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Latest Gene therapy Stories

2011-12-13 11:27:31

Gene found in humans and mice that protects transparency of cornea, may lead to new therapy A transparent cornea is essential for vision, which is why the eye has evolved to nourish the cornea without blood vessels. But for millions of people around the world, diseases of the eye or trauma spur the growth of blood vessels and can cause blindness. A new Northwestern Medicine study has identified a gene that plays a major role in maintaining clarity of the cornea in humans and mice -- and...

2011-12-13 06:00:00

RICHMOND, Calif., Dec. 13, 2011 /PRNewswire/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced the presentation of new data demonstrating the ability to permanently correct a disease gene in an adult mouse model of hemophilia B using systemic delivery of zinc finger nucleases (ZFNs) at the 53nd Annual Meeting of the American Society of Hematology (ASH). "We have demonstrated functional correction of a human gene for the clotting factor, Factor IX, with a single, systemic...

2011-12-10 11:30:00

SAN DIEGO, Dec. 10, 2011 /PRNewswire-USNewswire/ -- Despite major research advances over the last several decades that have helped deliver improved therapeutic options for leukemia, the condition remains deadly. Specialists are in need of new options to help diagnose the condition earlier and new therapies that will extend patients' lives. New research addressing important updates on the diagnosis and treatment of leukemia will be presented today at the 53rd Annual Meeting of the American...

2011-12-10 10:00:00

NASHVILLE, Tenn., Dec. 10, 2011 /PRNewswire/ -- Symptoms improved significantly in adults with the bleeding disorder hemophilia B following a single treatment with gene therapy developed by researchers at St. Jude Children's Research Hospital in Memphis and demonstrated to be safe in a clinical trial conducted at the University College London (UCL) in the U.K. To view the multimedia assets associated with this release, please click:...

2011-12-10 10:00:00

SAN DIEGO, Dec.10, 2011 /PRNewswire-USNewswire/ -- Bleeding and clotting disorders affect a large number of patients annually and, while accurate diagnosis is important, timely and effective treatment for these disorders is key, as they have the potential to quickly become life-threatening. Research examining novel treatment techniques for bleeding and clotting disorders will be presented today at the 53rd Annual Meeting of the American Society of Hematology. Bleeding disorders,...

2011-12-08 23:38:45

Three new locations for Crohn's Disease genes have been uncovered by scientists at UCL using a novel gene mapping approach. The complex genetic and environmental causes of Crohn's Disease (CD) have long been difficult to untangle. CD, a type of Inflammatory Bowel Disease that affects about 100 to 150 people per 100,000 in Europe, is characterised by inflammation in the gastrointestinal tract. Even though twin and family studies suggest a high heritability for CD of 50-60%, so far the...

2011-12-08 21:35:25

New gene editing technique would heal patients with their own cells Researchers at the Salk Institute for Biological Studies have developed a way to use patients' own cells to potentially cure sickle cell disease and many other disorders caused by mutations in a gene that helps produce blood hemoglobin. The technique uses cells from a patient's skin to generate induced pluripotent stem cells (iPSCs), which are capable of developing into various types of mature tissues -- including...

2011-12-07 20:02:13

In the bone marrow, blood-forming (hematopoietic) stem cells become one of the myriad kinds of blood cells in the body or they can self-renew, maintaining that pool. However, the lack or mutation of a gene for an enzyme called Dnmt3a (DNA methyltransferase 3a) results in an abundance of stem cells and a lack of blood cells, said a consortium of researchers led by those at in the Stem Cells and Regenerative Medicine Center at Baylor College of Medicine in a report online in the journal...

2011-12-01 10:52:46

Researchers at the University of North Carolina at Chapel Hill have shown that it is safe to cut and paste together different viruses in an effort to create the ultimate vehicle for gene therapy. In a phase I clinical trial, the investigators found no side effects from using a "chimeric" virus to deliver replacement genes for an essential muscle protein in patients with muscular dystrophy. "This trial demonstrates that gene therapy is no longer limited by the viruses we find in nature, and...

2011-12-01 01:46:31

New research in the FASEB Journal suggests that site-specific recombinases from either yeast or phages act not only to tag and target but also to exchange specific genes in DNA A combination of two techniques promises to improve the efficiency and effectiveness of experimental gene therapies, while also reducing potential side effects says a new research report published in the December 2011 issue of the FASEB Journal (http://www.fasebj.org). The report describes how scientists from...