Organoids are self-organized, three-dimensional structures derived from stem cells that can
mimic the structure and physiology of human organs. Patient-specific induced pluripotent …

The TRAPP (TRAnsport Protein Particle) protein complex is a multi-subunit complex
involved in vesicular transport between intracellular compartments. The TRAPP complex …

Introduction: Glycogen storage disease type III (GSDIII) is a rare genetic disease caused by
mutations in the AGL gene encoding the glycogen debranching enzyme (GDE). The …

Stem cells are a highly desirable tool for regenerative medicine due to unique
characteristics such as immunomodulation and angiogenesis (multipotent cells) and high …

Neuromuscular disorders (NMDs) are a large group of diseases associated with either
alterations of skeletal muscle fibers, motor neurons or neuromuscular junctions. Most of …

Emery-Dreifuss muscular dystrophy type 1 (EDMD1) is a rare genetic disease caused by
mutations in the EMD gene, which encodes the nuclear envelope protein emerin. Despite …

Glycogen storage disease type III (GSDIII) is a rare genetic disorder leading to abnormal
glycogen storage in liver and skeletal muscle. In this study, we conducted a comparative …

Для разработки методов генной терапии и геномного редактирования при моногенных
заболеваниях необходимы клеточные модели из тканей человека, полученных …