Congenital Muscular Dystrophy Type 1A (CMD1A) usually presents in the neonatal period with marked muscle weakness and severe hypotonia. CMD1A patients show deficiency in laminin-alpha2 (LAMA2) protein caused by the genetic mutations leading to weaker and unstable muscle tissue. Gene therapy experts at Nationwide Children’s Hospital have developed a gene and protein therapy approach enabling delivery of key domains of LAMA2 using adeno-associated virus (AAV). In addition, our experts have engineered fusion proteins that assist in anchoring LAMA2 to the muscle membrane thereby improving the muscle-matrix interaction and muscle integrity.