过去一年中添加的文章，按日期排序
Challenges and opportunities in spinal muscular atrophy therapeutics
112 天前 - … , gene replacement therapy with onasemnogene abeparvovec, and the small-molecule
splicing modifier … in infants and children with spinal muscular atrophy. Given the lifelong …
splicing modifier … in infants and children with spinal muscular atrophy. Given the lifelong …
Gene replacement therapy in spinal muscular atrophy: filling the data gaps
T Hagenacker, U Schara-Schmidt - The Lancet Regional Health …, 2024 - thelancet.com
143 天前 - … The genetically-based therapy of spinal muscular atrophy (… development of therapies
for hereditary neuromuscular … splicing modifiers nusinersen and risdiplam and the gene …
for hereditary neuromuscular … splicing modifiers nusinersen and risdiplam and the gene …
To examine the role of a nuclear import protein karyopherin-β2 in FUS-mediated amyotrophic lateral sclerosis pathogenesis
CP Zammerilla - 2023 - d-scholarship.pitt.edu
264 天前 - … that KAPβ2 is a modifier of FUS-induced ALS in Drosophila neurons. As KAPβ2 is
a modifier of FUS-mediated ALS KAPβ2 could be used as a possible therapeutic for ALS. The …
a modifier of FUS-mediated ALS KAPβ2 could be used as a possible therapeutic for ALS. The …
Is it time for genetic modifiers to predict prognosis in Duchenne muscular dystrophy?
L Bello, EP Hoffman, E Pegoraro - Nature Reviews Neurology, 2023 - nature.com
336 天前 - … genetic modifier studies in DMD to date and discusses the effect of genetic modifiers
on … (inclusion of genotype-stratified subgroup analyses) and therapeutic approaches. The …
on … (inclusion of genotype-stratified subgroup analyses) and therapeutic approaches. The …