Cell Therapy News Volume 11.31 | Aug 16 2010

Stem Cell Technology Yields First ‘Knockout’ Rats
Researchers have, for the first time, generated “knockout” rats through embryonic stem cell-based gene targeting. [Press release from the University of Southern California discussing online prepublication in Nature]

Researchers Use Stem Cells to Treat Children with Life-Threatening, Blistering Skin Disease
Researchers have for the first time used stem cells from bone marrow to repair the skin of patients with a fatal skin disease called recessive dystrophic epidermolysis bullosa. [Press release from the University of Minnesota discussing online prepublication in the New England Journal of Medicine]

Researchers Discover That One Type of Stem Cell Creates a Niche for Another Type Within Bone Marrow
Researchers report that hematopoietic stem cells (HSCs) retain their unique features in response to signals from another stem cell population, mesenchymal stem cells, which create a supportive bone marrow niche for the HSCs. [Press release from Cold Spring Harbor Laboratory discussing online prepublication in Nature]

Discovered Gene Causes Kabuki Syndrome
Using a new, rapid and less expensive DNA sequencing strategy, scientists have discovered genetic alterations that account for most cases of Kabuki syndrome. Instead of sequencing the entire human genome, the new approach sequences just the exome. [Press release from the National Institutes of Health discussing online prepublication in Nature Genetics]

Immune System Gene Linked with Parkinson's: Study
A gene linked with the immune system may play a role in developing Parkinson's disease, researchers said, marking a possible advance in the search for effective treatments. [Press release from Reuters discussing online prepublication in Nature Genetics]

Genetic Differences That Make Some People Susceptible to Meningitis Revealed
Genetic differences that make some people susceptible to developing meningococcal meningitis and septicaemia, and others naturally immune, are revealed in a new study of over 6,000 people. [Press release from Imperial College London discussing online prepublication in Nature Genetics]

More Cancer-Fighting Power — Mouse with Highly Effective Components of the Human Immune System Developed
Researchers modified T cell receptors so that they would no longer ignore cancer cells, but instead specifically track and recognize them. [Press release from the Max Delbrück Center for Molecular Medicine discussing online prepublication in Nature Medicine]

Human Embryonic Stem Cells and Reprogrammed Cells Virtually Identical
Human embryonic stem cells and induced pluripotent stem cells exhibit very few differences in their gene expression signatures and are nearly indistinguishable in their chromatin state. [Press release from the Whitehead Institute for Biomedical Research discussing online prepublication in Cell Stem Cell]

Death Dance Reveals Secrets of Apoptosis in Dissociated Human Embryonic Stem Cells
Researchers have unraveled the mystery of why human embryonic stem cells and induced pluripotent stem cells undergo programmed cell death when cultured in isolation. [Press release from the RIKEN Center for Developmental Biology discussing online prepublication in Cell Stem Cell]

SUMO Works with Replication Protein A Complex to Repair DNA
A team of investigators has shown for the first time that the small protein SUMO can team up with the replication protein A complex to facilitate DNA repair. [Press release from the University of Texas MD Anderson Cancer Center discussing online prepublication in Molecular Cell]

Merlin Protein Found to Control Liver Stem Cells, Prevent Tumor Development
A research team describes finding that the protein called merlin, encoded by the NF2 (neurofibromatosis type 2) gene, controls the activity of adult stem cells that give rise to the two major types of liver cells. [Press release from EurekAlert! discussing online prepublication in Genes and Development]

Scientists Map All Mammalian Gene Interactions
In one of the first efforts of its kind, researchers have taken mammalian genome maps, including human maps, one step further by showing not just the order in which genes fall in the genome but which genes actually interact. [Press release from the University of California, Los Angeles discussing online prepublication in Genome Research]

A Strategy to Fix a Broken Heart
Engineers and physicians have built a scaffold that supports the growth and integration of stem cell-derived cardiac muscle cells. [Press release from the University of Washington discussing online prepublication in the Proceedings of the National Academy of Sciences USA]

MicroRNA Molecule Increases Number of Blood Stem Cells, May Help Improve Cancer Treatment 
Researchers identified a tiny RNA molecule that increases the number of hematopoietic stem cells, an advance that may improve treatment of blood system cancers. [Press release from Massachusetts General Hospital discussing online prepublication in the Proceedings of the National Academy of Sciences USA]

