Adeno-associated viral vector serotype 9-based gene replacement therapy for SURF1-related Leigh syndrome
Qinglan Ling, Matthew Rioux, Yuhui Hu, MinJae Lee, Steven J. Gray
Abstract
-related Leigh syndrome patients.
Topics & Concepts
Adeno-associated virusSerotypeGeneVector (molecular biology)Genetic enhancementLocus (genetics)VirologyViral vectorBiologyGeneticsRecombinant DNAMitochondrial Function and PathologyATP Synthase and ATPases ResearchMetabolism and Genetic Disorders