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

AAV1.NT-3 gene therapy for X-linked Charcot-Marie-Tooth neuropathy type 1.

Gene therapy - 1 Apr 2022

Ozes Burcak, Myers Morgan, Moss Kyle, Mckinney Jennifer, Ridgley Alicia, Chen Lei, Bai Shasha, Abrams Charles K, Freidin Mona M, Mendell Jerry R, Sahenk Zarife

Abstract excerpt

X-linked Charcot-Marie-Tooth neuropathy (CMTX) is caused by mutations in the gene encoding Gap Junction Protein Beta-1 (GJB1)/Connexin32 (Cx32) in Schwann cells. Neurotrophin-3 (NT-3) is an important autocrine factor supporting Schwann cell survival and differentiation and stimulating axon regeneration and myelination. Improvements in these parameters have been shown previously in a CMT1 model, TremblerJ mouse,...

Read the complete abstract on PubMed

Topics

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

Share to Mice
Share to Axons
Share to Charcot-Marie-Tooth Disease

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Connexins

Share to Genetic Therapy

Share to Mice, Knockout

Share to Nerve Regeneration

Share to Schwann Cells

Source

Open in PubMed Open full text

Identifiers

PMID: 33542455
DOI: 10.1038/s41434-021-00231-3
PMCID: PMC9013664

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.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

AAV1.NT-3 gene therapy for X-linked Charcot-Marie-Tooth neuropathy type 1.PMID 33542455Read ↗

AAV9-mediated Schwann cell-targeted gene therapy rescues a model of demyelinating neuropathySemantic proximity Read ↗

AAV1.NT-3 Gene Therapy for Charcot–Marie–Tooth NeuropathySemantic proximity Read ↗

Intraneural GJB 1 gene delivery improves nerve pathology in a model of X ‐linked C harcot– M arie– T ooth diseaseSemantic proximity Read ↗

Gene replacement therapy after neuropathy onset provides therapeutic benefit in a model of CMT1XSemantic proximity Read ↗

Gene replacement therapy in two Golgi-retained CMT1X mutants before and after the onset of demyelinating neuropathySemantic proximity Read ↗

Two new mouse models of Gjb1-associated Charcot-Marie-Tooth disease type 1X.Semantic proximity Read ↗

Transgenic expression of human connexin32 in myelinating Schwann cells prevents demyelination in connexin32-null mice.Semantic proximity Read ↗

Knock-in mouse models for CMTX1 show a loss of function phenotype in the peripheral nervous system.Semantic proximity Read ↗

Abnormalities in the axonal cytoskeleton induced by a connexin32 mutation in nerve xenografts.Semantic proximity Read ↗

Connexins, gap junctions and peripheral neuropathy.Semantic proximity Read ↗

Intrathecal gene therapy in mouse models expressing CMT1X mutations.Semantic proximity Read ↗

Connexin32-null mice develop demyelinating peripheral neuropathySemantic proximity Read ↗

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