Materials and methods for treatment of Duchenne Muscular Dystrophy

Patent metadata

Field	Value
Publication number	US-11369692-B2
Application number	US-201615763328-A
Country	US
Kind code	B2
Filing date	Oct 28, 2016
Priority date	Oct 28, 2015
Publication date	Jun 28, 2022
Grant date	Jun 28, 2022

Abstract

The present application provides materials and methods for treating a patient with Duchenne Muscular Dystrophy (DMD) both ex vivo and in vivo. In addition, the present application provides materials and methods for editing a dystrophin gene in a cell by genome editing.

First claim

What is claimed is:

1. A single-molecule guide RNA (sgRNA) comprising in the 5′ to 3′ direction, a spacer sequence, a minimum CRISPR repeat sequence and a tracrRNA sequence, wherein the spacer sequence consists of an RNA sequence encoded by SEQ ID NO: 1410444, and wherein the sgRNA is capable of removing an AG sequence upstream of Exon 51 in the DMD gene when administered to a cell with a Cas9 protein or a nucleic acid encoding a Cas9 protein.

2. The sgRNA of claim 1 , wherein the sgRNA comprises an RNA modification.

3. The sgRNA of claim 1 , wherein the sgRNA is complexed with a Cas9 protein.

Assignees

• Vertex Pharma

Inventors

• Kabadi Ami Meda
• Cowan Chad Albert
• Lundberg Ante Sven

Classifications

• C12N5/0696

  Artificially induced pluripotent stem cells, e.g. iPS · CPC title

• C12N9/22

  Ribonucleases {[RNase]; Deoxyribonucleases [DNase]} · CPC title

• A61P25/14

  for treating abnormal movements, e.g. chorea, dyskinesia · CPC title

• C12N15/907 Primary

  in mammalian cells · CPC title

• C12N15/113

  Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; {Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing (when used in plants C12N15/8218)} · CPC title