Self-regulating AAV Vectors for Safe Expression of MeCP2 in Rett Syndrome

摘要

In some aspects, the present disclosure relates to compositions and methods for manipulating transgenes. In some embodiments, the present disclosure provides self-regulating recombinant nucleic acids, viral vectors, and pharmaceutical compositions comprising a MeCP2 transgene. In some embodiments, the compositions and methods described by the present disclosure are useful for treating diseases and disorders associated with loss of function mutations, such as Rett syndrome.