Therapeutic strategy for spinal muscular atrophy by combining gene supplementation and genome editin...
Therapeutic strategy for spinal muscular atrophy by combining gene supplementation and genome editing
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Publisher
London: Nature Publishing Group UK
Journal title
Language
English
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Publisher
London: Nature Publishing Group UK
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Scope and Contents
Contents
Defect in the
SMN1
gene causes spinal muscular atrophy (SMA), which shows loss of motor neurons, muscle weakness and atrophy. While current treatment strategies, including small molecules or viral vectors, have shown promise in improving motor function and survival, achieving a definitive and long-term correction of SMA’s endogenous mutations...
Alternative Titles
Full title
Therapeutic strategy for spinal muscular atrophy by combining gene supplementation and genome editing
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TN_cdi_doaj_primary_oai_doaj_org_article_10b9828b196546fa8165e5097af9a12a
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_10b9828b196546fa8165e5097af9a12a
Other Identifiers
ISSN
2041-1723
E-ISSN
2041-1723
DOI
10.1038/s41467-024-50095-5
