Treatment of GM2 gangliosidosis: past experiences, implications, and future prospects.
Rattazzi
M C
MC
Department of Human Genetics, NYS Institute for Basic Research in Developmental Disabilities, Staten Island, New York, USA.
Dobrenis
K
K
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
D001619:beta-N-Acetylhexosaminidases