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.

Date of Patent:
Mar. 08, 2022

Filed:

Dec. 29, 2017
Applicants:

Rula Zain-luqman, Taby, SE;

C. I. Edvard Smith, Stockholm, SE;

Inventors:

Rula Zain-Luqman, Taby, SE;

C. I. Edvard Smith, Stockholm, SE;

Eman Zaghloul, Huddinge, SE;

Pedro Moreno, Maia, PT;

Olof Gissberg, Hagersten, SE;

Karin Lundin, Malmkoping, SE;

Jesper Wengel, Langeskov, DK;

Liam Good, London, GB;

Helen Bergquist, Kista, SE;

Assignee:

Other;

Attorney:
Primary Examiner:
Int. Cl.
CPC ...
C07H 21/04 (2006.01); C12N 15/113 (2010.01); A61P 25/28 (2006.01); A61K 31/7088 (2006.01); A61K 47/42 (2017.01); C12Q 1/6883 (2018.01);
U.S. Cl.
CPC ...
C12N 15/113 (2013.01); A61K 31/7088 (2013.01); A61K 47/42 (2013.01); A61P 25/28 (2018.01); C12Q 1/6883 (2013.01); C12N 2310/11 (2013.01); C12Q 2600/156 (2013.01);
Abstract

The present invention relates to anti-gene oligonucleotides adapted to hybridize to DNA in a HTT gene, which are based on locked nucleic acids, phosphorodiamidate morpholino oligomers, (PMO) or equivalent oligonucleotide analogues comprising a (CAG)sequence, and whose target is a sequence where the majority of the repeats are CAG/CTG, for use in down regulating the expression of the HTT gene, resulting in reduced HTT mRNA and protein levels in afflicted subjects, or in diagnosis, treatment and/or prevention of Huntington's disease, and where the anti-gene oligonucleotides target non-canonical DNA structures, including hairpin and cruciform. The invention also relates to a delivery system comprising said oligonucleotides and said use thereof.


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