Latest Gene therapy Stories

PHILADELPHIA, Nov. 15, 2011 /PRNewswire-USNewswire/ -- Researchers at Hadassah Hospital in Israel, led by Dr. Eyal Banin, have completed a clinical trial that tested the use of gene therapy to restore sight to patients suffering from Leber's Congenital Amaurosis (LCA). Dana and Yossi, two participants in this study, suffer from Leber's Congenital Amaurosis (LCA), the most severe form of all inherited retinal dystrophies causing congenital blindness. Like others affected, they have...

CLEVELAND and GAITHERSBURG, Md., Nov. 14, 2011 /PRNewswire/ -- University Hospitals (UH) Case Medical Center, Case Western Reserve University School of Medicine and Lentigen Corporation announced today the initiation of a novel Phase I clinical trial of LG631 gene therapy for the protection of hematopoietic stem cells (HSCs) from the dose limiting toxicity of chemotherapy with Temodar. Approximately 17,000 Americans are diagnosed with glioblastoma every year and only two percent of them...

Study of hereditary Parkinson's finds that mitochondria can't be cleared out when damaged BOSTON, Nov. 10, 2011 /PRNewswire-USNewswire/ -- Current thinking about Parkinson's disease is that it's a disorder of mitochondria, the energy-producing organelles inside cells, causing neurons in the brain's substantia nigra to die or become impaired. A study from Children's Hospital Boston now shows that genetic mutations causing a hereditary form of Parkinson's disease cause mitochondria to...

Scientists at Penn’s Perelman School of Medicine Center for Research in FOP and Related Disorders have developed a new genetic approach to specifically block the damaged copy of the gene for a rare bone disease, while leaving the normal copy untouched. Lead author Josef Kaplan, PhD, postdoctoral fellow; and senior authors Eileen M. Shore, PhD, and Frederick S. Kaplan, MD, both from the Department of Orthopaedic Surgery, published this new proof-of-principle approach for treating the...

For the first time, researchers have combined gene therapy and stem cell transplantation to successfully reverse the severe, crippling bleeding disorder hemophilia A in large animals, opening the door to the development of new therapies for human patients. Researchers at Wake Forest Baptist Medical Center's Institute for Regenerative Medicine, collaborating with other institutions, report in Experimental Hematology that a single injection of genetically-modified adult stem cells in two...

HOUSTON, Nov. 2, 2011 /PRNewswire/ -- Bellicum Pharmaceuticals, Inc. today announced publication in The New England Journal of Medicine of clinical results using a new therapy that demonstrated rapid and complete reversal of graft-versus-host disease (GVHD). The paper, Inducible Apoptosis as a Safety Switch for Adoptive Cell Therapy, reports on the use of Bellicum's CaspaCIDe(TM) technology to eliminate donor T cells causing GVHD, a serious and sometimes fatal complication of allogeneic...

Researchers at Ohio State University Medical Center and Nationwide Children’s Hospital have developed a viral vector designed to deliver a gene into the eyes of people born with an inherited, progressive form of blindness that affects mainly males. The vector is part of a clinical trial investigating the use of gene therapy to cure choroideremia, a disease that affects an estimated 100,000 people worldwide. The trial is being conducted by researchers at the University of Oxford in...

OXFORD, England, October 31, 2011 /PRNewswire/ -- - Research and development agreement signed with Mayo Clinic - Oxford BioMedica plc ("Oxford BioMedica" or "the Company") (LSE: OXB), the leading gene-based biopharmaceutical company, today announces that it has entered into a research and development collaboration with Mayo Clinic, Rochester (USA) to develop a novel gene therapy for the treatment of chronic glaucoma. Under the terms of the agreement, Mayo Clinic and...

A UK man is being treated for the progressive genetic disorder, choroideraemia, in an attempt to reverse his blindness. Jonathan Wyatt was able to see normally until about the age of 19 when he began having problems seeing in the dark. He was told by doctors that his vision would get progressively worse until eventually going blind, reports BBC News. Poor eyesight didn’t stop Wyatt, now 63 years old, from a career as a barrister but 10 years ago he found himself having difficulty...

AMSTERDAM, October 25, 2011 /PRNewswire/ -- Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, today provided an update on operations and disclosed the outcome of a strategic review of its gene therapy product pipeline conducted by management and AMT's Board of Directors that enables the company to remain at the forefront of gene therapy development. The CHMP's recent opinion, while not approving Glybera(R), did not identify any...