Generation of heterozygous and homozygous NF1 lines from human-induced pluripotent stem cells using CRISPR/Cas9 to investigate bone defects associated with neurofibromatosis type 1
Dystrophin deficiency impairs cell junction formation during embryonic myogenesis from pluripotent stem cells
Molecular Analysis of a Congenital Myasthenic Syndrome Due to a Pathogenic Variant Affecting the C-Terminus of ColQ
Nucleolar reorganization after cellular stress is orchestrated by SMN shuttling between nuclear compartments
Generation of three induced pluripotent stem cell lines from patients with glycogen storage disease type III.
Unlocking the Complexity of Neuromuscular Diseases : Insights from Human Pluripotent Stem Cell-Derived Neuromuscular Junctions
