FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W2080267978> ?p ?o ?g. }

• W2080267978 endingPage "356" @default.
• W2080267978 startingPage "353" @default.
• W2080267978 abstract "Spinal muscular atrophy (SMA), one of the most frequent and devastating genetic disorders causing neuromuscular degeneration, has reached the forefront of clinical translation. The quite unique genetic situation of SMA patients, who lack functional SMN1 but carry the misspliced SMN2 copy gene, creates the possibility of correcting SMN2 splicing by antisense oligonucleotides or drugs. Both strategies showed impressive results in pre-clinical trials and are now in Phase II-III clinical trials. SMN gene therapy approaches using AAV9-SMN vectors are also highly promising and have entered a Phase I clinical trial. However, careful analysis of SMA animal models and patients has revealed some limitations that need to be taken very seriously, including: i) a limited time-window for successful therapy delivery, making neonatal screening of SMA mandatory; ii) multi-organ impairment, requiring systemic delivery of therapies; and iii) a potential need for combined therapies that both increase SMN levels and target pathways that preserve/rescue motor neuron function over the lifespan. Meeting these challenges will likely be crucial to cure SMA, instead of only ameliorating symptoms, particularly in its most severe form. This review discusses therapies currently in clinical trials, the hopes for SMA therapy, and the potential limitations of these new approaches." @default.
• W2080267978 created "2016-06-24" @default.
• W2080267978 creator A5014981525 @default.
• W2080267978 creator A5022587380 @default.
• W2080267978 creator A5030820510 @default.
• W2080267978 creator A5042253588 @default.
• W2080267978 creator A5051623556 @default.
• W2080267978 date "2015-04-28" @default.
• W2080267978 modified "2023-09-30" @default.
• W2080267978 title "Moving towards treatments for spinal muscular atrophy: hopes and limits" @default.
• W2080267978 cites W162861282 @default.
• W2080267978 cites W1986111537 @default.
• W2080267978 cites W1988037421 @default.
• W2080267978 cites W1992985034 @default.
• W2080267978 cites W2000725203 @default.
• W2080267978 cites W2030993809 @default.
• W2080267978 cites W2045117117 @default.
• W2080267978 cites W2058262490 @default.
• W2080267978 cites W2062270769 @default.
• W2080267978 cites W2069454280 @default.
• W2080267978 cites W2084774766 @default.
• W2080267978 cites W2085375372 @default.
• W2080267978 cites W2115579134 @default.
• W2080267978 cites W2119878613 @default.
• W2080267978 cites W2124852855 @default.
• W2080267978 cites W2135636923 @default.
• W2080267978 cites W2140814127 @default.
• W2080267978 cites W2147552101 @default.
• W2080267978 cites W2153719074 @default.
• W2080267978 cites W2157458455 @default.
• W2080267978 cites W2160739594 @default.
• W2080267978 cites W2166884070 @default.
• W2080267978 cites W2170787818 @default.
• W2080267978 doi "https://doi.org/10.1517/14728214.2015.1041375" @default.
• W2080267978 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/25920617" @default.
• W2080267978 hasPublicationYear "2015" @default.
• W2080267978 type Work @default.
• W2080267978 sameAs 2080267978 @default.
• W2080267978 citedByCount "42" @default.
• W2080267978 countsByYear W20802679782015 @default.
• W2080267978 countsByYear W20802679782016 @default.
• W2080267978 countsByYear W20802679782017 @default.
• W2080267978 countsByYear W20802679782018 @default.
• W2080267978 countsByYear W20802679782019 @default.
• W2080267978 countsByYear W20802679782021 @default.
• W2080267978 countsByYear W20802679782022 @default.
• W2080267978 crossrefType "journal-article" @default.
• W2080267978 hasAuthorship W2080267978A5014981525 @default.
• W2080267978 hasAuthorship W2080267978A5022587380 @default.
• W2080267978 hasAuthorship W2080267978A5030820510 @default.
• W2080267978 hasAuthorship W2080267978A5042253588 @default.
• W2080267978 hasAuthorship W2080267978A5051623556 @default.
• W2080267978 hasBestOaLocation W20802679781 @default.
• W2080267978 hasConcept C104317684 @default.
• W2080267978 hasConcept C111599444 @default.
• W2080267978 hasConcept C114614502 @default.
• W2080267978 hasConcept C142724271 @default.
• W2080267978 hasConcept C161921814 @default.
• W2080267978 hasConcept C169760540 @default.
• W2080267978 hasConcept C19527891 @default.
• W2080267978 hasConcept C2778045022 @default.
• W2080267978 hasConcept C2778558090 @default.
• W2080267978 hasConcept C2779134260 @default.
• W2080267978 hasConcept C3020000666 @default.
• W2080267978 hasConcept C33923547 @default.
• W2080267978 hasConcept C535046627 @default.
• W2080267978 hasConcept C54355233 @default.
• W2080267978 hasConcept C60644358 @default.
• W2080267978 hasConcept C71924100 @default.
• W2080267978 hasConcept C86803240 @default.
• W2080267978 hasConceptScore W2080267978C104317684 @default.
• W2080267978 hasConceptScore W2080267978C111599444 @default.
• W2080267978 hasConceptScore W2080267978C114614502 @default.
• W2080267978 hasConceptScore W2080267978C142724271 @default.
• W2080267978 hasConceptScore W2080267978C161921814 @default.
• W2080267978 hasConceptScore W2080267978C169760540 @default.
• W2080267978 hasConceptScore W2080267978C19527891 @default.
• W2080267978 hasConceptScore W2080267978C2778045022 @default.
• W2080267978 hasConceptScore W2080267978C2778558090 @default.
• W2080267978 hasConceptScore W2080267978C2779134260 @default.
• W2080267978 hasConceptScore W2080267978C3020000666 @default.
• W2080267978 hasConceptScore W2080267978C33923547 @default.
• W2080267978 hasConceptScore W2080267978C535046627 @default.
• W2080267978 hasConceptScore W2080267978C54355233 @default.
• W2080267978 hasConceptScore W2080267978C60644358 @default.
• W2080267978 hasConceptScore W2080267978C71924100 @default.
• W2080267978 hasConceptScore W2080267978C86803240 @default.
• W2080267978 hasIssue "3" @default.
• W2080267978 hasLocation W20802679781 @default.
• W2080267978 hasLocation W20802679782 @default.
• W2080267978 hasOpenAccess W2080267978 @default.
• W2080267978 hasPrimaryLocation W20802679781 @default.
• W2080267978 hasRelatedWork W1575119521 @default.
• W2080267978 hasRelatedWork W2005397880 @default.
• W2080267978 hasRelatedWork W2006837643 @default.
• W2080267978 hasRelatedWork W2013301809 @default.
• W2080267978 hasRelatedWork W2080267978 @default.
• W2080267978 hasRelatedWork W2546023982 @default.

• next