Related references
Note: Only part of the references are listed.Reversing a model of Parkinson's disease with in situ converted nigral neurons
Hao Qian et al.
NATURE (2020)
Targeting Huntingtin Expression in Patients with Huntington's Disease
Sarah J. Tabrizi et al.
NEW ENGLAND JOURNAL OF MEDICINE (2019)
Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy
R. S. Finkel et al.
NEW ENGLAND JOURNAL OF MEDICINE (2017)
Antisense masking of an hnRNP A1/A2 intronic splicing silencer corrects SMN2 splicing in Transgenic mice
Yimin Hua et al.
AMERICAN JOURNAL OF HUMAN GENETICS (2008)