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Article

Self-Complementary AAV9 Gene Delivery Partially Corrects Pathology Associated with Juvenile Neuronal Ceroid Lipofuscinosis (CLN3).

The Journal of neuroscience : the official journal of the Society for Neuroscience - 14 Sept 2016

Bosch Megan E, Aldrich Amy, Fallet Rachel, Odvody Jessica, Burkovetskaya Maria, Schuberth Kaitlyn, Fitzgerald Julie A, Foust Kevin D, Kielian Tammy

Abstract excerpt

UNLABELLED: Juvenile neuronal ceroid lipofuscinosis (JNCL) is a fatal lysosomal storage disease caused by autosomal-recessive mutations in CLN3 for which no treatment exists. Symptoms appear between 5 and 10 years of age, beginning with blindness and seizures, followed by progressive cognitive and motor decline and premature death (late teens to 20s). We explored a gene delivery approach for JNCL by generating...

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Source

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Identifiers

PMID: 27629717
DOI: 10.1523/JNEUROSCI.1635-16.2016
PMCID: PMC6601943

Genes

CLN3
GFAP

Provenance

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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Self-Complementary AAV9 Gene Delivery Partially Corrects Pathology Associated with Juvenile Neuronal Ceroid Lipofuscinosis (CLN3).PMID 27629717Read ↗

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