Researchers Make Major Stem Cell Discovery
Using adult cells obtained from humans and the bone marrow, spleens and kidneys of mice, researchers reprogrammed the cells into a stem cell-like condition. [Press release from the Oklahoma Medical Research Foundation discussing online prepublication in Stem Cells]

Scorpion Venom Improves Anti-Brain Cancer Therapy
Scientists have found that an ingredient in the venom of the ‘deathstalker’ scorpion could help gene therapy become an effective treatment for brain cancer. [Press release from the Times of India discussing online prepublication in ACS Nano]

Personalized Genome Sequencing in Cancer Treatment–A Major Breakthrough in Care
Researchers have provided the first published example of genome-scale RNA and DNA sequencing of a tumor to aid in clinical decision making and therapeutic choice. [Press release from the B.C. Cancer Agency Genome Sciences Centre discussing online prepublication in Genome Biology]

Portuguese Scientists Discover an Extraordinary New Type of White Blood Cell
Researchers found a new type of white blood cell baptized NKTreg that, once activated, migrates into the liver and suppress any immune response in its vicinity. [Press release from Medical News Today discussing online prepublication in the Journal of Immunology]

Scientists Use Salmonella Bug to Kill Cancer Cells
Treating tumors with salmonella bacteria can induce an immune response that kills cancer cells, scientists have found — a discovery that may help them create tumor-killing immune cells to inject into patients. [Press release from Reuters discussing online prepublication in Science Translational Medicine]

Taiwanese Researchers Find Breakthrough in Stem Cell Study
Scientists at the National Health Research Institutes announced a breakthrough in stem cell research through experiments that successfully turn cells in the umbilical vein into “all-purpose” induced pluripotent stem cells. [The China Post]

CURRENT PUBLICATIONS (Ranked by Impact Factor of the Journal)

Bone Marrow Transplantation for Recessive Dystrophic Epidermolysis Bullosa
Increased type VII collagen deposition and a sustained presence of donor cells were found in the skin of children with recessive dystrophic epidermolysis bullosa after allogeneic bone marrow transplantation. [N Engl J Med]

Production of p53 Gene Knockout Rats by Homologous Recombination in Embryonic Stem Cells
Researchers report the generation of gene knockout rats using the embryonic stem cell-based gene targeting technology. [Nature]

Mesenchymal and Hematopoietic Stem Cells Form a Unique Bone Marrow Niche
Results uncover an unprecedented partnership between two distinct somatic stem-cell types and are indicative of a unique niche in the bone marrow made of heterotypic stem-cell pairs. [Nature]

Exome Sequencing Identifies MLL2 Mutations as a Cause of Kabuki Syndrome
Researchers demonstrate the successful application of exome sequencing to discover a gene for an autosomal dominant disorder, Kabuki syndrome. [Nat Genet]

Common Genetic Variation in the HLA Region is Associated with Late-Onset Sporadic Parkinson's Disease
The genetic association with HLA supports the involvement of the immune system in Parkinson's disease and offers new targets for drug development. [Nat Genet]

Genome-Wide Association Study Identifies Variants in the CFH Region Associated with Host Susceptibility to Meningococcal Disease
The study suggests that host genetic variation in the regulators of complement activation plays a role in determining the occurrence of invasive disease versus asymptomatic colonization by this pathogen. [Nat Genet]

Transgenic Mice with a Diverse Human T Cell Antigen Receptor Repertoire
Researchers generated transgenic mice with the entire human TCR(alpha)(beta) gene loci (1.1 and 0.7 Mb), whose T cells express a diverse human T cell receptor (TCR) repertoire that compensates for mouse TCR deficiency. [Nat Med]

Chromatin Structure and Gene Expression Programs of Human Embryonic and Induced Pluripotent Stem Cells
Genome-wide maps of nucleosomes with histone H3K4me3 and H3K27me3 modifications indicate that there is little difference between embryonic stem cells (ESCs) and induced pluripotent stem cells (iPSCs) with respect to these marks. Gene expression profiles confirm that the transcriptional programs of ESCs and iPSCs show very few consistent differences. [Cell Stem Cell]

