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