FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W2912811937> ?p ?o ?g. }

• W2912811937 endingPage "635" @default.
• W2912811937 startingPage "623" @default.
• W2912811937 abstract "Gene therapies using adeno-associated viral (AAV) vectors have advanced into clinical trials for several diseases, including Duchenne muscular dystrophy (DMD). A limitation of AAV is the carrying capacity (∼5 kb) available for genes and regulatory cassettes (RCs). These size constraints are problematic for the 2.2-Mb dystrophin gene. We previously designed a variety of miniaturized micro-dystrophins (μDys) that displayed significant, albeit incomplete, function in striated muscles. To develop μDys proteins with improved performance, we explored structural modifications of the dystrophin central rod domain. Eight μDys variants were studied that carried unique combinations of between four and six of the 24 spectrin-like repeats present in the full-length protein, as well as various hinge domains. Expression of μDys was regulated by a strong but compact muscle-restricted RC (CK8e) or by the ubiquitously active cytomegalovirus (CMV) RC. Vectors were evaluated by intramuscular injection and systemic delivery to dystrophic mdx4cv mice, followed by analysis of skeletal muscle pathophysiology. Two μDys designs were identified that led to increased force generation compared with previous μDys while also localizing neuronal nitric oxide synthase to the sarcolemma. An AAV vector expressing the smaller of these (μDys5) from the CK8e RC is currently being evaluated in a DMD clinical trial." @default.
• W2912811937 created "2019-02-21" @default.
• W2912811937 creator A5033494387 @default.
• W2912811937 creator A5051863075 @default.
• W2912811937 creator A5052317872 @default.
• W2912811937 creator A5054318565 @default.
• W2912811937 creator A5067357791 @default.
• W2912811937 creator A5083984298 @default.
• W2912811937 date "2019-03-01" @default.
• W2912811937 modified "2023-10-16" @default.
• W2912811937 title "Development of Novel Micro-dystrophins with Enhanced Functionality" @default.
• W2912811937 cites W124278200 @default.
• W2912811937 cites W1936484224 @default.
• W2912811937 cites W1963803693 @default.
• W2912811937 cites W1966493859 @default.
• W2912811937 cites W1967886094 @default.
• W2912811937 cites W1976919537 @default.
• W2912811937 cites W1979870849 @default.
• W2912811937 cites W1983974537 @default.
• W2912811937 cites W1990433203 @default.
• W2912811937 cites W1992677182 @default.
• W2912811937 cites W1996773323 @default.
• W2912811937 cites W1998841070 @default.
• W2912811937 cites W2004740563 @default.
• W2912811937 cites W2022628418 @default.
• W2912811937 cites W2028134688 @default.
• W2912811937 cites W2030810535 @default.
• W2912811937 cites W2032097547 @default.
• W2912811937 cites W2032902046 @default.
• W2912811937 cites W2037305406 @default.
• W2912811937 cites W2037724953 @default.
• W2912811937 cites W2042747686 @default.
• W2912811937 cites W2046753954 @default.
• W2912811937 cites W2049442129 @default.
• W2912811937 cites W2052928078 @default.
• W2912811937 cites W2058807250 @default.
• W2912811937 cites W2059155059 @default.
• W2912811937 cites W2062056760 @default.
• W2912811937 cites W2064385916 @default.
• W2912811937 cites W2065979540 @default.
• W2912811937 cites W2075055529 @default.
• W2912811937 cites W2077773751 @default.
• W2912811937 cites W2083455105 @default.
• W2912811937 cites W2088419282 @default.
• W2912811937 cites W2095152514 @default.
• W2912811937 cites W2096901436 @default.
• W2912811937 cites W2098016509 @default.
• W2912811937 cites W2101579071 @default.
• W2912811937 cites W2101685875 @default.
• W2912811937 cites W2103358381 @default.
• W2912811937 cites W2109668527 @default.
• W2912811937 cites W2116885047 @default.
• W2912811937 cites W2119812021 @default.
• W2912811937 cites W2121650438 @default.
• W2912811937 cites W2126792732 @default.
• W2912811937 cites W2128561011 @default.
• W2912811937 cites W2132935601 @default.
• W2912811937 cites W2133862895 @default.
• W2912811937 cites W2139865704 @default.
• W2912811937 cites W2145502582 @default.
• W2912811937 cites W2147735923 @default.
• W2912811937 cites W2152859894 @default.
• W2912811937 cites W2171492836 @default.
• W2912811937 cites W2208696160 @default.
• W2912811937 cites W2588637030 @default.
• W2912811937 cites W2606840399 @default.
• W2912811937 cites W2736939674 @default.
• W2912811937 cites W2741576602 @default.
• W2912811937 cites W2766754144 @default.
• W2912811937 cites W2794488721 @default.
• W2912811937 cites W2800569219 @default.
• W2912811937 cites W2804183139 @default.
• W2912811937 cites W2889510460 @default.
• W2912811937 cites W2950109307 @default.
• W2912811937 cites W89647785 @default.
• W2912811937 doi "https://doi.org/10.1016/j.ymthe.2019.01.002" @default.
• W2912811937 hasPubMedCentralId "https://www.ncbi.nlm.nih.gov/pmc/articles/6403485" @default.
• W2912811937 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/30718090" @default.
• W2912811937 hasPublicationYear "2019" @default.
• W2912811937 type Work @default.
• W2912811937 sameAs 2912811937 @default.
• W2912811937 citedByCount "69" @default.
• W2912811937 countsByYear W29128119372019 @default.
• W2912811937 countsByYear W29128119372020 @default.
• W2912811937 countsByYear W29128119372021 @default.
• W2912811937 countsByYear W29128119372022 @default.
• W2912811937 countsByYear W29128119372023 @default.
• W2912811937 crossrefType "journal-article" @default.
• W2912811937 hasAuthorship W2912811937A5033494387 @default.
• W2912811937 hasAuthorship W2912811937A5051863075 @default.
• W2912811937 hasAuthorship W2912811937A5052317872 @default.
• W2912811937 hasAuthorship W2912811937A5054318565 @default.
• W2912811937 hasAuthorship W2912811937A5067357791 @default.
• W2912811937 hasAuthorship W2912811937A5083984298 @default.
• W2912811937 hasBestOaLocation W29128119371 @default.
• W2912811937 hasConcept C104317684 @default.
• W2912811937 hasConcept C111599444 @default.
• W2912811937 hasConcept C135983454 @default.

• next