Alternative experimental gene therapy restores hearing, boosts vision in tests
Harvard Medical School researchers have taken another decisive step in their efforts to develop a gene therapy for people ...
Harvard Medical School7h
Second Gene Therapy Shows Promise for Syndrome Involving Blindness, Deafness
Harvard Medical School researchers have taken another decisive step in their efforts to develop a gene therapy for people ...
Science Daily18h
New discovery enables gene therapy for muscular dystrophies, other disorders
StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.
Discovery enables effective use of gene therapy for muscular dystrophies and other large-gene diseases
Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is ...
FierceBiotech1d
Muscular dystrophy gene therapy uses cells to stitch together mRNA strands in mice
Gene therapy for some diseases, including Duchenne muscular dystrophy (DMD), can be tricky because the needed gene is often ...
BioPharma Dive23h
PTC wins US approval of gene therapy for fatal enzyme disorder
Kebilidi is the first approved gene therapy that can be directly administered to the brain. Its OK secures a priority review ...
GEN15h
Gene Therapy for Muscular Dystrophy Could Use StitchR to Assemble XL Gene from S Segments
StitchR makes it possible to deliver larger payloads with current vectors, expanding the list of diseases targetable by gene therapies.
ClearPoint Neuro Announces FDA De Novo Marketing Authorization of SmartFlow Cannula for Direct Delivery of Gene Therapy to the Brain
Modifying Treatment for AADC Deficiency in the United States SOLANA BEACH, CA / ACCESSWIRE / November 13, 2024 / ...
FiercePharma1d
PTC wins FDA approval for first brain-delivered gene therapy Kebilidi
The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy ...
EurekAlert!2d
Mass General Brigham Gene and Cell Therapy Institute names Spark Grant recipients
The Mass General Brigham Gene and Cell Therapy Institute, a hub of innovation dedicated to accelerating groundbreaking research, conducting clinical trials and developing FDA-approved treatments, ...
New Atlas6d
RNA therapy an effective alternative to lifelong, painful eye injections
An RNA-editing gene therapy has been developed that switches off the key driver of common eye conditions affecting diabetics ...
News Medical on MSN18h
StitchR technology delivers large genes for muscular dystrophy treatment
Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is a big problem: size.