A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells-Reference-Cited by-同舟云学术

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A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells

Published:2016-04 Issue:4 Volume:18 Page:533-540
ISSN:1934-5909
Container-title:Cell Stem Cell
language:en
Short-container-title:Cell Stem Cell

Author:

Young Courtney S.,Hicks Michael R.,Ermolova Natalia V.,Nakano Haruko,Jan Majib,Younesi Shahab,Karumbayaram Saravanan,Kumagai-Cresse Chino,Wang Derek,Zack Jerome A.,Kohn Donald B.,Nakano Atsushi,Nelson Stanley F.,Miceli M. Carrie,Spencer Melissa J.,Pyle April D.

Funder

NIAMS

NIH

CURE

JCCC

BSCRC

CFAR

NIH/NIAID

National Science Foundation Graduate Research Fellowship Program

Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research

Rose Hills Foundation Research

Publisher

Elsevier BV

Subject

Cell Biology,Genetics,Molecular Medicine

Reference23 articles.

1. Antisense oligonucleotide-mediated exon skipping for Duchenne muscular dystrophy: progress and challenges;Arechavala-Gomeza;Curr. Gene Ther.,2012

2. Multiexon skipping leading to an artificial DMD protein lacking amino acids from exons 45 through 55 could rescue up to 63% of patients with Duchenne muscular dystrophy;Béroud;Hum. Mutat.,2007

3. The TREAT-NMD DMD Global Database: analysis of more than 7,000 Duchenne muscular dystrophy mutations;Bladen;Hum. Mutat.,2015

4. miR-31 modulates dystrophin expression: new implications for Duchenne muscular dystrophy therapy;Cacchiarelli;EMBO Rep.,2011

5. Analysis of dystrophin gene deletions indicates that the hinge III region of the protein correlates with disease severity;Carsana;Ann. Hum. Genet.,2005

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1. Correction of exon 2, exon 2–9 and exons 8–9 duplications in DMD patient myogenic cells by a single CRISPR/Cas9 system;Scientific Reports;2024-09-11

2. Respiratory characterization of a humanized Duchenne muscular dystrophy mouse model;Respiratory Physiology & Neurobiology;2024-08

3. iPS cell therapy 2.0: Preparing for next‐generation regenerative medicine;BioEssays;2024-06-25

4. A Novel CRISPR-Cas9 Strategy to Target DYSTROPHIN Mutations Downstream of Exon 44 in Patient-Specific DMD iPSCs;Cells;2024-06-04

5. Designing molecules: directing stem cell differentiation;Frontiers in Bioengineering and Biotechnology;2024-05-10

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