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 Targeting the Inner Retina Rescues the Retinal Phenotype in a Mouse Model of CLN3 Batten Disease.

Human gene therapy - 1 Jul 2020

Kleine Holthaus Sophia-Martha, Aristorena Mikel, Maswood Ryea, Semenyuk Olha, Hoke Justin, Hare Aura, Smith Alexander J, Mole Sara E, Ali Robin R

Abstract excerpt

The neuronal ceroid lipofuscinoses (NCLs), often referred to as Batten disease, are inherited lysosomal storage disorders that represent the most common neurodegeneration during childhood. Symptoms include seizures, vision loss, motor and cognitive decline, and premature death. The development of brain-directed treatments for NCLs has made noteworthy progress in recent years. Clinical trials are currently ongoing...

Read the complete abstract on PubMed

Topics

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

Share to Dependovirus
Share to Genetic Therapy

Methodology Data sources Editorial policy Privacy Delete account & data About Contact Helena Bioinformatics

Share to Membrane Glycoproteins

Share to Mice, Inbred C57BL

Share to Molecular Chaperones

Share to Neuronal Ceroid-Lipofuscinoses

Share to Photoreceptor Cells

Share to Retinal Diseases

Source

Open in PubMed Open full text

Identifiers

PMID: 32578444
DOI: 10.1089/hum.2020.038
PMCID: PMC7404834

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence. Graph snapshot: 2026-09-29.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Gene Therapy Targeting the Inner Retina Rescues the Retinal Phenotype in a Mouse Model of CLN3 Batten Disease.PMID 32578444Read ↗

Visual deficits in a mouse model of Batten disease are the result of optic nerve degeneration and loss of dorsal lateral geniculate thalamic neurons.Semantic proximity Read ↗

Retinal pathology and function in a Cln3 knockout mouse model of juvenile Neuronal Ceroid Lipofuscinosis (batten disease).Semantic proximity Read ↗

Neurodegeneration and Epilepsy in a Zebrafish Model of CLN3 Disease (Batten Disease)Semantic proximity Read ↗

Therapeutic antisense oligonucleotide mitigates retinal dysfunction in a pig model of CLN3 Batten diseaseSemantic proximity Read ↗

Histopathologic and immunocytochemical analysis of the retina and ocular tissues in Batten disease.Semantic proximity Read ↗

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

Prevention of Photoreceptor Cell Loss in a Cln6 Mouse Model of Batten Disease Requires CLN6 Gene Transfer to Bipolar CellsSemantic proximity Read ↗

Genetic and Cellular Basis of Impaired Phagocytosis and Photoreceptor Degeneration in CLN3 Disease.Semantic proximity Read ↗

Progress towards understanding the neurobiology of Batten disease or neuronal ceroid lipofuscinosisSemantic proximity Read ↗

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

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

A mouse gene knockout model for juvenile ceroid-lipofuscinosis (Batten disease).Semantic proximity Read ↗

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