News

Nature17y
Identification of seven novel cryptic exons embedded in the dystrophin gene and characterization of 14 cryptic dystrophin exons
The dystrophin gene, which is mutated in Duchenne and Becker muscular dystrophy, is characterized by its extremely large introns. Seven cryptic exons from the intronic sequences of the dystrophin ...
Monthly Prescribing Reference3y
Gene Therapy Gets Priority Review for Cerebral Adrenoleukodystrophy
Credit: Getty Images. Elivaldogene autotemcel (eli-cel) is a one-time gene therapy designed to add functional copies of the ABCD1 gene into a patient’s own ...
Business Wire3y
SwanBio Therapeutics Announces New Preclinical Data Demonstrating Proof of Principle for SBT101 Gene therapy as a Potential Treatment of Adrenomyeloneuropathy
PHILADELPHIA--(BUSINESS WIRE)--SwanBio Therapeutics, a gene therapy company focused on developing treatments for patients with genetically defined neurological conditions, today announced new ...
Hosted on MSN7mon
Boys Treated With Gene Therapy for Rare Brain Disease Doing Well 6 Years Later
THURSDAY, Oct.10, 2024 (HealthDay News) -- Most boys treated with breakthrough gene therapy for a rare but deadly brain illness are faring well six years later, two new reports find.
Nature23y
Computational identification of promoters and first exons in the human genome
Although programs for delineating the internal coding exons of a gene (including Genscan 4, FGENES 5 and MZEF 6) have reached a high degree of sophistication and accuracy, with a sensitivity and ...
Business Wire3y
FDA Grants Fast Track Designation to SBT101, the First Investigational AAV-Based Gene Therapy for Patients With Adrenomyeloneuropathy (AMN)
PHILADELPHIA--(BUSINESS WIRE)--SwanBio Therapeutics, a gene therapy company advancing AAV-based therapies for the treatment of devastating, genetically defined neurological conditions, today ...
MedPage Today6mon
FDA Investigating Blood Cancer Risk With Gene Therapy Skysona
Share on Facebook. Opens in a new tab or window Share on Bluesky. Opens in a new tab or window Share on X. Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window The FDA said ...
Reuters2y
U.S. FDA approves bluebird bio's gene therapy for a rare neurological disorder
Sept 16 - The U.S. Food and Drug Administration (FDA) has approved bluebird bio's (BLUE.O), opens new tab gene therapy for the treatment of a rare neurological disorder, the company said late on ...