Genome Editing in iPSC-Based Neural Systems: From Disease Models to Future Therapeutic Strategies.

ABSTRACT: Therapeutic advances for neurological disorders are challenging due to limited accessibility of the human central nervous system and incomplete understanding of disease mechanisms. Many neurological diseases lack precision treatments, leading to significant disease burden and poor outcome for affected patients. Induced pluripotent stem cell (iPSC) technology provides human neuronal cells that facilitate disease modeling and development of therapies. The use of genome editing, in particular CRISPR-Cas9 technology, has extended the potential of iPSCs, generating new models for a number of disorders, including Alzheimers and Parkinson Disease. Editing of iPSCs, in particular with CRISPR-Cas9, allows generation of isogenic pairs, which differ only in the disease-causing mutation and share the

SUBMITTER: McTague A 

PROVIDER: S-EPMC8525405 | biostudies-literature | 2021

REPOSITORIES: biostudies-literature