Molecular Pathway and Cell State Responsible for Dissociation-Induced Apoptosis in Human Pluripotent Stem Cells
Researchers show that the apoptosis of human embryonic stem cells, which is of a nonanoikis type, is caused by ROCK-dependent hyperactivation of actomyosin and efficiently suppressed by the myosin inhibitor Blebbistatin. [Cell Stem Cell]

Regulation of DNA Repair through DeSUMOylation and SUMOylation of Replication Protein A Complex
Results demonstrate that SUMOylation status of the 70 kDa subunit of replication protein A complex plays a critical role in the regulation of DNA repair through homologous recombination. [Mol Cell]

Nf2/Merlin Controls Progenitor Homeostasis and Tumorigenesis in the Liver
Researchers report that liver-specific deletion of the neurofibromatosis type 2 (Nf2) tumor suppressor gene in the developing or adult mouse specifically yields a dramatic, progressive expansion of progenitor cells throughout the liver without affecting differentiated hepatocytes. [Genes Dev]

EMA and U.S. FDA Seek Potential Candidate Companies for Joint GMP Inspection Program
The European Medicines Agency (EMA) and the Food and Drug Administration of the United States of America (U.S. FDA) continue to seek potential candidate companies for a joint GMP inspection pilot programmed for manufacturers of medicinal products. [European Medicines Agency, European Union]

Human Genetics Commission Sets New Standards for Direct-to-Consumer Genetic Tests
The Common Framework of Principles for direct-to-consumer genetic testing services, published by the Human Genetics Commission, aims to ensure that people who choose to buy genetic tests themselves can have confidence that the companies selling them follow basic principles of consent, data protection, truth in marketing, scientific rigor and balanced interpretation. [Human Genetics Commission, United Kingdom]

EU Agency Allows Access to Drug Side Effects Data
The European Medicines Agency has agreed to provide access to studies about drug side effects, following criticism over data secrecy from the European Ombudsman. [European Medicines Agency, European Union]

University of California, Berkeley Alters DNA Testing Program
The California Department of Public Health has instructed the University of California, Berkeley, not to proceed with a portion of its ground-breaking program to educate students about genetic testing and personalized medicine. [California Department of Public Health, United States]

International Society for Cellular Therapy (ISCT) Joins Forces with Roche and Genzyme to Launch Cell Therapy Industry Partnership
Many companies in the cell therapy regenerative medicine market including Roche, Genzyme, Athersys, Miltenyi Biotec, Hospira, Lonza and Life Technologies will join ISCT to launch a series of new initiatives that will create greater strategic alignment within the industry and drive late stage clinical development. [International Society for Cellular Therapy Press Release]

Alectos Therapeutics Announces Collaboration with Merck to Develop Drugs for Alzheimer’s Disease
Alectos Therapeutics Inc. announced a research collaboration with Merck to identify and develop compounds that modulate O-linked N-acetylglucosaminidase (O-GlcNAcase), an enzyme that is believed to be involved in the development of Alzheimer’s disease and potentially other disorders. [Alectos Therapeutics Inc. Press Release]

Numerate and Intellikine Conclude Research Collaboration to Discover Selective Inhibitors Against PI3 Kinase Alpha
Numerate, Inc. and Intellikine, Inc. announced the successful conclusion of their research collaboration to discover novel small molecule compounds targeting the PI3K/mTOR signaling pathway. [Numerate, Inc. Press Release]

OriGene Technologies Acquires Blue Heron Biotechnology, a Leading U.S. Based Gene Synthesis Company
OriGene Technologies, Inc. announced the acquisition of Blue Heron Biotechnology. [OriGene Technologies, Inc. Press Release]

Acquisition of Sigma Pharmaceuticals Limited by Aspen
Aspen announced the acquisition of the pharmaceutical business of Sigma Pharmaceuticals Limited. [Aspen Press Release] 

ThermoGenesis Announces Stockholder Authorization of Reverse Split
ThermoGenesis Corp. said that its Board of Directors has unanimously approved a reverse split of the Company’s common stock at a ratio of one new share for each four existing shares, effective at the close of business on Thursday, August 26. [ThermoGenesis Corporation Press Release]

