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• W3136237371 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 same genetic background, for assessment of phenotypic differences and downstream effects. Moreover, genome-wide CRISPR screens allow high-throughput interrogation for genetic modifiers in neuronal phenotypes, leading to discovery of novel pathways, and identification of new therapeutic targets. CRISPR-Cas9 has now evolved beyond altering gene expression. Indeed, fusion of a defective Cas9 (dCas9) nuclease with transcriptional repressors or activation domains allows down-regulation or activation of gene expression (CRISPR interference, CRISPRi; CRISPR activation, CRISPRa). These new tools will improve disease modeling and facilitate CRISPR and cell-based therapies, as seen for epilepsy and Duchenne muscular dystrophy. Genome engineering holds huge promise for the future understanding and treatment of neurological disorders, but there are numerous barriers to overcome. The synergy of iPSC-based model systems and gene editing will play a vital role in the route to precision medicine and the clinical translation of genome editing-based therapies." @default.
• W3136237371 created "2021-03-29" @default.
• W3136237371 creator A5017916851 @default.
• W3136237371 creator A5023853299 @default.
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• W3136237371 creator A5060489608 @default.
• W3136237371 creator A5067840336 @default.
• W3136237371 date "2021-03-15" @default.
• W3136237371 modified "2023-10-16" @default.
• W3136237371 title "Genome Editing in iPSC-Based Neural Systems: From Disease Models to Future Therapeutic Strategies" @default.
• W3136237371 cites W1553983967 @default.
• W3136237371 cites W1951800697 @default.
• W3136237371 cites W1968461255 @default.
• W3136237371 cites W1973260880 @default.
• W3136237371 cites W1976083636 @default.
• W3136237371 cites W1978120353 @default.
• W3136237371 cites W1980775054 @default.
• W3136237371 cites W1981788339 @default.
• W3136237371 cites W1982846895 @default.
• W3136237371 cites W1983772095 @default.
• W3136237371 cites W1999305754 @default.
• W3136237371 cites W2002767268 @default.
• W3136237371 cites W2003171404 @default.
• W3136237371 cites W2003835684 @default.
• W3136237371 cites W2012894262 @default.
• W3136237371 cites W2013482405 @default.
• W3136237371 cites W2015735925 @default.
• W3136237371 cites W2017887802 @default.
• W3136237371 cites W2018286352 @default.
• W3136237371 cites W2019670881 @default.
• W3136237371 cites W2033799616 @default.
• W3136237371 cites W2034469326 @default.
• W3136237371 cites W2036176224 @default.
• W3136237371 cites W2036632122 @default.
• W3136237371 cites W2037991731 @default.
• W3136237371 cites W2038583081 @default.
• W3136237371 cites W2038845286 @default.
• W3136237371 cites W2045435533 @default.
• W3136237371 cites W2050876258 @default.
• W3136237371 cites W2052457398 @default.
• W3136237371 cites W2056495061 @default.
• W3136237371 cites W2057431271 @default.
• W3136237371 cites W2063668515 @default.
• W3136237371 cites W2064815984 @default.
• W3136237371 cites W2080551186 @default.
• W3136237371 cites W2085420625 @default.
• W3136237371 cites W2085563294 @default.
• W3136237371 cites W2086943348 @default.
• W3136237371 cites W2094539666 @default.
• W3136237371 cites W2096912149 @default.
• W3136237371 cites W2099062342 @default.
• W3136237371 cites W2101749257 @default.
• W3136237371 cites W2102127267 @default.
• W3136237371 cites W2102678193 @default.
• W3136237371 cites W2103355834 @default.
• W3136237371 cites W2103433909 @default.
• W3136237371 cites W2113022445 @default.
• W3136237371 cites W2113446817 @default.
• W3136237371 cites W2117526575 @default.
• W3136237371 cites W2121389760 @default.
• W3136237371 cites W2125991715 @default.
• W3136237371 cites W2127862779 @default.
• W3136237371 cites W2129171086 @default.
• W3136237371 cites W2130128490 @default.
• W3136237371 cites W2138977668 @default.
• W3136237371 cites W2146729586 @default.
• W3136237371 cites W2146890278 @default.
• W3136237371 cites W2147076673 @default.
• W3136237371 cites W2154160398 @default.
• W3136237371 cites W2154679217 @default.
• W3136237371 cites W2161365711 @default.
• W3136237371 cites W2163173465 @default.
• W3136237371 cites W2165869860 @default.
• W3136237371 cites W2168460045 @default.
• W3136237371 cites W2168579634 @default.
• W3136237371 cites W2170039745 @default.
• W3136237371 cites W2188223075 @default.
• W3136237371 cites W2223493846 @default.
• W3136237371 cites W2224398381 @default.
• W3136237371 cites W2227580497 @default.
• W3136237371 cites W2231439883 @default.
• W3136237371 cites W2257132471 @default.
• W3136237371 cites W2289704315 @default.
• W3136237371 cites W2292422784 @default.
• W3136237371 cites W2300298646 @default.
• W3136237371 cites W2302008724 @default.
• W3136237371 cites W2315503074 @default.
• W3136237371 cites W2324816762 @default.
• W3136237371 cites W2336828812 @default.
• W3136237371 cites W2338378167 @default.
• W3136237371 cites W2341041396 @default.
• W3136237371 cites W2347191528 @default.
• W3136237371 cites W2406449858 @default.
• W3136237371 cites W2407632744 @default.
• W3136237371 cites W2409671300 @default.
• W3136237371 cites W2464834014 @default.
• W3136237371 cites W2468765937 @default.
• W3136237371 cites W2490424988 @default.
• W3136237371 cites W2503636044 @default.
• W3136237371 cites W2514239685 @default.

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