Matches in SemOpenAlex for { <https://semopenalex.org/work/W4366418124> ?p ?o ?g. }
- W4366418124 endingPage "535" @default.
- W4366418124 startingPage "522" @default.
- W4366418124 abstract "Duchenne muscular dystrophy (DMD) is a fatal X-linked recessive disease of progressive muscle weakness and wasting caused by the absence of dystrophin protein. Current gene therapy approaches using antisense oligonucleotides require lifelong dosing and have limited efficacy in restoring dystrophin production. A gene editing approach could permanently correct the genome and restore dystrophin protein expression. Here, we describe single-swap editing, in which an adenine base editor edits a single base pair at a splice donor site or splice acceptor site to enable exon skipping or reframing. In human induced pluripotent stem cell-derived cardiomyocytes, we demonstrate that single-swap editing can enable beneficial exon skipping or reframing for the three most therapeutically relevant exons-DMD exons 45, 51, and 53-which could be beneficial for 30% of all DMD patients. Furthermore, an adeno-associated virus delivery method for base editing components can efficiently restore dystrophin production locally and systemically in skeletal and cardiac muscles of a DMD mouse model containing a deletion of Dmd exon 44. Our studies demonstrate single-swap editing as a potential gene editing therapy for common DMD mutations." @default.
- W4366418124 created "2023-04-21" @default.
- W4366418124 creator A5001071159 @default.
- W4366418124 creator A5007822895 @default.
- W4366418124 creator A5017329586 @default.
- W4366418124 creator A5024308650 @default.
- W4366418124 creator A5029617420 @default.
- W4366418124 creator A5065859286 @default.
- W4366418124 creator A5071139285 @default.
- W4366418124 creator A5071339229 @default.
- W4366418124 creator A5071773009 @default.
- W4366418124 creator A5071880908 @default.
- W4366418124 creator A5077065142 @default.
- W4366418124 creator A5084182062 @default.
- W4366418124 date "2023-06-01" @default.
- W4366418124 modified "2023-09-30" @default.
- W4366418124 title "Single-swap editing for the correction of common Duchenne muscular dystrophy mutations" @default.
- W4366418124 cites W1968342623 @default.
- W4366418124 cites W1979870849 @default.
- W4366418124 cites W1987745724 @default.
- W4366418124 cites W1997490017 @default.
- W4366418124 cites W2018483078 @default.
- W4366418124 cites W2018862827 @default.
- W4366418124 cites W2044446206 @default.
- W4366418124 cites W2066180106 @default.
- W4366418124 cites W2112604043 @default.
- W4366418124 cites W2123408008 @default.
- W4366418124 cites W2125665330 @default.
- W4366418124 cites W2233864423 @default.
- W4366418124 cites W2252568502 @default.
- W4366418124 cites W2336828812 @default.
- W4366418124 cites W2548654043 @default.
- W4366418124 cites W2754552929 @default.
- W4366418124 cites W2766599608 @default.
- W4366418124 cites W2784812244 @default.
- W4366418124 cites W2784992484 @default.
- W4366418124 cites W2801589383 @default.
- W4366418124 cites W2803479625 @default.
- W4366418124 cites W2806820493 @default.
- W4366418124 cites W2809042973 @default.
- W4366418124 cites W2883697525 @default.
- W4366418124 cites W2889510460 @default.
- W4366418124 cites W2889513238 @default.
- W4366418124 cites W2912811937 @default.
- W4366418124 cites W2914658811 @default.
- W4366418124 cites W2915459742 @default.
- W4366418124 cites W2918808095 @default.
- W4366418124 cites W2970098032 @default.
- W4366418124 cites W2976425815 @default.
- W4366418124 cites W3000375097 @default.
- W4366418124 cites W3004643830 @default.
- W4366418124 cites W3008434296 @default.
- W4366418124 cites W3012327875 @default.
- W4366418124 cites W3015621743 @default.
- W4366418124 cites W3032206113 @default.
- W4366418124 cites W3033007748 @default.
- W4366418124 cites W3034029360 @default.
- W4366418124 cites W3035764705 @default.
- W4366418124 cites W3092065379 @default.
- W4366418124 cites W3097033736 @default.
- W4366418124 cites W3124760643 @default.
- W4366418124 cites W3155362558 @default.
- W4366418124 cites W3157701048 @default.
- W4366418124 cites W3169303078 @default.
- W4366418124 cites W3169311152 @default.
- W4366418124 cites W3188287630 @default.
- W4366418124 cites W3198514921 @default.
- W4366418124 cites W3210736819 @default.
- W4366418124 cites W4200043625 @default.
- W4366418124 cites W4220838245 @default.
- W4366418124 doi "https://doi.org/10.1016/j.omtn.2023.04.009" @default.
- W4366418124 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/37215149" @default.
- W4366418124 hasPublicationYear "2023" @default.
- W4366418124 type Work @default.
- W4366418124 citedByCount "2" @default.
- W4366418124 countsByYear W43664181242023 @default.
- W4366418124 crossrefType "journal-article" @default.
- W4366418124 hasAuthorship W4366418124A5001071159 @default.
- W4366418124 hasAuthorship W4366418124A5007822895 @default.
- W4366418124 hasAuthorship W4366418124A5017329586 @default.
- W4366418124 hasAuthorship W4366418124A5024308650 @default.
- W4366418124 hasAuthorship W4366418124A5029617420 @default.
- W4366418124 hasAuthorship W4366418124A5065859286 @default.
- W4366418124 hasAuthorship W4366418124A5071139285 @default.
- W4366418124 hasAuthorship W4366418124A5071339229 @default.
- W4366418124 hasAuthorship W4366418124A5071773009 @default.
- W4366418124 hasAuthorship W4366418124A5071880908 @default.
- W4366418124 hasAuthorship W4366418124A5077065142 @default.
- W4366418124 hasAuthorship W4366418124A5084182062 @default.
- W4366418124 hasBestOaLocation W43664181241 @default.
- W4366418124 hasConcept C104317684 @default.
- W4366418124 hasConcept C109825262 @default.
- W4366418124 hasConcept C144501496 @default.
- W4366418124 hasConcept C194583182 @default.
- W4366418124 hasConcept C2778776201 @default.
- W4366418124 hasConcept C2778943923 @default.
- W4366418124 hasConcept C2779030066 @default.
- W4366418124 hasConcept C2780394045 @default.