Diseases that affect the retina, the light-sensitive layer at the back of the eye, are a significant cause of visual impairment and blindness. Gene therapy holds promise for treating some of these ...
In gene therapy, a gene (a piece of DNA) is introduced into cells. This process uses a virus that has been stripped of its disease-causing properties—referred to as a viral vector—as the delivery ...
A study assessing the real-world commercial roll-out of gene therapies for sickle cell disease and beta thalassemia offers ...
University of Iowa researchers developed a gene therapy that could prevent craniosynostosis, a condition where babies’ skull bones fuse too early, potentially eliminating the need for invasive skull ...
Alison Clare has received funding from the Wellcome Trust and the Medical Research Council. Her position is currently funded by National Institute for Health and Care Research (NIHR) Biomedical ...
TriLink BioTechnologies senior scientist Dr Cody Palumbo discusses the blockers to manufacturing mRNA-based gene editing ...
CT Insider on MSN
CT joins national push to open access to million-dollar sickle cell gene therapies through Medicaid
About 100,000 people in the U.S. have sickle cell disease, a condition that primarily affects people of color, the CDC says.
-- Muscular Dystrophy Association Calls FDA Approval of Novartis' Itvisma (onasemnogene abeparvovec-brve) a Major Step Forward for the Spinal Muscular Atrophy Community Mary Fiance, National Vice ...
Vence L. Bonham Jr., Acting Deputy Director at the National Human Genome Research Institute (NHGRI) and an investigator in the National Human Genome Research Institute’s (NHGRI) Division of Intramural ...
After more than 30 years, gene therapy trailblazer James Wilson M.D., Ph.D., is leaving the University of Pennsylvania. He will be spearheading two new companies meant to translate the scientific ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results
Feedback