Efficacy and Safety of AAV2-REP1 for the Treatment of Choroideremia

2018-04-18 09:14:11 | BioPortfolio

Published on BioPortfolio: 2018-04-18T09:14:11-0400

Clinical Trials [24 Associated Clinical Trials listed on BioPortfolio]

A Safety Study of Retinal Gene Therapy for Choroideremia

This is a multi-centre, open-label, prospective, two-period, interventional safety study of bilateral use of AAV2-REP1 in adult male subjects with genetically confirmed CHM.

Long-term Safety and Efficacy Follow-up of AAV2-REP1 for the Treatment of Choroideremia (SOLSTICE)

This is a long-term follow up evaluating the safety and efficacy of AAV2-REP1 used in antecedent choroideremia studies.

THOR - Tübingen Choroideremia Gene Therapy Trial

An open label monocentric phase II trial in adult males with a clinical phenotype of choroideremia and a confirmed molecular diagnosis of a null mutation in the gene encoding REP1 to asses...

Choroideremia Gene Therapy Clinical Trial

Phase II gene therapy study, involving a total of 6 male patients with choroideremia. The study will be conducted at the Bascom Palmer Eye Institute, University of Miami. Patients will be ...

Safety and Dose Escalation Study of AAV2-hCHM in Subjects With CHM (Choroideremia) Gene Mutations

This clinical study evaluates the safety and tolerability of AAV2-hCHM in subjects with Choroideremia gene mutations. Two dose groups will be evaluated.

PubMed Articles [18 Associated PubMed Articles listed on BioPortfolio]

Two-Year Results after AAV2-Mediated Gene Therapy for Choroideremia: The Alberta Experience.

To assess the safety of a recombinant adeno-associated viral vector expressing REP1 (rAAV2.REP1) in choroideremia subjects.


Choroideremia (CHM) is a rare inherited retinal degeneration resulting from mutation of the CHM gene, which results in absence of functional Rab escort protein 1 (REP1). We evaluated retinal gene ther...

Crystals and Fatty Acid Abnormalities Are Not Present in Circulating Cells From Choroideremia Patients.

To confirm whether choroideremia (CHM) is a systemic disease characterized by blood lipid abnormalities and crystals found in, or associated with, circulating peripheral blood cells of patients.

Choroideremia Gene Therapy Phase 2 Clinical Trial: 24-Month Results.

To report the final results of a phase 2 high-dose gene therapy clinical trial in choroideremia.

Validation and Safety of visual restoration by ectopic expression of human melanopsin in retinal ganglion cells.

To study whether ectopic human melanopsin (hMel) in retinal ganglion cells (RGCs) could restore the visual function in end-stage retinal degeneration, AAV2/8-CMV-hMel/FYP was injected into the intravi...

Medical and Biotech [MESH] Definitions

An X chromosome-linked abnormality characterized by atrophy of the choroid and degeneration of the retinal pigment epithelium causing night blindness.

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