Gene Therapy News and Research

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Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.

TransGenRx to produce protein-based therapeutics to support Esperion's research on HDL therapies

Esperion Therapeutics, a privately held biopharmaceutical company working to discover, develop and commercialize treatments for cardiovascular and metabolic diseases, today announced that TransGenRx, a global leader in transgenic technology, will produce protein-based therapeutics to be used in the company's research efforts targeting HDL therapies for the treatment of cardio-metabolic disease.

22 Jun 2010

Experimental cure for Type 1 diabetes

Researchers have developed an experimental cure for Type 1 diabetes, a disease that affects about one in every 400 to 600 children and adolescents. They will present their results in a mouse model of Type 1 diabetes on Sunday at The Endocrine Society's 92nd Annual Meeting in San Diego.

21 Jun 2010

End Duchenne Day to be celebrated on June 26, 2010: PPMD

Patricia A. Furlong, Founding President and CEO of Parent Project Muscular Dystrophy, the largest non-profit organization in the United States focused on finding a cure for Duchenne muscular dystrophy, announced that the Mayor of Denver, Colorado, John W. Hickenlooper, has proclaimed June 26, 2010 "End Duchenne Day" in recognition of PPMD's 16th Annual Connect Conference. Denver is playing host to the country's largest Duchenne-specific, international conference, June 24 – 27, 2010.

19 Jun 2010

CureDuchenne kicks off national fundraiser Pick Your Peak

CureDuchenne, a national 501(c)3 dedicated to raising funds and awareness in the fight against Duchenne Muscular Dystrophy, is kicking off a national fundraiser Pick Your Peak. Outdoor enthusiasts, philanthropists, families of boys with DMD, and anyone excited to get out and climb for a cause will climb the peak of their choice on June 19-20, 2010.

19 Jun 2010

E'shee Clinical Esthetic introduces revolutionary new products for skin care

E'shee Skin Care products are the result of a careful collaboration of skin care professionals, doctors and chemists; these revolutionary new products are the next step in skin care.

19 Jun 2010

UF administers Lumizyme for late-onset Pompe disease

The first commercially available treatment in the United States for patients with late-onset Pompe disease was administered Wednesday, June 16, at the University of Florida.

18 Jun 2010

Scientific American publishes article on "rebirth" of DNA vaccines

Inovio Pharmaceuticals, Inc., a leader in the development of therapeutic and preventive vaccines against cancers and infectious diseases, announced today that Scientific American magazine has published in its July issue an article entitled "DNA Drugs Come of Age." The article was co-authored by Dr. David Weiner, Chairman, Scientific Advisory Board, Inovio Pharmaceuticals, and Professor, Department of Pathology & Laboratory Medicine, at the University of Pennsylvania and Dr. Matthew Morrow, a post-doctorate research fellow at University of Pennsylvania.

17 Jun 2010

UF doctors administer newly available therapy for late-onset Pompe disease

The first commercially available treatment in the United States for patients with late-onset Pompe disease was administered today (Wednesday, June 16) at the University of Florida.

17 Jun 2010

Researchers find new way to prevent blindness caused by retinitis pigmentosa

Researchers at the University of Oklahoma Health Sciences Center have found a way to use a radical new type of gene therapy to prevent blindness caused by retinitis pigmentosa, giving hope to the estimated 100,000 Americans who suffer from this debilitating disease.

14 Jun 2010

UK scientists discover cancer-blocking activity in Down's syndrome chromosome

Cancer Research UK scientists have discovered cancer-blocking activity for genes carried on chromosome 21 - an extra copy of which is carried by people with Down's syndrome, reveals research published in Nature.

11 Jun 2010

Research may lead to better treatments for depression and substance abuse

In what has been hailed as a breakthrough, scientists from Columbia University Medical Center and Weill Cornell Medical College have outlined the molecular mechanism of membrane transport. The research shows how a protein transforms its shape to transport substances across the cell membrane in order to regulate transmission of the brain's messages across the synaptic gap from one neuron to another.

11 Jun 2010

PPMD to fund two promising research initiatives in Duchenne muscular dystrophy

Patricia A. Furlong, Founding President and CEO of Parent Project Muscular Dystrophy (PPMD), the largest non-profit organization in the United States focused on finding a cure for Duchenne muscular dystrophy (Duchenne), announced today that PPMD will be funding two critical, promising research initiatives in Duchenne.

11 Jun 2010

Novel gene replacement therapy holds promise for Spinal Muscular Atrophy

Children who suffer from the devastating disease Spinal Muscular Atrophy are set to benefit from a new breakthrough in therapy developments by researchers at the University of Sheffield.

10 Jun 2010

Takara Bio to distribute NuGEN's solutions in Japan, South Korea, China

NuGEN, the provider of innovative RNA and DNA sample preparation technologies that enable breakthrough genomic analysis, today announced it has signed a non-exclusive agreement with Takara Bio to distribute the full spectrum of NuGEN's next-generation sequencing, microarray and q-PCR nucleic acid amplification and sample preparation products in Japan, South Korea and China. In addition, Takara is authorized to offer services using NuGEN products in the same territory.

9 Jun 2010

SERCA2a gene therapy proves effective in people with advanced heart failure: Researchers

Researchers at Mount Sinai School of Medicine have developed a new gene therapy that is safe and effective in reversing advanced heart failure. SERCA2a (produced as MYDICAR-) is a gene therapy designed to stimulate production of an enzyme that enables the failing heart to pump more effectively.

4 Jun 2010

Sticky gel composed of MPEG-pDHA may help seal wounds created by surgery: Study

A compound found in sunless tanning spray may help to heal wounds following surgery, according to new results published by plastic surgeons from NewYork-Presbyterian Hospital/Weill Cornell Medical Center in New York City and biomedical engineers at Cornell University in Ithaca, N.Y., where the novel compound was developed.

1 Jun 2010

Celladon announces 6-month data from Phase 2 clinical trial of MYDICAR in advanced heart failure patients

Celladon Corp., a biopharmaceutical company focused on the discovery and development of innovative treatments for cardiovascular diseases, today announced that six-month data from its Phase 2 clinical trial of MYDICAR® show improvements in clinical outcomes and disease markers in advanced heart failure patients treated with the genetically targeted enzyme replacement therapy.

31 May 2010

Targeted Genetics's partner Celladon presents Phase II trial data with MYDICAR at Heart Failure Congress 2010

Targeted Genetics Corporation announced that its partner, Celladon Corporation, presented full data today that show the first Phase II trial with MYDICAR® for the treatment of advanced heart failure has met its primary safety and efficacy endpoints, as prospectively defined in the Phase II trial protocol.

31 May 2010

Hebrew Union College honors Dr. Dean Lorich with 2010 Roger E. Joseph Prize for humanitarian efforts

Dr. Dean Lorich was honored with the 2010 Roger E. Joseph Prize by Hebrew Union College for his humanitarian efforts in treating victims of the recent Haiti earthquake and in Landstuhl, Germany treating soldiers injured in Iraq and in Afghanistan. The award recognizes individuals making lasting contributions to the causes of human rights.

29 May 2010

SonoSite signs agreement to acquire privately held Visualsonics

SonoSite, Inc., the world leader and specialist in bedside and point-of-care ultrasound, today announced that it has signed an agreement to acquire privately held Visualsonics, a Toronto-based company focused on ultra high-frequency micro-ultrasound technology. The aggregate transaction value will be approximately $71 million net of cash and debt.

27 May 2010