Retinal gene therapy in patients with choroideremia: Initial fi ndings from a phase 1/2 clinical trial
Background Choroideremia is an X-linked recessive disease that leads to blindness due to mutations in the CHM gene, which encodes the Rab escort protein 1 (REP1). We assessed the eff ects of retinal gene therapy with an adenoassociated viral (AAV) vector encoding REP1 (AAV.REP1) in patients with thi...
| Hlavní autoři: | , , , , , , , , , , , , , |
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| Médium: | Journal article |
| Vydáno: |
2014
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