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Published on BioPortfolio: 2018-04-18T09:14:11-0400
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.
This is a long-term follow up evaluating the safety and efficacy of AAV2-REP1 used in antecedent choroideremia studies.
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...
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 ...
This clinical study evaluates the safety and tolerability of AAV2-hCHM in subjects with Choroideremia gene mutations. Two dose groups will be evaluated.
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...
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.
To report the final results of a phase 2 high-dose gene therapy clinical trial in choroideremia.
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...
An X chromosome-linked abnormality characterized by atrophy of the choroid and degeneration of the retinal pigment epithelium causing night blindness.