Neuromuscular disorders (NMDs) are a wide-ranging group of diseases that seriously affect
the quality of life of affected individuals. The development of next-generation sequencing …

Owing to the advances in genome editing technologies, research on human pluripotent stem
cells (hPSCs) have recently undergone breakthroughs that enable precise alteration of …

Precise genome editing in human pluripotent stem cells (hPSCs) has potential applications
in isogenic disease modeling and ex vivo stem cell therapy, necessitating diverse genome …

The preferred method for disease modeling using induced pluripotent stem cells (iPSCs) is
to generate isogenic cell lines by correcting or introducing pathogenic mutations. Base …

Precise genome editing is crucial for establishing isogenic human disease models and ex
vivo stem cell therapy from the patient-derived hPSCs. Unlike Cas9-mediated knock-in …

Hereditary inclusion-body myopathies (HIBMs) are rare muscle disorders with autosomal-
recessive or dominant inheritance and presence of muscle fibers with rimmed vacuoles and …

GNE myopathy is a hereditary muscle disorder characterized by muscle atrophy and
weakness initially involving the lower distal extremities. The treatment of GNE myopathy …

The recent advances in human pluripotent stem cells (hPSCs) enable to precisely edit the
desired bases in hPSCs to be used for the establishment of isogenic disease models and …

Stem cells such as induced pluripotent stem cells, embryonic stem cells, and hematopoietic
stem and progenitor cells are growing in importance in disease modeling and regenerative …

Neuromuscular diseases (NMDs) are a genetically or clinically heterogeneous group of
diseases that involve injury or dysfunction of neuromuscular tissue components, including …