scAAv9/JeT-GAN

GENETIC2 trials

Sponsors

National Institute of Neurological Disorders and Stroke (NINDS), University of Texas Southwestern Medical Center

Conditions

Gene TransferGiant Axonal NeuropathyGiant Axonal Neuropathy (GAN)

Phase 1

Intrathecal Administration of scAAV9/JeT-GAN for the Treatment of Giant Axonal Neuropathy

CompletedNCT02362438

National Institute of Neurological Disorders and Stroke (NINDS)Gene Transfer, Giant Axonal Neuropathy

Start: 2015-04-24End: 2026-04-10Updated: 2026-08-27

Intraneural Administration of scAAV9/JeT-GAN Into the Vagus Nerve for Patients With Giant Axonal Neuropathy (GAN)

RecruitingNCT07543991

University of Texas Southwestern Medical CenterGiant Axonal Neuropathy (GAN)

Start: 2026-03-01End: 2032-06-30Target: 4Updated: 2026-04-22

Related Papers

AAV gene therapy for hereditary spastic paraplegia type 50: a phase 1 trial in a single patient.

2024-06-2819 citations

Intrathecal Gene Therapy for Giant Axonal Neuropathy

New England Journal of Medicine2024-03-2045 citations

Extensive rod and cone photoreceptor-cell degeneration in rat models of giant axonal neuropathy: implications for gene therapy of human disease

Ophthalmic Genetics2021-05-063 citations

Advancing the pathologic phenotype of giant axonal neuropathy: early involvement of the ocular lens

UNC Libraries2019-08-18

Advancing the pathologic phenotype of giant axonal neuropathy: early involvement of the ocular lens.

2019-02-0111 citations

Development of Intrathecal AAV9 Gene Therapy for Giant Axonal Neuropathy.

2018-02-1597 citations