Prizes?
Several parts of the NIH agency held a meeting in which various government and private organizations that offer research prizes described their successes, prompting speculation over whether NIH will follow suit.

Center for Scientific Review; Notice of Closed Meeting (FR Doc No: 2011-18148)

National Cancer Institute; Notice of Closed Meeting (FR Doc No: 2011-18141)

National Cancer Institute; Notice of Closed Meeting (FR Doc No: 2011-18145)

National Institute of General Medical Sciences Notice of Closed Meeting (FR Doc No: 2011-18291)

Office of Biotechnology Activities; Recombinant DNA Research: Action Under the NIH Guidelines for Research Involving Recombinant DNA Molecules (NIH Guidelines) (FR Doc No: 2011-18726)

Research Project Grant (Parent R01) (PA-11-260)

Public Workshop – Quarantine Release Errors in Blood Establishments


FDA Center for Drug Evaluation and Research Develops Strategic Science and Research Agenda
The U.S. Food and Drug Administration report from its Center for Drug Evaluation and Research (CDER) identifies the current regulatory science needs that will guide CDER’s strategic planning of internal research initiatives and contributions to the development of agency regulatory science efforts.

Science Board Advisory Committee; Notice of Meeting [Docket No. FDA-2011-N-0002]


TGC Meeting 37, 6 April 2011
This summary has been prepared to provide advice of the Resolutions made by the Therapeutic Goods Committee at its 37th Meeting held on 6 April 2011.


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