Matches in SemOpenAlex for { <https://semopenalex.org/work/W4360985259> ?p ?o ?g. }
- W4360985259 abstract "Inborn errors of immunity (IEIs) are a group of inherited disorders caused by mutations in the protein-coding genes involved in innate and/or adaptive immunity. Hematopoietic stem cell transplantation (HSCT) is a mainstay definitive therapy for many severe IEIs. However, the lack of HLA-matched donors increases the risk of developing severe immunological complications. Gene therapy provides long-term clinical benefits and could be an attractive therapeutic strategy for IEIs. In this review, we describe the development and evolution of clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated proteins (Cas) gene-editing systems, including double-strand break (DSB)-based gene editing and DSB-free base editing or prime editing systems. Here, we discuss the advances in and issues associated with CRISPR/Cas gene editing tools and their potential as therapeutic alternatives for IEIs. We also highlight the progress of preclinical studies for the treatment of human genetic diseases, including IEIs, using CRISR/Cas and ongoing clinical trials based on this versatile technology." @default.
- W4360985259 created "2023-03-30" @default.
- W4360985259 creator A5001882175 @default.
- W4360985259 creator A5048903694 @default.
- W4360985259 creator A5049293174 @default.
- W4360985259 creator A5057361452 @default.
- W4360985259 creator A5058152911 @default.
- W4360985259 creator A5066862228 @default.
- W4360985259 date "2023-03-27" @default.
- W4360985259 modified "2023-09-27" @default.
- W4360985259 title "Advances in CRISPR/Cas gene therapy for inborn errors of immunity" @default.
- W4360985259 cites W1583717976 @default.
- W4360985259 cites W1666496871 @default.
- W4360985259 cites W1710674277 @default.
- W4360985259 cites W1855348513 @default.
- W4360985259 cites W1966530560 @default.
- W4360985259 cites W1968342623 @default.
- W4360985259 cites W1981160614 @default.
- W4360985259 cites W1982831423 @default.
- W4360985259 cites W2001082593 @default.
- W4360985259 cites W2005467872 @default.
- W4360985259 cites W2014747507 @default.
- W4360985259 cites W2019670881 @default.
- W4360985259 cites W2039333435 @default.
- W4360985259 cites W2043972484 @default.
- W4360985259 cites W2045435533 @default.
- W4360985259 cites W2050419057 @default.
- W4360985259 cites W2056833586 @default.
- W4360985259 cites W2064815984 @default.
- W4360985259 cites W2070614858 @default.
- W4360985259 cites W2072299899 @default.
- W4360985259 cites W2089093986 @default.
- W4360985259 cites W2098669942 @default.
- W4360985259 cites W2107086950 @default.
- W4360985259 cites W2115044545 @default.
- W4360985259 cites W2119324304 @default.
- W4360985259 cites W2122648489 @default.
- W4360985259 cites W2126374378 @default.
- W4360985259 cites W2130174662 @default.
- W4360985259 cites W2137440275 @default.
- W4360985259 cites W2142159826 @default.
- W4360985259 cites W2149480270 @default.
- W4360985259 cites W2156319424 @default.
- W4360985259 cites W2159530833 @default.
- W4360985259 cites W2165869860 @default.
- W4360985259 cites W2167380395 @default.
- W4360985259 cites W2177261700 @default.
- W4360985259 cites W2196662084 @default.
- W4360985259 cites W2197802602 @default.
- W4360985259 cites W2203460689 @default.
- W4360985259 cites W2204875887 @default.
- W4360985259 cites W2208696160 @default.
- W4360985259 cites W2209402024 @default.
- W4360985259 cites W2260024370 @default.
- W4360985259 cites W2260480416 @default.
- W4360985259 cites W2270544154 @default.
- W4360985259 cites W2273050936 @default.
- W4360985259 cites W2275463106 @default.
- W4360985259 cites W2323613689 @default.
- W4360985259 cites W2336531194 @default.
- W4360985259 cites W2336828812 @default.
- W4360985259 cites W2344579973 @default.
- W4360985259 cites W2480829753 @default.
- W4360985259 cites W2511090954 @default.
- W4360985259 cites W2511966007 @default.
- W4360985259 cites W2513580483 @default.
- W4360985259 cites W2529663648 @default.
- W4360985259 cites W2547418311 @default.
- W4360985259 cites W2548203818 @default.
- W4360985259 cites W2562477383 @default.
- W4360985259 cites W2567931568 @default.
- W4360985259 cites W2577713652 @default.
- W4360985259 cites W2579991322 @default.
- W4360985259 cites W2582314111 @default.
- W4360985259 cites W2588556918 @default.
- W4360985259 cites W2590764628 @default.
- W4360985259 cites W2591888959 @default.
- W4360985259 cites W2593100189 @default.
- W4360985259 cites W2606493012 @default.
- W4360985259 cites W2607299435 @default.
- W4360985259 cites W2621647941 @default.
- W4360985259 cites W2621848900 @default.
- W4360985259 cites W2740810575 @default.
- W4360985259 cites W2751183108 @default.
- W4360985259 cites W2751474071 @default.
- W4360985259 cites W2752073586 @default.
- W4360985259 cites W2752453466 @default.
- W4360985259 cites W2755227731 @default.
- W4360985259 cites W2755653587 @default.
- W4360985259 cites W2758269208 @default.
- W4360985259 cites W2761794675 @default.
- W4360985259 cites W2761956671 @default.
- W4360985259 cites W2766599608 @default.
- W4360985259 cites W2778806019 @default.
- W4360985259 cites W2781931843 @default.
- W4360985259 cites W2784423079 @default.
- W4360985259 cites W2787024379 @default.
- W4360985259 cites W2791408070 @default.
- W4360985259 cites W2791744212 @default.
- W4360985259 cites W2791876458 @default.