Sentences with phrase «aav gene transfer»
«This regenerative technology, termed AAV gene transfer, provided long - lasting benefit to the entire musculature of affected dogs that would have otherwise perished, extending a healthy lifespan for more than 4 years,» said Dr. Martin Childers, senior author of the Muscle & Nerve study and a UW Medicine researcher in Seattle.
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Adeno - associated viral (AAV) vectors are the most frequently used gene - transfer tools in the study of the brain and spinal cord, which together are known as the central nervous system (CNS).
Manno, Catherine et al. «AAV - Mediated Factor IX Gene Transfer to Skeletal Muscle in Patients with Severe Hemophilia B.» Blood.
Go Deeper Cholinergic Mesopontine Signals Govern Locomotion and Reward through Dissociable Midbrain Pathways Cre - dependent Selection Yields AAV Variants for Widespread Gene Transfer to the Adult Brain
Additional Caltech authors on the paper, «Cre - dependent selection yields AAV variants for widespread gene transfer to the adult brain,» are Sripriya Ravindra Kumar, Ken Y. Chan, Abhik Banerjee, Wei - Li Wu, and Bin Yang, as well as former Caltech students Piers L. Pravdo and Bryan P. Simpson.
AAV gene therapy vectors are emerging as the gene transfer vehicle with high potential for use in the CNS as they transduce post mitotic cells that mediate the sustained, long term gene expression required in chronic progressive diseases.