title: Treatment of GM2 gangliosidosis: past experiences, implications, and future prospects.
authors.affiliation: Department of Human Genetics, NYS Institute for Basic Research in Developmental Disabilities, Staten Island, New York, USA.
authors.forename: M C
authors.initials: MC
authors.lastname: Rattazzi
authors.forename: K
authors.initials: K
authors.lastname: Dobrenis
mesh_terms: D000818:Animals; D016026:Bone Marrow Transplantation / Q000379:methods; D001921:Brain / Q000378:metabolism; D002415:Cats; D004195:Disease Models, Animal; D020143:Gangliosidoses, GM2 / Q000235:genetics* / Q000628:therapy*; D015316:Genetic Therapy / Q000379:methods; D006801:Humans; D009474:Neurons / Q000378:metabolism; D001619:beta-N-Acetylhexosaminidases / Q000235:genetics
chemical_list: D001619:beta-N-Acetylhexosaminidases