Mitochondria replacement from transplanted amniotic fluid stem cells:a promising therapy for non-neuronal defects in spinal muscular atrophy
Michela Pozzobon1
Camilla Bean2
1.Women's and Children's Health Department,University of Padova;Foundation Institute of Pediatric Research Città della Speranza,Padova,Italy2.Department of Medicine,University of Udine,Udine,Italy
摘要：Spinal muscular atrophy (SMA) is a genetic disorder that primarily affects infants and leads to muscle weakness, atrophy, and paralysis. The main cause is the homozygous mutation or deletion of the SMN1 gene, resulting in inadequate levels of the survival motor neuron (SMN) protein. Approved treatments focus on restoring SMN levels through various approaches, but there is a need for "SMN-independent" therapies that target other pathological processes.
机标关键词：stemcellsfromamnioticatrophydefectsfluidmitochondria
论文发表日期：2024-05-28
在线出版日期：2025-08-15（本平台首次上网日期，不代表文献的发表时间）
页数：2( 971-972 )