Emergent BioSolutions to Acquire Trubion Pharmaceuticals
Emergent BioSolutions Inc. announced it has entered into a definitive agreement to acquire Trubion Pharmaceuticals, Inc. for upfront consideration of $96.8 million of value and up to $38.7 million of success-based milestones, resulting in a total consideration of up to $135.5 million. [Emergent BioSolutions Inc. Press Release]

Dicerna Closes $25 Million Series B Financing
Dicerna Pharmaceuticals, Inc. announced that it has closed a $25 million Series B round of financing. [Dicerna Pharmaceuticals, Inc. Press Release]

Parent Project Muscular Dystrophy Awards $600K to Nationwide Children’s Hospital for Gene Therapy Study
Parent Project Muscular Dystrophy announced that it will award a $600,000 grant to Nationwide Children’s Hospital in Columbus, Ohio to conduct clinical testing of a promising gene therapy technique for muscle disease. [Nationwide Children’s Hospital Press Release]

NIH EUREKA Award Will Enable Design of New Brain Tumor Treatment
The Georgia Institute of Technology has received a EUREKA grant from the National Institutes of Health (NIH) to design a new way to treat invasive brain tumors by capturing the migrating cells that spread the disease. [Georgia Institute of Technology Press Release]

FDA Approves Innovative Liver Cell Therapy for Clinical Trial in the U.S.
The liver cell preparation by Cytonet GmbH & Co. KG received an Investigational New Drug allowance from the U.S Food and Drug Administration (FDA). This is the first time a later stage clinical trial with liver cell therapy can now be started in the U.S. [Cytonet GmbH & Co. KG Press Release]

Drug Managers Medco, CVS Giving Gene Tests to Millions in U.S.
Medco Health Solutions Inc. and CVS Caremark Corp., pharmacy benefit managers for about 120 million people in the U.S., are spearheading the use of genetic tests by offering them to some members before providing certain drugs. [Bloomberg]

Tissue Regenix Receives Approval to Commence Sales of Its Lead Product, the dCELL® Vascular Patch
Tissue Regenix announced that it has received European CE Marking for its dCELL® Vascular Patch. [Tissue Regenix Press Release]

Fibrocell Science, Inc. Submits Clinical Study Report to U.S. Food & Drug Administration for Phase II/III Trial of Azficel-T in Acne Scarring
Fibrocell Science, Inc. announced that it has submitted a clinical study report for its Phase II/III study of azficel-T for the treatment of moderate to severe acne scars to the U.S. Food & Drug Administration (FDA). [Fibrocell Science, Inc. Press Release]

Halozyme Announces Issuance of U.S. Patent for rHuPH20 Enzyme Platform
Halozyme Therapeutics, Inc. announced the issuance of U.S. Patent No. 7,767,429 claiming its proprietary recombinant human hyaluronidase enzyme platform (rHuPH20). [Halozyme Therapeutics, Inc. Press Release]

Cytori Issued Celution® System Patent for Orthopedic and Spinal Disc Diseases
Cytori Therapeutics has received U.S. Patent No. 7,771,716 (the '716 patent) for Celution® System based methods of treating musculoskeletal disorders such as spinal disc disease. [Cytori Therapeutics Press Release]

BioMimetic Therapeutics Receives Orphan Drug Designation for rhPDGF-BB Treatment of Osteochondritis Dissecans
BioMimetic Therapeutics, Inc. announced that it has received orphan drug designation from the Food and Drug Administration for its protein therapeutic, recombinant human platelet derived growth factor (rhPDGF-BB), to be used in conjunction with autograft and/or commercially available osteochondral allograft for the treatment of osteochondritis dissecans of the knee, elbow or ankle.

