Cell Therapy News Volume 11.33 | Aug 30 2010

Researcher Discovers Key Protein Involved in DNA Damage and Repair
Researchers have uncovered a protein called OTUB1 that blocks DNA damage in the cell. [Press release from Mount Sinai Hospital discussing online prepublication in Nature]

Targeted Drug Leads to Rapid Regression of Metastatic Melanoma in Patients with Mutated BRAF Gene
Use of an experimental targeted drug to treat metastatic melanoma tumors with a specific genetic signature was successful in more than 80 percent of patients in a Phase I clinical trial. [Press release from Massachusetts General Hospital discussing online prepublication in the New England Journal of Medicine]

Gene Involved in Fuchs Corneal Dystrophy Is Found
A research team has found a gene likely responsible for Fuchs corneal dystrophy. [Press release from the University of Oregon discussing online prepublication in the New England Journal of Medicine]

Gene Scan Finds Link Across Array of Childhood Brain Disorders
Mutations in a single gene can cause several types of developmental brain abnormalities that experts have traditionally considered different disorders. Researchers found those mutations through whole exome sequencing — a new gene scanning technology that cuts the cost and time of searching for rare mutations. [Press release from the National Institutes of Health discussing online prepublication in Nature]

Team Finds a Genetic Rarity: A Mutation That Restores Health
Scientists have unraveled the secrets of a rare phenomenon with potential therapeutic implications — disease-causing genes that show a high frequency of self-repair. [Press release from Yale University discussing online prepublication in Science]

Scientists Unveil Structure of Adenovirus, the Largest High-Resolution Complex Ever Found
Scientists have pieced together the structure of a human adenovirus — the largest complex ever determined at atomic resolution. The new findings about the virus may lead to more effective gene therapy and to new anti-viral drugs. [Press release from the Scripps Research Institute discussing online prepublication in Science]

A Better Way to Grow Stem Cells
Researchers have devised a synthetic surface that includes no foreign animal material and allows stem cells to stay alive and continue reproducing themselves for at least three months. [Press release from the Massachusetts Institute of Technology discussing online prepublication in Nature Materials]

Liver Cells Created from Patients’ Skin Cells
By creating diseased liver cells from a small sample of human skin, scientists have for the first time shown that stem cells can be used to model a diverse range of inherited disorders. [Press release from the University of Cambridge discussing online prepublication in the Journal of Clinical Investigation]

Gene Therapy to Treat Epilepsy a Step Closer
Researchers introduced genes that increase the expression of certain receptors for neuropeptides in the brain. [Press release from Lund University discussing online prepublication in Brain]

Double-Therapy Approach Effectively Inhibited Brain Cancer Recurrence
Researchers have identified a novel approach of combining chemotherapy with a targeted therapy to decrease the recurrence of glioblastoma multiforme. [Press release from the University of Massachusetts Medical School discussing online prepublication in Cancer Research]

Human Umbilical Cord Blood Cells Aid Lab Animal Brain Cell Survival After Simulated Stroke
Human umbilical cord blood cells used to treat cultured rat brain cells deprived of oxygen appear to protect astrocytes from cell death after stroke-like damage. [Press release from EurekAlert! discussing online prepublication in Stem Cell Reviews and Reports]

Seeing the World with New Eyes: Biosynthetic Corneas Restore Vision in Humans
A new study has shown that biosynthetic corneas can help regenerate and repair damaged eye tissue and improve vision in humans. [Press release from Linköping University discussing online prepublication in Science Translational Medicine]

CURRENT PUBLICATIONS (Ranked by Impact Factor of the Journal)

Inhibition of Mutated, Activated BRAF in Metastatic Melanoma
Treatment of metastatic melanoma with PLX4032 in patients with tumors that carry the V600E BRAF mutation resulted in complete or partial tumor regression in the majority of patients. [N Engl J Med]

E2-2 Protein and Fuchs's Corneal Dystrophy
Genetic variation in transcription factor 4 gene (TCF4) contributes to the development of Fuchs's corneal dystrophy. [N Engl J Med]

