Connecting You to Tomorrow's Biotechnology Innovations
An advisory board at the Food and Drug Administration today endorsed the first gene therapy for an inherited disorder — a rare condition that causes a progressive form of blindness that usually starts in childhood. The recommendation came in a unanimous 16-0 vote after a day full of hearings that included emotional testimonials by doctors, […]
Recent data presented at the Society for Investigative Dermatology (SID) conference demonstrated that EB-101, a gene therapy provided significant wound healing in patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB), a severe form of epidermolysis bullosa (EB). RDEB is a subtype of an inherited genetic skin disorder characterized by chronic skin blistering, open and painful wounds, […]
Using targeted immunotherapy, doctors have succeeded in curing a type of autoimmune enteritis caused by a recently discovered genetic mutation. This report comes from researchers at the Department of Biomedicine of the University of Basel and University Hospital Basel. Their results raise new possibilities for the management of diarrhea, which is often a side effect […]
Experiments designed to reveal how a protein protects the lungs from asthma-related damage suggest a new way to treat a rare disease marked by the inability of cells to break down fats, according to a report in EBioMedicine published online Oct. 25. The study results address Gaucher’s disease, which is caused by a genetic glitch […]
A team of physicians and laboratory scientists has taken a key step toward a cure for sickle cell disease, using CRISPR-Cas9 gene editing to fix the mutated gene responsible for the disease in stem cells from the blood of affected patients. For the first time, they have corrected the mutation in a proportion of stem […]
A team of scientists at The University of Texas Health Science Center at Houston (UTHealth) and Baylor College of Medicine, led by Eric Boerwinkle, Ph.D., Richard Gibbs, Ph.D., and Bing Yu, Ph.D., have identified powerful predictors of congestive heart failure, a major cause of hospitalization and death in the United States. The discovery, published today […]
Scientists have found a variation of the miR-182 gene in patients with primary open-angle glaucoma that results in this overexpression, said Dr. Yutao Liu, vision scientist and human geneticist in the Department of Cellular Biology and Anatomy at the Medical College of Georgia at Augusta University. Its impact appears to reduce the ability of the […]
A major milestone was reached when nusinersen, an investigational treatment for spinal muscular atrophy (SMA), was shown to significantly improve achievement of motor milestones in babies with infantile-onset SMA, according to an interim analysis of the double-blind, randomized, placebo controlled Phase 3 clinical trial called ENDEAR. Babies born with SMA, a genetic disorder affecting nerves […]