Gene therapy for rare diseases.
D002986:Clinical Trials as Topic / Q000191:economics / Q000331:legislation & jurisprudence; D019992:Databases as Topic; D000229:Dependovirus / Q000235:genetics; D015316:Genetic Therapy / Q000191:economics / Q000639:trends*; D005822:Genetic Vectors / Q000009:adverse effects*; D033161:Government Regulation; D006801:Humans; D009316:National Institutes of Health (U.S.); D011367:Professional Staff Committees; D014481:United States; D014486:United States Food and Drug Administration