AAV-mediated gene therapy in central nervous system of dystrophin-Dp71 deficient mouse


The understanding of dystrophin-dependent physiological mechanisms responsible for DMD led to the development of innovative tools for gene-therapy strategies based on splice-switching correction or replacement strategies. However, much less is known on their potency to alleviate the retina and brain deficiencies associated with DMD. Molecular tools specifically targeting the Dp71 protein responsible for altered glial physiology and intellectual disability have long been lacking. In both brain and retina Dp71 is mainly expressed in macroglial cells, which poses the challenge to engineer specific properties for viral vectors to selectively target these cells, penetrate CNS barriers and deeply spread in neural tissues.


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