Onasemnogene abeparvovec (OAV101 or Zolgensma®) is a gene replacement therapy based on a self-complementary adeno-associated virus serotype 9 (AAV9) vector that carries a functional copy of the humanSMN1gene.90This gene replacement therapy aims to provide continuous expression of SMN protein in the body, effectively addressing the underlying cause of SMA-5q. The gene discussed is SMN1; the disease is proximal spinal muscular atrophy.