Large-volume intrathecal enzyme delivery increases survival of a mouse model of late infantile neuronal ceroid lipofuscinosis

Molecular Therapy

Volumn 19, Issue 10, 2011, Pages 1842-1848

  Xu, Su ^a,b   Wang, Lingling ^b   El Banna, Mukarram ^a   Sohar, Istvan ^a   Sleat, David E ^a,b   Lobel, Peter ^a,b  

^a RUTGERS UNIVERSITY   (United States)

^b RUTGERS ROBERT WOOD JOHNSON MEDICAL SCHOOL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

RECOMBINANT PROTEIN; TRIPEPTIDYL PEPTIDASE I; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CEREBROSPINAL FLUID; CONTROLLED STUDY; DRUG DISTRIBUTION; DRUG EFFICACY; DRUG MEGADOSE; GENE THERAPY; LATE INFANTILE NEURONAL CEROID LIPOFUSCINOSIS; LIFESPAN; MOUSE; NEURONAL CEROID LIPOFUSCINOSIS; NONHUMAN; PHENOTYPE; SURVIVAL; TREATMENT OUTCOME;

MUS;

EID: 80053568718     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2011.130     Document Type: Article

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