Recent Overview of the Use of iPSCs Huntington's Disease Modeling and Therapy.

摘要: Huntington’s disease (HD) is an inherited, autosomal dominant, degenerative characterized by involuntary movements, cognitive decline, and behavioral impairment ending in death. HD caused expansion the number of CAG repeats huntingtin gene on chromosome 4. To date, no effective therapy for preventing onset or progression has been found, many symptoms do not respond to pharmacologic treatment. However, recent results pre-clinical trials suggest a beneficial effect stem-cell-based therapy. Induced pluripotent stem cells (iPSCs) represent unlimited cell source are most suitable among various types autologous due their patient specificity ability differentiate into variety both vitro vivo. Furthermore, cultivation iPSC-derived neural offers possibility studying etiopathology neurodegenerative diseases, such as HD. Moreover, differentiated can organize three-dimensional (3D) organoids, mimicking complex architecture brain. In this article, we present comprehensive review models, methods differentiating HD–iPSCs desired types, progress editing techniques leading toward