Mechanism of action
AAV9 viral vector carrying SMN1 transgene crosses blood-brain barrier and transduces motor neurons, producing functional survival motor neuron (SMN) protein to restore motor neuron survival and function.
Medical Articles
One-time intravenous gene therapy for spinal muscular atrophy (SMA). Delivers functional SMN1 gene to motor neurons to address genetic root cause of disease.
© 2025[current_year] The Doctors Platform. All Right Reserved.
© 2025 The Doctors Platform. All Right Reserved.