Non-Canonical Inhibition of DNA Damage-Dependent Ubiquitination by OTUB1
Researchers report that OTUB1, a deubiquitinating enzyme, is an inhibitor of double-strand break-induced chromatin ubiquitination. [Nature]

Whole Exome Sequencing Identifies WDR62 Mutations in Severe Brain Cortical Malformations
Researchers demonstrate the use of whole-exome sequencing to identify recessive mutations in WD repeat domain 62 (WDR62) as the cause of a wide spectrum of severe cerebral cortical malformations including microcephaly, pachygyria with cortical thickening as well as hypoplasia of the corpus callosum. [Nature]
 
Mitotic Recombination in Patients with Ichthyosis Causes Reversion of Dominant Mutations in KRT10
The high frequency of somatic reversion in ichthyosis with confetti suggests that revertant stem cell clones are under strong positive selection and/or that the rate of mitotic recombination is elevated in individuals with this disorder. [Science]
 
Crystal Structure of Human Adenovirus at 3.5 Angstrom Resolution
Researchers report the x-ray structure at 3.5 angstrom resolution of the 150-megadalton adenovirus capsid containing nearly 1 million amino acids. [Science]

Combinatorial Development of Biomaterials for Clonal Growth of Human Pluripotent Stem Cells
Researchers developed the first chemically defined, xeno-free, feeder-free synthetic substrates to support robust self-renewal of fully dissociated human embryonic stem and induced pluripotent stem cells. [Nat Mater]

Modeling Inherited Metabolic Disorders of the Liver Using Human Induced Pluripotent Stem Cells
Researchers report a simple and effective platform for hepatocyte generation from patient-specific human induced pluripotent stem cells. [J Clin Invest]

A Biosynthetic Alternative to Human Donor Tissue for Inducing Corneal Regeneration: 24-Month Follow-Up of a Phase I Clinical Study
With further optimization, biosynthetic corneal implants could offer a safe and effective alternative to the implantation of human tissue to help address the current donor cornea shortage. [Sci Transl Med]

Dynamic MicroRNA Expression Programs During Cardiac Differentiation of Human Embryonic Stem Cells: Role for miR-499
Data give significant insight into the regulatory networks that govern human embryonic stem cells (hESC) differentiation, and highlights the ability of microRNAs to perturb, and even control, the genes that are involved in cardiac specification of hESCs. [Circ Cardiovasc Genet]

Federal Judge: Obama Stem Cell Policy Halted
Research on embryonic stem cells funded by the U.S. government must stop immediately for a court-ordered review, Judge Royce Lamberth of the U.S. District Court for the District of Columbia ruled. [United States District Court for the District of Columbia]

Request for Comments on Synthetic Biology (FR Doc. 2010-21359) [Department of Health and Human Services, United States]

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Roche Enters $1.1 Billion Drug Deal With U.S.'s Aileron
Roche Holding AG entered into a partnership deal with U.S.-based biopharmaceutical firm Aileron Therapeutics to produce a novel class of drugs to treat diseases such as inflammation and metabolic ailments, speeding up recent efforts to broaden its R&D pipeline by entering into licensing agreements and acquisitions. [The Wall Street Journal]

Wake Forest Institute for Regenerative Medicine Signs a Memorandum of Understanding with NUI Galway
The Network of Excellence for Functional Biomaterials and the Regenerative Medicine Institute at National University of Ireland (NUI) Galway have formally signed a Memorandum of Understanding with Wake Forest Institute for Regenerative Medicine. [National University of Ireland, Galway Press Release]

Soligenix Announces Award of $1.2 Million FDA Orphan Products Development Grant
Soligenix, Inc. announced that it has been awarded a three year $1.2 million clinical research grant by the Office of Orphan Products Development of the U.S. Food and Drug Administration (FDA) pursuant to its Orphan Products Development Program. [Soligenix, Inc. Press Release]
 
