This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. A recombinant adeno-associated virus serotype 2 rAAV2 vector has been altered to carry the human RPE65 hRPE65 gene. This vector has been shown to restore vision in animal models that resemble human RPE65-associated Leber congenital amaurosis LCA, an incurable retinal degeneration that causes severe vision loss. The proposed study is an open label, Phase I clinical trial of subretinal rAAV2-CBSB-hRPE65 administration to individuals with RPE65-associated retinal disease. Two cohorts of three subjects each will be included in this trial. Both cohorts will receive the same dose of rAAV2-CBSB-hRPE65 . Cohort 1 will consist of individuals 18 years of age and older and Cohort 2 will consist of individuals between the ages of 8 and 17. Enrollment in Cohort 2 will begin only after confirming the safety of rAAV2-CBSB-hRPE65 administration in the older group of participants. This trial will lead to a greater understanding of the safety and thereby potential value of gene transfer in RPE65-associated retinal disease and will have implications for other forms of retinal degenerative disease amenable to this type of intervention.
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