Clarification of Eligible Institutions for PAR-10-221 (R01) and PAR-10-222 (R21): Biophysical and Biomechanical Aspects of Embryonic Development (NOT-HD-10-020)

Proposed Collection; Comment Request; NIH NCI Central Institutional Review Board (CIRB) Initiative (NCI) (FR Doc. 2010-20167)

Center for Scientific Review, Notice of Closed Meetings (FR Doc. 2010-19938)

Center for Scientific Review, Notice of Closed Meetings (FR Doc. 2010- 20023)

Center for Scientific Review, Notice of Closed Meetings (FR Doc. 2010- 20044)

Office of the Director, National Institutes of Health; Notice of Meeting (FR Doc. 2010-20021)

National Human Genome Research Institute; Notice of Closed Meeting (FR Doc. 2010-19939)

Basic Research on Self-Regulation (R21) (RFA-AG-11-010)

National Center for Research Resources; Notice of Meeting (FR Doc. 2010-20014)

7342.007 Addendum – Imported Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps)

7342.007 – Imported CBER-Regulated Products 

Currently Cleared CBER 510(k) Clearances

July 26-27, 2010: Blood Products Advisory Committee Meeting Presentations

Complete List of Licensed Products and Establishments

FOOD AND DRUG ADMINISTRATION (United States)

FDA Acts on Unapproved Cell Therapy for Bad Knees
The FDA is seeking to stop a Colorado company from offering an autologous stem cell therapy for arthritic knees and other orthopedic problems, saying that the therapy is unapproved, misbranded, and adulterated.

Memorandum of Understanding Between United States Food and Drug Administration and the Centers for Medicare and Medicaid Services [Docket No. FDA-2010-N-0004]

Workshop on Optimizing Clinical Trial Design for the Development of Pediatric Cardiovascular Devices [Docket No. FDA-2010-N-0001]

Comprehensive List of Guidance Documents at the Food and Drug Administration [Docket No. FDA-1998-N-0050] (formerly Docket No. 1998N-0046)

EVENTS (Listed by Date)

CHI's 5th Annual Immunotherapeutics and Vaccine Summit (ImVacS)
August 17-19, 2010
Cambridge, United States

Select Biosciences 3rd Annual Stem Cells Europe Conference
August 24-25, 2010
Edinburgh, Scotland

Select Biosciences 2nd Annual World Biobanking Summit
August 24-25, 2010
Edinburgh, Scotland

Select Biosciences Inaugural Cellular Therapy Summit
August 24-25, 2010
Edinburgh, Scotland

Viral Vectors in Gene Therapy: Applications and Novel Production Methods
August 26-September 4, 2010
Kuopio, Finland

European Workshop on Skin Stem Cells
August 31-September 2, 2010
Cambridge, United Kingdom

Clinical Trials International Summit 2010
September 1-2, 2010
Shanghai, China

Third Annual Limbal Stem Cell Meeting
September 10, 2010
Reading, United Kingdom

International Society for Cellular Therapy (ISCT) – Europe 2nd Regional Meeting
September 11-14, 2010
Belgirate, Italy

Hydra VI: The European Summer School on Stem Cells & Regenerative Medicine
September 11-17, 2010
Hydra, Greece

Joint Metastasis Research Society – American Association of Cancer Research Conference on Metastasis and the Tumor Microenvironment
September 12-15, 2010
Philadelphia, United States

Stem Cells USA & Regenerative Medicine Congress 2010
September 13-15, 2010
Philadelphia, United States

Select Biosciences 7th Annual RNAi & miRNA Europe
September 14-15, 2010
Dublin, Ireland

Select Biosciences Inaugural Epigenetics Europe Conference
September 14-15, 2010
Dublin, Ireland

Select Biosciences Inaugural Genomics Automation Europe Conference
September 14-15, 2010
Dublin, Ireland

Tissue Engineering & Regenerative Medicine International Society (TERMIS) – Asia Pacific 2010
September 15-17, 2010
Sydney, Australia

First Annual BIO India International Partnering Conference
September 21-22, 2010
Hyderabad, India

Cellular Therapy of Cancer Symposium – ATTACK Project Meeting
September 24-27, 2010
Montpellier, France

International Society for Cellular Therapy 2nd Annual Symposium on Stem Cell Translation: Best Practices & Regulatory Considerations
September 27-28, 2010
San Francisco, United States

International Society for Cell and Gene Therapy of Cancer (ISCGT) Congress
September 27-29, 2010
Doha, Qatar

4th American Association of Cancer Research International Conference on Molecular Diagnostics in Cancer Therapeutic Development
September 27-30, 2010
Denver, United States

