GENETIC CORRECTION OF MYOTONIC DYSTROPHY TYPE 1

European Patent Office Patent

APP PUB NO EP-3142706-A1
SERIAL NO

15725546

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Abstract

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The invention relates to polynucleotides suitable for reducing or eliminating the expression of expanded repeat RNA (CUGexp) of the dystrophy myotonic-protein kinase (DMPK) gene in a cell of a DM-1 patient. The polynucleotides are a combination of a polynucleotide for a site specific nuclease targeting the dystrophy myotonic- protein kinase (DMPK) gene locus, and a donor polynucleotide having 5' and 3' regions which are homologous with the sequence of DMPK gene which flank the target site of the nuclease. The invention further relate to in vivo and in vitro methods to reduce or eliminate CTG repeats in the DMPK gene. The invention further relates to the medical use of polynucleotides and cells for treating DM-1 patient.

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Patent Owner(s)

Patent OwnerAddress
UNIV BRUSSEL VRIJEBE

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Inventor(s)

Inventor Name Address
CHUAH MARINEE 3360 BIERBEEK
VANDENDRIESSCHE THIERRY BIERBEEK
JOUNG J KEITH 1 MAGNOLIA WAY WINCHESTER MASSACHUSETTS 01890 01890
FU YANFANG 55 CAMBRIDGE PARKWAY SUITE 800E CAMBRIDGE MASSACHUSETTS 02142 02142
REYON DEEPAK MALDEN MA 02148

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