Matches in SemOpenAlex for { <https://semopenalex.org/work/W2121735520> ?p ?o ?g. }
Showing items 1 to 75 of
75
with 100 items per page.
- W2121735520 endingPage "378" @default.
- W2121735520 startingPage "373" @default.
- W2121735520 abstract "Duchenne muscular dystrophy (DMD) is an X-linked recessive disorder caused by defects in the dystrophin gene. Patients exhibit progressive muscle degeneration, generally begin using a wheelchair before 11 years of age, and die from respiratory or cardiac failure by their early to mid-twenties. Currently no treatment is available for this disease that affects 1 in 3500 newborn males worldwide. As a result, intensive efforts are under way to develop methods to treat DMD by gene therapy. This Commentary will present some of the successes of this research and identify a few of the most challenging obstacles to be overcome before effective treatment is available. Success in treating any disorder by gene therapy hinges on a thorough understanding of the biological parameters of the disease process and the tissues involved. Much of this groundwork has been laid for DMD, and this research effort can serve as a paradigm for the assault" @default.
- W2121735520 created "2016-06-24" @default.
- W2121735520 creator A5052317872 @default.
- W2121735520 creator A5061016246 @default.
- W2121735520 date "1996-06-01" @default.
- W2121735520 modified "2023-09-25" @default.
- W2121735520 title "Progress towards gene therapy for Duchenne muscular dystrophy" @default.
- W2121735520 doi "https://doi.org/10.1677/joe.0.1490373" @default.
- W2121735520 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/8691095" @default.
- W2121735520 hasPublicationYear "1996" @default.
- W2121735520 type Work @default.
- W2121735520 sameAs 2121735520 @default.
- W2121735520 citedByCount "17" @default.
- W2121735520 countsByYear W21217355202016 @default.
- W2121735520 countsByYear W21217355202021 @default.
- W2121735520 countsByYear W21217355202023 @default.
- W2121735520 crossrefType "journal-article" @default.
- W2121735520 hasAuthorship W2121735520A5052317872 @default.
- W2121735520 hasAuthorship W2121735520A5061016246 @default.
- W2121735520 hasConcept C104317684 @default.
- W2121735520 hasConcept C111599444 @default.
- W2121735520 hasConcept C126322002 @default.
- W2121735520 hasConcept C177713679 @default.
- W2121735520 hasConcept C1862650 @default.
- W2121735520 hasConcept C187212893 @default.
- W2121735520 hasConcept C2776356578 @default.
- W2121735520 hasConcept C2776888751 @default.
- W2121735520 hasConcept C2778943923 @default.
- W2121735520 hasConcept C2779030066 @default.
- W2121735520 hasConcept C2779134260 @default.
- W2121735520 hasConcept C2780394045 @default.
- W2121735520 hasConcept C54355233 @default.
- W2121735520 hasConcept C60644358 @default.
- W2121735520 hasConcept C71924100 @default.
- W2121735520 hasConcept C86803240 @default.
- W2121735520 hasConcept C99508421 @default.
- W2121735520 hasConceptScore W2121735520C104317684 @default.
- W2121735520 hasConceptScore W2121735520C111599444 @default.
- W2121735520 hasConceptScore W2121735520C126322002 @default.
- W2121735520 hasConceptScore W2121735520C177713679 @default.
- W2121735520 hasConceptScore W2121735520C1862650 @default.
- W2121735520 hasConceptScore W2121735520C187212893 @default.
- W2121735520 hasConceptScore W2121735520C2776356578 @default.
- W2121735520 hasConceptScore W2121735520C2776888751 @default.
- W2121735520 hasConceptScore W2121735520C2778943923 @default.
- W2121735520 hasConceptScore W2121735520C2779030066 @default.
- W2121735520 hasConceptScore W2121735520C2779134260 @default.
- W2121735520 hasConceptScore W2121735520C2780394045 @default.
- W2121735520 hasConceptScore W2121735520C54355233 @default.
- W2121735520 hasConceptScore W2121735520C60644358 @default.
- W2121735520 hasConceptScore W2121735520C71924100 @default.
- W2121735520 hasConceptScore W2121735520C86803240 @default.
- W2121735520 hasConceptScore W2121735520C99508421 @default.
- W2121735520 hasIssue "3" @default.
- W2121735520 hasLocation W21217355201 @default.
- W2121735520 hasLocation W21217355202 @default.
- W2121735520 hasOpenAccess W2121735520 @default.
- W2121735520 hasPrimaryLocation W21217355201 @default.
- W2121735520 hasRelatedWork W1997975349 @default.
- W2121735520 hasRelatedWork W2032097547 @default.
- W2121735520 hasRelatedWork W2059017658 @default.
- W2121735520 hasRelatedWork W2376176428 @default.
- W2121735520 hasRelatedWork W2594150046 @default.
- W2121735520 hasRelatedWork W2615469091 @default.
- W2121735520 hasRelatedWork W2788314063 @default.
- W2121735520 hasRelatedWork W2990626067 @default.
- W2121735520 hasRelatedWork W4239269329 @default.
- W2121735520 hasRelatedWork W2062804991 @default.
- W2121735520 hasVolume "149" @default.
- W2121735520 isParatext "false" @default.
- W2121735520 isRetracted "false" @default.
- W2121735520 magId "2121735520" @default.
- W2121735520 workType "article" @default.