Recombinant adeno associated virus products and methods for treating dystroglycanopathy and laminin deficient muscular dystrophy

摘要

There are products and methods for treating dystroglycananosis and laminin deficient muscular dystrophy.In this method, a protein containing a linker domain such as heparin binding domain of heparin binding epidermal growth factor like growth factor (HBEGF) is delivered to the patient.Methods and products for the treatment of CMD such as distroglycananosis and laminin deficient muscular dystrophy are provided herein.The product includes a ravav encoding therapeutic protein and a therapeutic protein disclosed.