RECOMBINANT ADENO-ASSOCIATED VIRUS PRODUCTS AND METHODS FOR TREATING DYSTROGLYCANOPATHIES AND LAMININ-DEFICIENT MUSCULAR DYSTROPHIES

摘要

Products and methods for treating dystroglycanopathies and laminin-deficient muscular dystrophies are provided. In the methods, a protein including a linker domain, such as the heparin-binding domain of Heparin-Binding Epidermal Growth Factor-Like Growth Factor (HBEGF), is delivered to patients.