Ascl1 Gene Augmentation Protects Mouse Photoreceptors From Blue Light Damage
Delivering the transcription factor Ascl1 using an adeno-associated virus reduced photoreceptor degeneration and preserved retinal function in a mouse model ...
Tag: AAV vectors
Rubidium chloride ultracentrifugation resolves and quantifies dual-AAV gene therapy vectors
A density gradient equilibrium analytical ultracentrifugation method using rubidium chloride achieved high-resolution separation and accurate quantification of the two highly ...
Gene Therapy for Duchenne Muscular Dystrophy Fails Key Trials, Review Finds
A systematic review and GRADE appraisal found that both phase 3 AAV-based shortened dystrophin gene therapy programs failed their primary ...
Platelet-guided gene therapy reprograms heart metabolism to promote repair in male mice
A platelet-engineered AAV system that silences the fatty acid transporter CD36 reshaped cardiac lipid metabolism and improved repair after ischemia-reperfusion ...
AAV3B antibodies affect eligibility for liver-targeted gene therapy
A study found anti-AAV3B neutralizing antibodies in 33%–36% of healthy adults, with intermediate titers in 19% of positive individuals, while ...
Gene Therapy Restores Hearing in Congenital Deafness Trial Milestone
A new review charts the rapid rise of AAV-mediated OTOF gene replacement therapy, from the first human trials to the ...
Gene Therapy Slows ALS Onset in Mice Models
In a groundbreaking study, researchers have unveiled a potential breakthrough in the treatment of Amyotrophic Lateral Sclerosis (ALS) through innovative ...
