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Choroideremia: molecular mechanisms and development of AAV gene therapy.

Journal article

Patrício MI. et al, (2018), Expert Opin Biol Ther, 18, 807 - 820

Novel non-contiguous exon duplication in choroideremia.

Journal article

Edwards TL. et al, (2018), Clin Genet, 93, 144 - 148

Prenylation of Rab6a as a Potency Assay for Choroideremia Gene Therapy

Journal article

Patricio MI. et al, (2017), MOLECULAR THERAPY, 25, 142 - 143