Matches in SemOpenAlex for { <https://semopenalex.org/work/W2413253062> ?p ?o ?g. }
- W2413253062 endingPage "73" @default.
- W2413253062 startingPage "39" @default.
- W2413253062 abstract "Genome mutagenesis can be achieved in a variety of ways, though a select few are suitable for therapeutic settings. Among them, the harnessing of intracellular homologous recombination affords the safety and efficacy profile suitable for such settings. Recombinagenic donor DNA and mutagenic triplex-forming molecules co-opt this natural recombination phenomenon to enable the specific, heritable editing and targeting of the genome. Editing the genome is achieved by designing the sequence-specific recombinagenic donor DNA to have base mismatches, insertions, and deletions that will be incorporated into the genome when it is used as a template for recombination. Targeting the genome is similarly achieved by designing the sequence-specific mutagenic triplex-forming molecules to further recruit the recombination machinery thereby upregulating its activity with the recombinagenic donor DNA. This combination of extracellularly introduced, designed synthetic molecules and intercellularly ubiquitous, evolved natural machinery enables the mutagenesis of chromosomes and engineering of whole genomes with great fidelity while limiting nonspecific interactions. Herein, we demonstrate the harnessing of recombinagenic donor DNA and mutagenic triplex-forming molecular technology for potential therapeutic applications. These demonstrations involve, among others, utilizing this technology to correct genes so that they become physiologically functional, to induce dormant yet functional genes in place of non-functional counterparts, to place induced genes under regulatory elements, and to disrupt genes to abrogate a cellular vulnerability. Ancillary demonstrations of the design and synthesis of this recombinagenic and mutagenic molecular technology as well as their delivery and assayed interaction with duplex DNA reveal a potent technological platform for engineering specific changes into the living genome." @default.
- W2413253062 created "2016-06-24" @default.
- W2413253062 creator A5004151685 @default.
- W2413253062 creator A5051738749 @default.
- W2413253062 date "2014-11-03" @default.
- W2413253062 modified "2023-09-27" @default.
- W2413253062 title "Therapeutic Genome Mutagenesis Using Synthetic Donor DNA and Triplex-Forming Molecules" @default.
- W2413253062 cites W1502136193 @default.
- W2413253062 cites W1511176131 @default.
- W2413253062 cites W1549673200 @default.
- W2413253062 cites W1576259632 @default.
- W2413253062 cites W1591626938 @default.
- W2413253062 cites W1861710003 @default.
- W2413253062 cites W1896714280 @default.
- W2413253062 cites W1920681646 @default.
- W2413253062 cites W1967262291 @default.
- W2413253062 cites W1967944576 @default.
- W2413253062 cites W1970033940 @default.
- W2413253062 cites W1971054272 @default.
- W2413253062 cites W1978414231 @default.
- W2413253062 cites W1979036577 @default.
- W2413253062 cites W1981362058 @default.
- W2413253062 cites W1982715279 @default.
- W2413253062 cites W1984694497 @default.
- W2413253062 cites W1986267994 @default.
- W2413253062 cites W1990170308 @default.
- W2413253062 cites W1993834457 @default.
- W2413253062 cites W1994278720 @default.
- W2413253062 cites W1994710668 @default.
- W2413253062 cites W1997096397 @default.
- W2413253062 cites W1999071805 @default.
- W2413253062 cites W2002514150 @default.
- W2413253062 cites W2003544045 @default.
- W2413253062 cites W2006790178 @default.
- W2413253062 cites W2010644163 @default.
- W2413253062 cites W2012550606 @default.
- W2413253062 cites W2012821943 @default.
- W2413253062 cites W2014805008 @default.
- W2413253062 cites W2015195452 @default.
- W2413253062 cites W2016513958 @default.
- W2413253062 cites W2018920280 @default.
- W2413253062 cites W2026444780 @default.
- W2413253062 cites W2029738167 @default.
- W2413253062 cites W2029788603 @default.
- W2413253062 cites W2030036223 @default.
- W2413253062 cites W2031971375 @default.
- W2413253062 cites W2032970221 @default.
- W2413253062 cites W2036495161 @default.
- W2413253062 cites W2039111227 @default.
- W2413253062 cites W2039438812 @default.
- W2413253062 cites W2039702376 @default.
- W2413253062 cites W2041487271 @default.
- W2413253062 cites W2042728511 @default.
- W2413253062 cites W2044179272 @default.
- W2413253062 cites W2047209080 @default.
- W2413253062 cites W2049918207 @default.
- W2413253062 cites W2052746828 @default.
- W2413253062 cites W2056022423 @default.
- W2413253062 cites W2057464878 @default.
- W2413253062 cites W2058755882 @default.
- W2413253062 cites W2058867131 @default.
- W2413253062 cites W2060640204 @default.
- W2413253062 cites W2065886740 @default.
- W2413253062 cites W2066547625 @default.
- W2413253062 cites W2067780050 @default.
- W2413253062 cites W2070771664 @default.
- W2413253062 cites W2072036245 @default.
- W2413253062 cites W2078197862 @default.
- W2413253062 cites W2078416859 @default.
- W2413253062 cites W2082061511 @default.
- W2413253062 cites W2082970980 @default.
- W2413253062 cites W2083952227 @default.
- W2413253062 cites W2085069351 @default.
- W2413253062 cites W2085162378 @default.
- W2413253062 cites W2085911449 @default.
- W2413253062 cites W2087330706 @default.
- W2413253062 cites W2087470272 @default.
- W2413253062 cites W2092495234 @default.
- W2413253062 cites W2095703410 @default.
- W2413253062 cites W2108192168 @default.
- W2413253062 cites W2116047439 @default.
- W2413253062 cites W2118710018 @default.
- W2413253062 cites W2123029073 @default.
- W2413253062 cites W2124058652 @default.
- W2413253062 cites W2125289455 @default.
- W2413253062 cites W2126466006 @default.
- W2413253062 cites W2127060939 @default.
- W2413253062 cites W2129242502 @default.
- W2413253062 cites W2133918133 @default.
- W2413253062 cites W2147983584 @default.
- W2413253062 cites W2148611875 @default.
- W2413253062 cites W2149114131 @default.
- W2413253062 cites W2150797448 @default.
- W2413253062 cites W2151668141 @default.
- W2413253062 cites W2152596636 @default.
- W2413253062 cites W2157616199 @default.
- W2413253062 cites W2168707500 @default.
- W2413253062 cites W2169019691 @default.