Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

Gene Therapy Corrects Brain and Behavioral Pathologies in CLN6-Batten Disease

10 Jul 2019

Abstract excerpt

CLN6-Batten disease, a form of neuronal ceroid lipofuscinosis is a rare lysosomal storage disorder presenting with gradual declines in motor, visual, and cognitive abilities and early death by 12-15 years of age. We developed a self-complementary adeno-associated virus serotype 9 (scAAV9) vector expressing the human CLN6 gene under the control of a chicken β-actin (CB) hybrid promoter. Intrathecal delivery of...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to Lysosomal Storage Disorders Research
Share to Neurogenetic and Muscular Disorders Research
Share to Virus-based gene therapy research

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Source

Identifiers

PMID: 31331814
DOI: 10.1016/j.ymthe.2019.06.015

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Gene Therapy Corrects Brain and Behavioral Pathologies in CLN6-Batten DiseasePMID 31331814Read ↗

Neonatal brain-directed gene therapy rescues a mouse model of neurodegenerative CLN6 Batten diseaseSemantic proximity Read ↗

Neonatal brain-directed gene therapy rescues a mouse model of neurodegenerative CLN6 Batten diseaseSemantic proximity Read ↗

AAV9 gene therapy restores lifespan and treats pathological and behavioral abnormalities in a mouse model of CLN8-Batten diseaseSemantic proximity Read ↗

AAV9 Gene Therapy Increases Lifespan and Treats Pathological and Behavioral Abnormalities in a Mouse Model of CLN8-Batten DiseaseSemantic proximity Read ↗

Longitudinal In Vivo Monitoring of the CNS Demonstrates the Efficacy of Gene Therapy in a Sheep Model of CLN5 Batten DiseaseSemantic proximity Read ↗

Long-term safety and dose escalation of intracerebroventricular CLN5 gene therapy in sheep supports clinical translation for CLN5 Batten diseaseSemantic proximity Read ↗

Characterisation of early changes in ovine CLN5 and CLN6 Batten disease neural cultures for the rapid screening of therapeutics.Semantic proximity Read ↗

A murine model of variant late infantile ceroid lipofuscinosis recapitulates behavioral and pathological phenotypes of human disease.Semantic proximity Read ↗

Characterization of neuropathology in ovine CLN5 and CLN6 neuronal ceroid lipofuscinoses (Batten disease).Semantic proximity Read ↗

Current Insights in Elucidation of Possible Molecular Mechanisms of the Juvenile Form of Batten Disease.Semantic proximity Read ↗

Reduction of Neuroinflammation and Seizures in a Mouse Model of CLN1 Batten Disease using the Small Molecule Enzyme Mimetic, N-Tert-Butyl HydroxylamineSemantic proximity Read ↗

Batten disease: clinical aspects, molecular mechanisms, translational science, and future directions.Semantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.