Systematic ranking of inborn errors of metabolism as targets for gene therapy.
Hofherr
Sean E
SE
Mayo Clinic College of Medicine, Biochemical Genetics Laboratory, 200 1st St. SW, Rochester, MN 55905, USA. Hofherr.Sean@Mayo.edu
Matern
Dietrich
D
D000818:Animals; D015316:Genetic Therapy; D006801:Humans; D007231:Infant, Newborn; D008661:Metabolism, Inborn Errors / Q000145:classification* / Q000628:therapy*; D015997:Neonatal Screening; D057170:Translational Research, Biomedical