TREATING AUTOSOMAL RECESSIVE BESTROPHINOPATHIES AND METHODS FOR EVALUATING SAME

摘要

Methods for treating bestrophinopathies are provided herein. The method includes, administering to an eye of the subject a dose of a recombinant adeno-associated virus (rAAV) vector comprising a nucleic acid sequence encoding a human BEST1 protein, wherein the subject has two mutant BEST1 alleles. Also provided are methods for evaluating treatments for retinal degeneration.