title: Candidate disorders for gene therapy: newborn screening facilitates ascertainment of presymptomatic individuals.
authors.forename: Kirit R
authors.initials: KR
authors.lastname: Pindolia
authors.forename: Barry
authors.initials: B
authors.lastname: Wolf
mesh_terms: D030342:Genetic Diseases, Inborn / Q000175:diagnosis* / Q000628:therapy; D005820:Genetic Testing; D015316:Genetic Therapy; D006801:Humans; D007231:Infant, Newborn; D015997:Neonatal Screening