Gene therapy for hemophilia B: AAV-mediated transfer of the gene for coagulation factor IX to human muscle.
Larson
P J
PJ
Department of Pediatrics, University of Pennsylvania School of Medicine, The Children's Hospital of Philadelphia, 19104, USA.
High
K A
KA
D000328:Adult; D000818:Animals; D017321:Clinical Trials, Phase I as Topic; D017322:Clinical Trials, Phase II as Topic; D015331:Cohort Studies; D000229:Dependovirus / Q000235:genetics*; D004283:Dog Diseases / Q000235:genetics / Q000628:therapy; D004285:Dogs; D005164:Factor IX / Q000096:biosynthesis / Q000235:genetics* / Q000276:immunology; D005500:Follow-Up Studies; D015316:Genetic Therapy; D005822:Genetic Vectors / Q000008:administration & dosage / Q000235:genetics*; D002836:Hemophilia B / Q000235:genetics / Q000628:therapy* / Q000662:veterinary; D006801:Humans; D007273:Injections, Intramuscular; D007518:Isoantibodies / Q000096:biosynthesis; D008297:Male; D051379:Mice; D008810:Mice, Inbred C57BL; D023421:Models, Animal; D018482:Muscle, Skeletal / Q000378:metabolism*; D014162:Transfection
D007518:Isoantibodies; D005164:Factor IX