Select Biosciences 6th Annual Ion Channel Targets Conference
September 28-29, 2010
Washington, DC, United States

Cytokines 2010: Cytokines in Infectious Diseases, Autoimmune Disorders and Cancer
October 3-7, 2010
Chicago, United States

2010 World Stem Cell Summit
October 4-6, 2010
Detroit, United States

9th Annual BIO Investor Forum
October 5-6, 2010
San Francisco, United States

5th World Congress on Preventive & Regenerative Medicine (WCRM)
October 5-7, 2010
Hannover, Germany

17th Annual Meeting German Society for Gene Therapy (DG-GT e.V.)
October 6-9, 2010
Munich, Germany

COGEM Symposium: GM Viruses as Medicine: Panacea or Pandora's Box?
October 7, 2010
Amsterdam, The Netherlands

American Association of Blood Banks (AABB) Annual Meeting & Cellular Therapy and Transfusion Medicine Expo (CTTXPO) 2010
October 9-12, 2010
Baltimore, United States

18th Annual BioPartnering Europe
October 10-12, 2010
London, United Kingdom

New York Stem Cell Foundation Fifth Annual Translational Stem Cell Research Meeting
October 12-13, 2010
New York, United States

Translational Cancer Research for Basic Scientists
October 17-22, 2010
Boston, United States

2010 NanoMedicine Summit
October 18-19, 2010
Cleveland, United States

BIO Intellectual Property Counsels Committee Fall Conference and Committee Meeting
October 18-20, 2010
Boston, United States

European Society of Gene and Cell Therapy (ESGCT) 18th Annual Congress
October 22-25, 2010
Milan, Italy

3rd Annual Dubai Congress on Anti-Aging and Aesthetic Medicine (DCAAAM)
October 26-27, 2010
Dubai, United Arab Emirates

World Stem Cells & Regenerative Medicine Congress Asia 2010
October 26-29, 2010
Singapore City, Singapore

Select Biosciences 3rd Annual Molecular Diagnostics World Congress
October 28-29, 2010
San Diego, United States

Fraunhofer Life Science Symposium 2010
October 29-30, 2010
Leipzig, Germany

Stem Cell Society Singapore Symposium 2010: Translating Science to Therapy
November 2-3, 2010
Singapore City, Singapore

NEW BioMalaysia 2010 Conference and Exhibition
November 2-4, 2010
Kuala Lumpur, Malaysia

World Cord Blood Congress
November 4-7, 2010
Marseille, France

Select Biosciences 5th Annual European Biomarkers Summit
November 9-10, 2010
Florence, Italy

Select Biosciences International Forum on Stem Cells
November 12-13, 2010
Tianjin, China

Select Biosciences 2nd Annual RNAi Asia Conference
November 15-16, 2010
Singapore City, Singapore

BIO-Europe International Partnering Conference
November 15-17, 2010
Munich, Germany

9th Annual Gene Therapy Symposium for Heart, Lung, and Blood Diseases
November 17-19, 2010
Sonoma, United States

8th Annual Commercial Translation of Regenerative Medicine Conference
November 18-19, 2010
London, United Kingdom

Stem Cell Network 10th Annual Scientific Meeting
November 22-24, 2010
Calgary, Canada

HealthTech and Medicines Knowledge Transfer Network 7th Annual BioProcessUK Conference
November 24-25, 2010
Manchester, United Kingdom

NEW 2nd European Association for Cardio-Thoracic Surgery (EACTS) Meeting on Cardiac and Pulmonary Regeneration
December 2-3, 2010
Vienna, Austria

Tissue Engineering & Regenerative Medicine International Society (TERMIS) – North America 2010 Annual Conference
December 5-8, 2010
Orlando, United States

Select Biosciences 5th Annual Stem Cells World Congress
January 24-25, 2011
San Diego, United States

BIO Asia International Partnering Conference
January 24-25, 2011
Tokyo, Japan

Phacilitate 7th Annual Cell & Gene Therapy Forum 2011
January 24-26, 2011
Washington DC, United States

Visit our events page to stay up to date with the latest events in the cell, gene and immunotherapy community.

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