title: Feasibility of gene therapy for late neuronal ceroid lipofuscinosis.
abstract: Late infantile neuronal ceroid lipofuscinosis is a progressive childhood neurodegenerative disorder characterized by intracellular accumulation of autofluorescent material resembling lipofuscin in neuronal cells. This report summarizes the new therapies under consideration for late infantile neuronal ceroid lipofuscinosis, with a focus on strategies for in vivo gene therapy for the retinal and central nervous system manifestations of the disease.
authors.affiliation: Institute of Genetic Medicine and Belfer Gene Therapy Core Facility, Weill Medical College of Cornell University, 515 E 71st St, Suite 1000, New York, NY 10021, USA.
authors.forename: D
authors.initials: D
authors.lastname: Sondhi
authors.forename: N R
authors.initials: NR
authors.lastname: Hackett
authors.forename: R L
authors.initials: RL
authors.lastname: Apblett
authors.forename: S M
authors.initials: SM
authors.lastname: Kaminsky
authors.forename: R G
authors.initials: RG
authors.lastname: Pergolizzi
authors.forename: R G
authors.initials: RG
authors.lastname: Crystal
mesh_terms: D000328:Adult; D000626:Aminopeptidases; D000818:Animals; D002648:Child; D002675:Child, Preschool; D002986:Clinical Trials as Topic; D004152:Dipeptidyl-Peptidases and Tripeptidyl-Peptidases; D010450:Endopeptidases / Q000235:genetics* / Q000627:therapeutic use; D015316:Genetic Therapy; D005822:Genetic Vectors; D006801:Humans; D009472:Neuronal Ceroid-Lipofuscinoses / Q000235:genetics* / Q000628:therapy*; D057057:Serine Proteases; D033581:Stem Cell Transplantation; D000091346:Tripeptidyl-Peptidase 1
chemical_list: D000091346:Tripeptidyl-Peptidase 1; D010450:Endopeptidases; D057057:Serine Proteases; D000626:Aminopeptidases; D004152:Dipeptidyl-Peptidases and Tripeptidyl-Peptidases