Investigators headed by a team at Brigham and Women’s Hospital have developed an adeno-associated virus (AAV) vector that studies show can far more efficiently cross the blood-brain barrier (BBB), in ...
Researchers have developed an engineered adeno-associated virus (AAV) vector that yields high transduction of brain vascular pericytes and smooth muscle cells. The study describing the ...
PITTSBURGH--(BUSINESS WIRE)--Avista Therapeutics, which recently launched as a spinout of leading health system UPMC, aims to develop innovative gene therapies for rare ophthalmic conditions. The new ...
HYOGO, Japan--(BUSINESS WIRE)--JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”) announced today that the Company presented preclinical data from its novel adeno-associated virus (AAV) gene therapy ...
In a recent review published in Signal Transduction and Targeted Therapy, researchers presented recombinant adeno-associated virus (rAAV)-based genetic applications to treat human diseases. Study: ...
Non-human primate study results demonstrate improvements in skeletal and cardiac muscle delivery efficacy, safety potential, human translatability, and manufacturability over prior AAV capsids ...
The first vector-delivered gene therapies to receive FDA approval for clinical use were Luxturna,1 Spark Therapeutics’ vision loss cure, and Zolgensma,2 Novartis’ spinal muscular atrophy treatment.
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