The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Nov. 05, 2019
Filed:
Feb. 09, 2017
Association Institut DE Myologie, Paris, FR;
Sorbonne Universite, Paris, FR;
Universitat Bern, Bern, CH;
Centre National DE LA Recherche Scientifique, Paris, FR;
Institut National DE LA Sante ET DE LA Recherche Medicale, Paris, FR;
Daniel Schumperli, Bern, CH;
Christian Leumann, Bern, CH;
Denis Furling, Paris, FR;
Luis Garcia, Bailly, FR;
Thomas Voit, London, GB;
ASSOCIATION INSTITUT DE MYOLOGIE, Paris, FR;
SORBONNE UNIVERSITE, Paris, FR;
UNIVERSITAT BERN, Bern, CH;
CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE, Paris, FR;
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE, Paris, FR;
Abstract
Provided are tricyclo-DNA (tc-DNA) AON and methods employing tc-DNA AON for modifying splicing events that occur during pre-mRNA processing. Tricyclo-DNA (tc-DNA) AON are described that may be used to facilitate exon skipping or to mask intronic silencer sequences and/or terminal stein-loop sequences during pre-mRNA processing and to target RNase-mediated destruction of processed mRNA. Tc-DNA AON described herein may be used in methods for the treatment of Duchenne Muscular Dystrophy by skipping a mutated exon 23 or exon 51 within a dystrophin gene to restore functionality of a dystrophin protein; in methods for the treatment of Spinal Muscular Atrophy by masking an intronic silencing sequence and/or a terminal stem-loop sequence within an SMN2 gene to yield modified functional SMN2 protein, including an amino acid sequence encoded by exon 7, which is capable of at least partially complementing a non-functional SMN1 protein; and in methods for the treatment of Steinert's Myotonic Dystrophy by targeting the destruction of a mutated DM1 mRNA comprising 3'-terminal CUG repeats.