Category Archives: Gene Therapy

Ann Arbor-based RetroSense Therapeutics LLC plans to start human trials of a gene-based therapy next year, and if those trials go as animal trials have gone, those blinded by degenerative eye diseases will regain some of their vision. What seemed more like an interesting science experiment four years ago than a real company -- can a light-sensing gene that helps pond scum find sunlight also help the blind see? -- is attracting interest and venture capital as it gears up for human trials. The therapy is based on the photosensitivity of a gene called channelrhodopsin-2. This gene allows blue-green algae to detect where the sun is shining on a pond so they can move in its direction and convert light to energy through photosynthesis. When the gene, which is inside a cultured medium called a vector, is injected into the eye, previously non-photosensitive retinal cells are converted into photosensitive cells, allowing limited vision. RetroSense and its research partners have tested mice, rats and nonhuman primates, with all species showing a return of some vision following treatment, said founder and CEO Sean Ainsworth. As a result, the company has just completed raising a venture capital round of $2.4 million, led by … Continue reading →

Contact Information Available for logged-in reporters only Newswise LA JOLLA, CA June 26, 2014 Scientists working to make gene therapy a reality have solved a major hurdle: how to bypass a blood stem cells natural defenses and efficiently insert disease-fighting genes into the cells genome. In a new study led by Associate Professor Bruce Torbett at The Scripps Research Institute (TSRI), a team of researchers report that the drug rapamycin, which is commonly used to slow cancer growth and prevent organ rejection, enables delivery of a therapeutic dose of genes to blood stem cells while preserving stem cell function. These findings, published recently online ahead of print by the journal Blood, could lead to more effective and affordable long-term treatments for blood cell disorders in which mutations in the DNA cause abnormal cell functions, such as in leukemia and sickle cell anemia. Improving Gene Delivery to Blood Stem Cells Viruses infect the body by inserting their own genetic material into human cells. In gene therapy, however, scientists have developed gutted viruses, such as the human immunodeficiency virus (HIV), to produce what are called viral vectors. Viral vectors carry therapeutic genes into cells without causing viral disease. Torbett and other scientists … Continue reading →

A level. R.7. Cystic fibrosis and gene therapy (Ms Cooper) What is cystic fibrosis? What systems in the body are affected? What are the symptoms? What is the genotype of someone with cystic fibrosis? What are the dif... By: Ms Cooper's Cambridge A level Biology … Continue reading →

Gene therapy for rare seeing impairment disorder shows promise for wider use, doctors say ,mesotheli http://www.moreaboutmesothelioma.com/ "about mesothelioma "asbestos class action "asbestosis mesothelioma "average mesothelioma settlement "best mesothelioma attorney "best mesothelioma law... By: aamer ali … Continue reading →

Gene Therapy for Beta Thalassemia By: bluebird bio … Continue reading →

Isis Pharmaceuticals Inc. (ISIS), a maker of gene therapy treatments, may seek to expand use of an experimental drug targeting triglycerides to fight heart disease, a move an analyst says could add $2 billion in sales. The drug targets the APOC3 gene and is now aimed solely at a rare disease thats linked to diabetes and pancreatitis and affects about 5,000 people. Two studies this week, however, have also tied the gene to lower risk of heart disease, an illness that kills 600,000 people yearly, a much larger target. The research, done independently of Isis, was highlighted in the New England Journal of Medicine. It may open a new window of opportunity for Isis once the drug, called ISIS-APOCIIIRx and heading to final testing, is approved and may spark interest from potential acquirers, analysts said. Were weighing how we want to take full advantage of this breakthrough, and as we do that we have to consider the potential broader development of the follow on product, Isis Chief Executive Officer Stanley Crooke said by telephone. We have a great deal of licensing interest for this drug. In the New England Journal of Medicine studies, mutations in the APOC3 gene decreased the … Continue reading →

New Gene Therapy Rapidly Helps Patients With Rare Blood Disorder Two patients who were given Bluebird Bio's experimental gene therapy for the rare blood disorder beta-thalassemia were able to stop receiving blood transfusi... By: WochitGeneralNews … Continue reading →

Gene Therapy - GMAT Free - GFSC120 This Sentence Correction question is part of GMAT Free, the completely free online GMAT course brought to you by the world's experts. For more information, s... By: GMAT Free … Continue reading →

Gene Therapy Partnering Terms and Agreements Market Research Reports, Inc. has announced the addition of "Gene Therapy Partnering Terms and Agreements" research report to their offering. See more at- http://mrr.cm/ZMF. By: Market Research Reports, Inc. … Continue reading →

MILAN (TheStreet) -- Two patients with a serious, inherited blood disorder have been able to stop blood transfusions following a single treatment with an experimental gene therapy developed by Bluebird Bio (BLUE), according to new but preliminary data presented Saturday. The two patients reported on today have beta-thalassemia (B-thal), a disease caused by a missing or defective gene which prevents oxygen-carrying hemoglobin from functioning properly. The BlueBird therapy, known as LentiGlobin, replaces the defective gene with one that is fully functional. Beta-thalassemia patients suffer from chronic anemia and typically require regular and lifelong blood transfusions. Following a single infusion of Bluebird's LentiGlobin gene therapy, the two B-thal patients started producing functional hemoglobin and within 10 and 12 days, respectively, were able to halt blood transfusions. The patients have now been blood-transfusion independent for approximately 6 and 3 months, respectively, according to Bluebird Chief Medical Officer David Davidson. The data are being presented Saturday at the European Hematology Association annual meeting. "Following the [gene] transplant, we're seeing near normal levels of hemoglobin," said Davidson. "These early results far exceeded our expectations." Bluebird only has limited data on two patients and the study, conducted in France, continues with seven B-thal patients expected … Continue reading →