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. 16, 2021

Filed:

Mar. 08, 2017
Applicant:

Ucl Business Ltd, London, GB;

Inventors:

Francesco Muntoni, London, GB;

Haiyan Zhou, London, GB;

Mary Reilly, London, GB;

Assignee:

UCL BUSINESS LTD, London, GB;

Attorney:
Primary Examiner:
Int. Cl.
CPC ...
A61K 48/00 (2006.01); C12N 15/11 (2006.01); C07H 21/02 (2006.01); C07H 21/04 (2006.01); C12N 15/113 (2010.01);
U.S. Cl.
CPC ...
C12N 15/1137 (2013.01); C12Y 203/0105 (2013.01); C12N 2310/11 (2013.01); C12N 2310/122 (2013.01); C12N 2310/14 (2013.01); C12N 2310/20 (2017.05); C12N 2310/315 (2013.01); C12N 2310/321 (2013.01); C12N 2310/322 (2013.01); C12N 2310/3231 (2013.01); C12N 2310/341 (2013.01); C12N 2310/3521 (2013.01); C12N 2310/3533 (2013.01); C12N 2320/34 (2013.01);
Abstract

The invention relates to an oligonucleotide that supresses the expression of an allele carrying a dominant mutation that causes hereditary sensory neuropathy type I (HSN1), wherein the suppression takes place through hybridisation of said oligonucleotide to the DNA of said allele or to an RNA transcript of said allele, and which either does not suppress the expression of a wild-type allele not containing the dominant mutation or suppresses the expression of said wild-type allele to a lesser extent than it suppresses the expression of the dominant mutant allele; and also pharmaceutical compositions comprising oligonucleotides of the invention and treatments of HSN1 using such oligonucleotides.


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