AAV vectors, the future workhorse of human gene therapy.
Samulski
R J
RJ
Gene Therapy Center, University of North Carolina, CB#7352, Chapel Hill, NC 27599-7352, USA. rjs@med.unc.edu
D000818:Animals; D018360:Crystallography, X-Ray; D000229:Dependovirus / Q000737:chemistry / Q000235:genetics*; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D002836:Hemophilia B / Q000628:therapy*; D006801:Humans; D008954:Models, Biological; D009132:Muscles / Q000378:metabolism