What Are Some Examples of Neonatal Disorders That Could Benefit from Gene Therapy?
Several neonatal disorders could potentially benefit from gene therapy, including:
- Spinal Muscular Atrophy (SMA): Gene therapy has shown promise in treating SMA by delivering a functional copy of the SMN gene. - Cystic Fibrosis: Efforts are underway to correct the defective CFTR gene responsible for cystic fibrosis. - Severe Combined Immunodeficiency (SCID): Gene therapy can help restore immune function in infants born with SCID.
