?Genetically modified animal models of hereditary diseases for testing of gene-directed therapy

Disease-causing genes have been identified for many severe muscular and neurological genetic disorders. Advances in the gene therapy field offer promising solutions drug development to treat these life-threatening conditions. Depending on how mutation affects function of product, different approaches may be beneficial. Gene replacement is appropriate diseases caused by mutations that result deficiency functional protein. suppression strategy suggested disorders toxic product mutant gene. Splicing modulators, genome editing, base editing techniques can applied with types underlying mutations. Testing potential drugs animal models human an indispensable step development. Given specific approach, generated using a variety technologies ranging from transgenesis precise editing. In this review, we discuss used generate small large most common We specifically focus were test therapies based adeno-associated vectors antisense nucleotides.