Matches in SemOpenAlex for { <https://semopenalex.org/work/W4387106370> ?p ?o ?g. }
- W4387106370 endingPage "14617" @default.
- W4387106370 startingPage "14617" @default.
- W4387106370 abstract "Splicing of pre-mRNA is a crucial regulatory stage in the pathway of gene expression. The majority of human genes that encode proteins undergo alternative pre-mRNA splicing and mutations that affect splicing are more prevalent than previously thought. Targeting aberrant RNA(s) may thus provide an opportunity to correct faulty splicing and potentially treat numerous genetic disorders. To that purpose, the use of engineered U1 snRNA (either modified U1 snRNAs or exon-specific U1s-ExSpeU1s) has been applied as a potentially therapeutic strategy to correct splicing mutations, particularly those affecting the 5' splice-site (5'ss). Here we review and summarize a vast panoply of studies that used either modified U1 snRNAs or ExSpeU1s to mediate gene therapeutic correction of splicing defects underlying a considerable number of genetic diseases. We also focus on the pre-clinical validation of these therapeutic approaches both in vitro and in vivo, and summarize the main obstacles that need to be overcome to allow for their successful translation to clinic practice in the future." @default.
- W4387106370 created "2023-09-28" @default.
- W4387106370 creator A5014391872 @default.
- W4387106370 creator A5061337372 @default.
- W4387106370 creator A5070831817 @default.
- W4387106370 creator A5073962920 @default.
- W4387106370 creator A5077355971 @default.
- W4387106370 date "2023-09-27" @default.
- W4387106370 modified "2023-10-17" @default.
- W4387106370 title "Development of Engineered-U1 snRNA Therapies: Current Status" @default.
- W4387106370 cites W1544956655 @default.
- W4387106370 cites W1556285913 @default.
- W4387106370 cites W1586291434 @default.
- W4387106370 cites W1975557611 @default.
- W4387106370 cites W1979907792 @default.
- W4387106370 cites W1980847944 @default.
- W4387106370 cites W1991945869 @default.
- W4387106370 cites W1992989994 @default.
- W4387106370 cites W1993215762 @default.
- W4387106370 cites W1999567515 @default.
- W4387106370 cites W2007227891 @default.
- W4387106370 cites W2024588724 @default.
- W4387106370 cites W2032434265 @default.
- W4387106370 cites W2032699759 @default.
- W4387106370 cites W2034793790 @default.
- W4387106370 cites W2038461956 @default.
- W4387106370 cites W2041902128 @default.
- W4387106370 cites W2043778560 @default.
- W4387106370 cites W2045142794 @default.
- W4387106370 cites W2046237610 @default.
- W4387106370 cites W2046880356 @default.
- W4387106370 cites W2050280863 @default.
- W4387106370 cites W2052731608 @default.
- W4387106370 cites W2058604878 @default.
- W4387106370 cites W2067628822 @default.
- W4387106370 cites W2078163685 @default.
- W4387106370 cites W2078824719 @default.
- W4387106370 cites W2085262691 @default.
- W4387106370 cites W2086008735 @default.
- W4387106370 cites W2088999828 @default.
- W4387106370 cites W2090849645 @default.
- W4387106370 cites W2096685689 @default.
- W4387106370 cites W2113696850 @default.
- W4387106370 cites W2117345872 @default.
- W4387106370 cites W2118600301 @default.
- W4387106370 cites W2126456725 @default.
- W4387106370 cites W2127719340 @default.
- W4387106370 cites W2133640184 @default.
- W4387106370 cites W2135222982 @default.
- W4387106370 cites W2139312181 @default.
- W4387106370 cites W2139957948 @default.
- W4387106370 cites W2144326066 @default.
- W4387106370 cites W2157365614 @default.
- W4387106370 cites W2205291443 @default.
- W4387106370 cites W2253196423 @default.
- W4387106370 cites W2300235659 @default.
- W4387106370 cites W2327484967 @default.
- W4387106370 cites W2338991277 @default.
- W4387106370 cites W2400311061 @default.
- W4387106370 cites W2401301750 @default.
- W4387106370 cites W2509473521 @default.
- W4387106370 cites W2522106010 @default.
- W4387106370 cites W2529023771 @default.
- W4387106370 cites W2557704684 @default.
- W4387106370 cites W2581883437 @default.
- W4387106370 cites W2587969176 @default.
- W4387106370 cites W2604933076 @default.
- W4387106370 cites W2739352864 @default.
- W4387106370 cites W2792155122 @default.
- W4387106370 cites W2799812275 @default.
- W4387106370 cites W2800794875 @default.
- W4387106370 cites W2801187834 @default.
- W4387106370 cites W2801391821 @default.
- W4387106370 cites W2900240444 @default.
- W4387106370 cites W2900294718 @default.
- W4387106370 cites W2901874474 @default.
- W4387106370 cites W2908279472 @default.
- W4387106370 cites W2941313684 @default.
- W4387106370 cites W2945739999 @default.
- W4387106370 cites W2963890011 @default.
- W4387106370 cites W2968156885 @default.
- W4387106370 cites W2969644417 @default.
- W4387106370 cites W2971115124 @default.
- W4387106370 cites W2980057685 @default.
- W4387106370 cites W3010660729 @default.
- W4387106370 cites W3042255581 @default.
- W4387106370 cites W3085484738 @default.
- W4387106370 cites W3103991856 @default.
- W4387106370 cites W3182503104 @default.
- W4387106370 cites W3209522964 @default.
- W4387106370 cites W4200471424 @default.
- W4387106370 cites W4214742808 @default.
- W4387106370 cites W4220838245 @default.
- W4387106370 cites W4283457719 @default.
- W4387106370 cites W4286486123 @default.
- W4387106370 cites W4288455553 @default.
- W4387106370 cites W4315784052 @default.
- W4387106370 cites W4321242341 @default.