AU2018205259B2 - Nucleic acid-polypeptide compositions and methods of inducing exon skipping - Google Patents

Nucleic acid-polypeptide compositions and methods of inducing exon skipping Download PDF

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Publication number
AU2018205259B2
AU2018205259B2 AU2018205259A AU2018205259A AU2018205259B2 AU 2018205259 B2 AU2018205259 B2 AU 2018205259B2 AU 2018205259 A AU2018205259 A AU 2018205259A AU 2018205259 A AU2018205259 A AU 2018205259A AU 2018205259 B2 AU2018205259 B2 AU 2018205259B2
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polynucleic acid
instances
acid molecule
exon
pmo
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AU2018205259A1 (en
Inventor
Rob BURKE
Michael Caramian COCHRAN
Venkata Ramana Doppalapudi
Andrew John Geall
Hanhua Huang
Arthur A. LEVIN
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Avidity Biosciences Inc
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Avidity Biosciences Inc
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    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
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    • A61K48/0041—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
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    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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AU2018205259A 2017-01-06 2018-01-05 Nucleic acid-polypeptide compositions and methods of inducing exon skipping Active AU2018205259B2 (en)

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