Between hope and reality: treatment of genetic diseases through nucleic acid-based drugs
Between hope and reality: treatment of genetic diseases through nucleic acid-based drugs
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Publisher
London: Nature Publishing Group UK
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Language
English
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London: Nature Publishing Group UK
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Contents
Rare diseases (RD) affect a small number of people compared to the general population and are mostly genetic in origin. The first clinical signs often appear at birth or in childhood, and patients endure high levels of pain and progressive loss of autonomy frequently associated with short life expectancy. Until recently, the low prevalence of RD an...
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Full title
Between hope and reality: treatment of genetic diseases through nucleic acid-based drugs
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TN_cdi_doaj_primary_oai_doaj_org_article_356b90573caf4a81804717faca9d0781
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_356b90573caf4a81804717faca9d0781
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ISSN
2399-3642
E-ISSN
2399-3642
DOI
10.1038/s42003-024-06121-9
