Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for F...
Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD
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Publisher
United States: Elsevier Limited
Journal title
Language
English
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Publisher
United States: Elsevier Limited
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Scope and Contents
Contents
RNAi emerged as a prospective molecular therapy nearly 15 years ago. Since then, two major RNAi platforms have been under development: oligonucleotides and gene therapy. Oligonucleotide-based approaches have seen more advancement, with some promising therapies that may soon reach market. In contrast, vector-based approaches for RNAi therapy have re...
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Full title
Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD
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TN_cdi_doaj_primary_oai_doaj_org_article_a85b27f6001d49549c5557e9c633b735
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_a85b27f6001d49549c5557e9c633b735
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ISSN
2329-0501
E-ISSN
2329-0501
DOI
10.1016/j.omtm.2017.12.005
