脊髓性肌萎缩症的基因治疗:优化 SMN1 递送以纠正所有神经和系统紊乱的可能性观点
Gene therapy for spinal muscular
atrophy: perspectives on the
possibility of optimizing SMN1
delivery to correct all neurological
and systemic perturbations
Sharon J. Brown, Rafael J. Yáñez-Muñoz, Heidi R. Fuller
中国神经再生研究(英文版)
.
2025, (7): 2011
-2012
.
DOI: 10.4103/NRR.NRR-D-24-00504