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 [22 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 [19 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.

SNCA REP1 and Parkinson's disease.

REP1 is a polymorphic dinucleotide repeat sequence located in the promoter region of the SNCA gene (OMIM 163890). Opinions regarding the interaction between the various REP1 alleles and Parkinson's di...


Choroideremia is a rare degenerative retinal disease that causes incurable blindness. It occurs as a result of the deficiency of the X-linked CHM gene, which encodes the Rab escort protein 1 (REP1). G...

Use of induced pluripotent stem cell models to probe the pathogenesis of Choroideremia and to develop a potential treatment.

Choroideremia (CHM) is a rare monogenic, X-linked recessive inherited retinal degeneration resulting from mutations in the Rab Escort Protein-1 (REP1) encoding CHM gene. The primary retinal cell type ...

Precision Fluorescent Labeling of an Adeno-Associated Virus Vector to Monitor the Viral Infection Pathway.

Adeno-associated virus 2 (AAV2) is a common vehicle for the delivery of a variety of therapeutic genes. A better understanding of the process of infection of AAV2 will advance our knowledge of AAV2 bi...

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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