Fox Center Receives Donation to Further Research on Stem Cell Therapy for Corneal Scarring
The Louis J. Fox Center for Vision Restoration of University of Pittsburgh Medical Center (UPMC) and the University of Pittsburgh received a $244,000 donation from the Western Pennsylvania Medical Eye Bank Foundation. The donation, to be matched by UPMC, will be used to further research on stem cell therapy for corneal scarring. [University of Pittsburgh Medical Center Press Release]
 
Neuralstem Files FDA Application for First Chronic Spinal Cord Injury Stem Cell Trial
Neuralstem, Inc. announced that it has filed an Investigational New Drug application with the United States Food and Drug Administration (FDA) to begin a Phase I safety clinical trial for chronic spinal cord injury with its spinal cord stem cells. [Neuralstem, Inc. Press Release]
 
Shire Announces European Approval of VPRIV® (velaglucerase alfa) for the Treatment of Type 1 Gaucher Disease
Shire plc announced that the European Commission has granted marketing authorization for VPRIV® (velaglucerase alfa), a human cell line derived enzyme replacement therapy for the long-term treatment of type 1 Gaucher disease. [Shire plc Press Release]
 
Celera Announces Issuance of United States Patent Relating to LPA Gene Variant Associated with Increased Risk for Myocardial Infarction
Celera Corporation announced that the United States Patent and Trademark Office has issued United States Patent 7,781,168 relating to methods of determining heart attack risk by detecting the Ile4399Met genetic polymorphism in the protease-like domain of LPA.
[Celera Corporation Press Release]

European Patent Office Allows Advaxis/University of Pennsylvania Modified Listeria Protein Listeriolysin O Patent
Advaxis, Inc. has been notified by the European Patent Office that patent application WO2001/072329, for the use of Prokaryotic PEST-Like Peptides for Enhancing Immunogenicity of Antigen has been allowed for issuance. [Advaxis, Inc. Press Release]

NIH Director Collins Says Cell Ruling Won't Halt Most Ongoing Grants
National Institutes of Health (NIH) Director Francis Collins said that the court injunction blocking federally funded research on human embryonic stem cells will not affect grant payments that have already gone out the door this year.

NIH Director's Response to Stem Cell Injunction
 
Office of the Director, National Institutes of Health; Notice of Meeting (FR Doc. 2010-20861)

Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2010-21357)
 
National Cancer Institute; Notice of Closed Meeting (FR Doc. 2010-21282)

National Human Genome Research Institute; Notice of Closed Meeting (FR Doc. 2010-21317)

National Human Genome Research Institute; Notice of Meetings (FR Doc. 2010-20858)
 
National Center for Research Resources; Notice of Closed Meeting (FR Doc. 2010-21280)
 
National Cancer Institute's Best Practices for Biospecimen Resources (FR Doc. 2010-20872)
 
Revolutionary Genome Sequencing Technologies — The $1000 Genome (SBIR [R43/R44]) (RFA-HG-10-014)

Public Workshop on Cell and Gene Therapy Clinical Trials in Pediatric Populations

Guidance for Industry: Electronic Submission of Lot Distribution Reports for Biological Products

FOOD AND DRUG ADMINISTRATION (United States)

Roche, Genentech Hit with Refuse-to-File Letter
The FDA has handed Roche and Genentech a refuse-to-file letter for the developers' BLA for trastuzumab-DM1.

Food and Drug Administration Clinical Trial Requirements, Regulations, Compliance, and Good Clinical Practice; Public Workshop [FR Doc. 2010-20834]

Public Workshop on Medical Devices and Nanotechnology: Manufacturing, Characterization, and Biocompatibility Considerations [FR Doc. 2010-20837]

EVENTS (Listed by Date)

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

3rd Biobridge “Generation Regeneration” Conference
October 11, 2010
Geneva, Switzerland

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

NEW 6th Modern Drug Discovery & Development Summit (M3D)
October 20-22, 2010
San Francisco, 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

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

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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