FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W4379185510> ?p ?o ?g. }

• W4379185510 endingPage "151326" @default.
• W4379185510 startingPage "151326" @default.
• W4379185510 abstract "Spinal muscular atrophy (SMA), the most common genetic cause of infantile death, is caused by a mutation in the survival of motor neuron 1 gene (SMN1), leading to the death of motor neurons and progressive muscle weakness. SMN1 normally produces an essential protein called SMN. Although humans possess a paralogous gene called SMN2, ∼90% of the SMN it produces is non-functional. This is due to a mutation in SMN2 that causes the skipping of a required exon during splicing of the pre-mRNA. The first treatment for SMA, nusinersen (brand name Spinraza), was approved by the FDA in 2016 and by the EMU in 2017. Nusinersen is an antisense oligonucleotide-based therapy that alters the splicing of SMN2 to make functional full-length SMN protein. Despite the recent advancements in antisense oligonucleotide therapy and SMA treatment development, nusinersen is faced with a multitude of challenges, such as intracellular and systemic delivery. In recent years, the use of peptide-conjugated phosphorodiamidate morpholino oligomers (PPMOs) in antisense therapy has gained interest. These are antisense oligonucleotides conjugated to cell-penetrating peptides such as Pips and DG9, and they have the potential to address the challenges associated with delivery. This review focuses on the historic milestones, development, current challenges, and future perspectives of antisense therapy for SMA." @default.
• W4379185510 created "2023-06-03" @default.
• W4379185510 creator A5080323628 @default.
• W4379185510 creator A5092076922 @default.
• W4379185510 date "2023-06-01" @default.
• W4379185510 modified "2023-10-14" @default.
• W4379185510 title "Challenges and future perspective of antisense therapy for spinal muscular atrophy: A review" @default.
• W4379185510 cites W1496645167 @default.
• W4379185510 cites W1520097000 @default.
• W4379185510 cites W1564447534 @default.
• W4379185510 cites W1914793851 @default.
• W4379185510 cites W1949952721 @default.
• W4379185510 cites W1965284203 @default.
• W4379185510 cites W1980477780 @default.
• W4379185510 cites W1981347986 @default.
• W4379185510 cites W1984680116 @default.
• W4379185510 cites W1986111537 @default.
• W4379185510 cites W1990393476 @default.
• W4379185510 cites W1990548509 @default.
• W4379185510 cites W1997908444 @default.
• W4379185510 cites W2000725203 @default.
• W4379185510 cites W2005432373 @default.
• W4379185510 cites W2007954199 @default.
• W4379185510 cites W2008628221 @default.
• W4379185510 cites W2011401642 @default.
• W4379185510 cites W2014921813 @default.
• W4379185510 cites W2015808797 @default.
• W4379185510 cites W2020678086 @default.
• W4379185510 cites W2025730023 @default.
• W4379185510 cites W2027990555 @default.
• W4379185510 cites W2045653120 @default.
• W4379185510 cites W2047268435 @default.
• W4379185510 cites W2047345323 @default.
• W4379185510 cites W2052064543 @default.
• W4379185510 cites W2056116259 @default.
• W4379185510 cites W2058591372 @default.
• W4379185510 cites W2061092005 @default.
• W4379185510 cites W2061302367 @default.
• W4379185510 cites W2065794464 @default.
• W4379185510 cites W2071230707 @default.
• W4379185510 cites W2071249297 @default.
• W4379185510 cites W2079335891 @default.
• W4379185510 cites W2090804804 @default.
• W4379185510 cites W2094108169 @default.
• W4379185510 cites W2096650232 @default.
• W4379185510 cites W2104723102 @default.
• W4379185510 cites W2107667231 @default.
• W4379185510 cites W2112430789 @default.
• W4379185510 cites W2113242990 @default.
• W4379185510 cites W2117294016 @default.
• W4379185510 cites W2122372964 @default.
• W4379185510 cites W2126079277 @default.
• W4379185510 cites W2129447667 @default.
• W4379185510 cites W2132343975 @default.
• W4379185510 cites W2136930242 @default.
• W4379185510 cites W2138739944 @default.
• W4379185510 cites W2148320915 @default.
• W4379185510 cites W2152766938 @default.
• W4379185510 cites W2153719074 @default.
• W4379185510 cites W2155526563 @default.
• W4379185510 cites W2167256464 @default.
• W4379185510 cites W2170780799 @default.
• W4379185510 cites W2184051949 @default.
• W4379185510 cites W2300951885 @default.
• W4379185510 cites W2309128823 @default.
• W4379185510 cites W2316132657 @default.
• W4379185510 cites W2324591068 @default.
• W4379185510 cites W2339139143 @default.
• W4379185510 cites W2496954959 @default.
• W4379185510 cites W2520424538 @default.
• W4379185510 cites W2560202024 @default.
• W4379185510 cites W2576909235 @default.
• W4379185510 cites W2579663022 @default.
• W4379185510 cites W2582137641 @default.
• W4379185510 cites W2594133976 @default.
• W4379185510 cites W2612305870 @default.
• W4379185510 cites W2765483614 @default.
• W4379185510 cites W2765825678 @default.
• W4379185510 cites W2771247031 @default.
• W4379185510 cites W2789114047 @default.
• W4379185510 cites W2801513916 @default.
• W4379185510 cites W2805396666 @default.
• W4379185510 cites W2895752535 @default.
• W4379185510 cites W2972356268 @default.
• W4379185510 cites W3005415323 @default.
• W4379185510 cites W3008340394 @default.
• W4379185510 cites W3037517625 @default.
• W4379185510 cites W3041597962 @default.
• W4379185510 cites W3044591863 @default.
• W4379185510 cites W3143238609 @default.
• W4379185510 cites W3167842465 @default.
• W4379185510 cites W4312083439 @default.
• W4379185510 doi "https://doi.org/10.1016/j.ejcb.2023.151326" @default.
• W4379185510 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/37295266" @default.
• W4379185510 hasPublicationYear "2023" @default.
• W4379185510 type Work @default.
• W4379185510 citedByCount "1" @default.
• W4379185510 crossrefType "journal-article" @default.

• next