Advertisement

Topics

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...

VASCULAR ALTERATIONS REVEALED WITH OPTICAL COHERENCE TOMOGRAPHY ANGIOGRAPHY IN PATIENTS WITH CHOROIDEREMIA.

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.

More From BioPortfolio on "Efficacy and Safety of AAV2-REP1 for the Treatment of Choroideremia"

Advertisement
Quick Search
Advertisement
Advertisement

 

Searches Linking to this Trial