Matches in SemOpenAlex for { <https://semopenalex.org/work/W2783265227> ?p ?o ?g. }
- W2783265227 abstract "The discovery that the bacterial CRISPR/Cas9 system can be translated into mammalian cells continues to have an unprecedented impact on the biomedical research community, as it largely facilitates efforts to experimentally interrogate or therapeutically modify the cellular genome. In particular, CRISPR promises the ability to correct disease‐associated genetic defects, or to target and destroy invading foreign DNA, in a simple, efficient, and selective manner directly in affected human cells or tissues. Here, we highlight a set of exciting new strategies that aim at further increasing the therapeutic index of CRISPR technologies, by reducing the size of Cas9 expression cassettes and thus enhancing their compatibility with viral gene delivery vectors. Specifically, we discuss the concept of splitCas9 whereby the Cas9 holo‐protein is segregated into two parts that are expressed individually and reunited in the cell by various means, including use of 1) the gRNA as a scaffold for Cas9 assembly; 2) the rapamycin‐controlled FKBP/FRB system; 3) the light‐regulated Magnet system; or 4) inteins. We describe how these avenues, despite pursuing the identical aim, differ in critical features comprising the extent of spatio‐temporal control of CRISPR activity, and discuss additional improvements to their efficiency or specificity that should foster their clinical translation." @default.
- W2783265227 created "2018-01-26" @default.
- W2783265227 creator A5055110680 @default.
- W2783265227 creator A5076476390 @default.
- W2783265227 date "2018-02-02" @default.
- W2783265227 modified "2023-09-25" @default.
- W2783265227 title "Split Cas9, Not Hairs − Advancing the Therapeutic Index of CRISPR Technology" @default.
- W2783265227 cites W1486227004 @default.
- W2783265227 cites W1570434164 @default.
- W2783265227 cites W1636931877 @default.
- W2783265227 cites W1800635765 @default.
- W2783265227 cites W1965175092 @default.
- W2783265227 cites W1969271703 @default.
- W2783265227 cites W1971237729 @default.
- W2783265227 cites W1976404496 @default.
- W2783265227 cites W1978094899 @default.
- W2783265227 cites W1981160614 @default.
- W2783265227 cites W1985910275 @default.
- W2783265227 cites W1991508224 @default.
- W2783265227 cites W1995793532 @default.
- W2783265227 cites W1995893329 @default.
- W2783265227 cites W1996595554 @default.
- W2783265227 cites W2003171404 @default.
- W2783265227 cites W2003364111 @default.
- W2783265227 cites W2003797386 @default.
- W2783265227 cites W2004590552 @default.
- W2783265227 cites W2006260503 @default.
- W2783265227 cites W2008289270 @default.
- W2783265227 cites W2010666698 @default.
- W2783265227 cites W2012894204 @default.
- W2783265227 cites W2022864103 @default.
- W2783265227 cites W2026520402 @default.
- W2783265227 cites W2032141669 @default.
- W2783265227 cites W2032991133 @default.
- W2783265227 cites W2034252391 @default.
- W2783265227 cites W2034329045 @default.
- W2783265227 cites W2036176224 @default.
- W2783265227 cites W2037965284 @default.
- W2783265227 cites W2041131616 @default.
- W2783265227 cites W2045435533 @default.
- W2783265227 cites W2050480410 @default.
- W2783265227 cites W2051546433 @default.
- W2783265227 cites W2056512830 @default.
- W2783265227 cites W2056602419 @default.
- W2783265227 cites W2063408699 @default.
- W2783265227 cites W2064815984 @default.
- W2783265227 cites W2070614858 @default.
- W2783265227 cites W2077659966 @default.
- W2783265227 cites W2079017842 @default.
- W2783265227 cites W2081064825 @default.
- W2783265227 cites W2088555526 @default.
- W2783265227 cites W2096261947 @default.
- W2783265227 cites W2099062342 @default.
- W2783265227 cites W2107959600 @default.
- W2783265227 cites W2108038398 @default.
- W2783265227 cites W2112295554 @default.
- W2783265227 cites W2123746851 @default.
- W2783265227 cites W2125451007 @default.
- W2783265227 cites W2127063807 @default.
- W2783265227 cites W2133171074 @default.
- W2783265227 cites W2134288637 @default.
- W2783265227 cites W2142515819 @default.
- W2783265227 cites W2147193337 @default.
- W2783265227 cites W2150028503 @default.
- W2783265227 cites W2152505144 @default.
- W2783265227 cites W2152569432 @default.
- W2783265227 cites W2156319424 @default.
- W2783265227 cites W2158243563 @default.
- W2783265227 cites W2158637234 @default.
- W2783265227 cites W2160051211 @default.
- W2783265227 cites W2160986529 @default.
- W2783265227 cites W2164527203 @default.
- W2783265227 cites W2165269455 @default.
- W2783265227 cites W2167380395 @default.
- W2783265227 cites W2167659424 @default.
- W2783265227 cites W2169244146 @default.
- W2783265227 cites W2173709025 @default.
- W2783265227 cites W2179681966 @default.
- W2783265227 cites W2182456467 @default.
- W2783265227 cites W2187840949 @default.
- W2783265227 cites W2188223075 @default.
- W2783265227 cites W2196662084 @default.
- W2783265227 cites W2203460689 @default.
- W2783265227 cites W2204875887 @default.
- W2783265227 cites W2208696160 @default.
- W2783265227 cites W2224398381 @default.
- W2783265227 cites W2253584908 @default.
- W2783265227 cites W2265714931 @default.
- W2783265227 cites W2470054126 @default.
- W2783265227 cites W2508903105 @default.
- W2783265227 cites W2511569157 @default.
- W2783265227 cites W2528022620 @default.
- W2783265227 cites W2550119581 @default.
- W2783265227 cites W2564226239 @default.
- W2783265227 cites W2588637030 @default.
- W2783265227 cites W2598668654 @default.
- W2783265227 cites W2603828830 @default.
- W2783265227 cites W2604706505 @default.
- W2783265227 cites W2604891032 @default.
- W2783265227 cites W2609019318 @default.