Designing and characterizing hammerhead ribozymes for use in AAV vector-mediated retinal gene therapies.
Fritz
Jason J
JJ
Department of Molecular Genetics and Microbiology, College of Medicine, University of Florida, Gainesville, Florida 32610, USA.
White
D Alan
DA
Lewin
Alfred S
AS
Hauswirth
William W
WW
D001483:Base Sequence; D000229:Dependovirus / Q000235:genetics*; D015316:Genetic Therapy; D005822:Genetic Vectors; D008969:Molecular Sequence Data; D009690:Nucleic Acid Conformation; D012313:RNA / Q000737:chemistry; D016337:RNA, Catalytic / Q000235:genetics* / Q000627:therapeutic use; D012172:Retinaldehyde / Q000378:metabolism*
D016337:RNA, Catalytic; D012313:RNA; D012172:Retinaldehyde