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 [19 Associated Clinical Trials listed on BioPortfolio]

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.

"Natural History" Study of Choroideremia

The purpose of this study is to understand the rate of progression of all stages of choroideremia using a variety of assessments performed in the clinic including visual field measures, sp...

Natural History of the Progression of Choroideremia Study

Choroideremia (CHM) is a rare, X-linked recessive, degenerative disease of the retina which begins in childhood with loss of night vision and gradually progresses to legal blindness by the...

PubMed Articles [21 Associated PubMed Articles listed on BioPortfolio]


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 ...

REP1 Modulates Autophagy and Macropinocytosis to Enhance Cancer Cell Survival.

Rab escort protein 1 (REP1), a component of the Rab geranyl-geranyltransferase 2 complex, plays a role in Rab protein recruitment in proper vesicles during vesicle trafficking. In addition to having w...

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...

SCLERAL PITS IN CHOROIDEREMIA: Implications for Retinal Gene Therapy.

We report a novel finding on spectral domain optical coherence tomography in patients with choroideremia, which we describe as scleral pits (SCPs).

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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