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Article

Enhanced survival of the LINCL mouse following CLN2 gene transfer using the rh.10 rhesus macaque-derived adeno-associated virus vector.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Mar 2007

Sondhi Dolan, Hackett Neil R, Peterson Daniel A, Stratton Jamie, Baad Michael, Travis Kelly M, Wilson James M, Crystal Ronald G

Abstract excerpt

Late infantile neuronal ceroid lipofuscinosis (LINCL) is a lysosomal storage disorder caused by mutations in the CLN2 gene and a deficiency of tripeptidyl peptidase I (TPP-I). Prior studies with adeno-associated virus (AAV) serotype 2 or 5 mediated transfer of the CLN2 complementary DNA to the central nervous system (CNS) of CLN2(-/-) mice cleared CNS storage granules, but provided no improvement in the phenotype...

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Identifiers

PMID: 17180118
DOI: 10.1038/sj.mt.6300049

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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