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Literature DB >> 26926391

Duchenne muscular dystrophy: CRISPR/Cas9 treatment.

Jerry R Mendell^1,2,3, Louise R Rodino-Klapac^1,2,3.

Abstract

A novel approach to gene correction by genome editing shows great promise as a treatment for Duchenne muscular dystrophy (DMD). CRISPR/Cas9 delivered by adeno-associated virus to a mouse model for DMD demonstrated improvement in function and histology.

Entities: Disease Species

Mesh：

Substances：
Dystrophin

Year: 2016 PMID： 26926391 PMCID： PMC4856761 DOI： 10.1038/cr.2016.28

Source DB: PubMed Journal: Cell Res ISSN： 1001-0602 Impact factor: 25.617

11 in total

1. Dystrophin immunity in Duchenne's muscular dystrophy.

Authors: Jerry R Mendell; Katherine Campbell; Louise Rodino-Klapac; Zarife Sahenk; Chris Shilling; Sarah Lewis; Dawn Bowles; Steven Gray; Chengwen Li; Gloria Galloway; Vinod Malik; Brian Coley; K Reed Clark; Juan Li; Xiao Xiao; Jade Samulski; Scott W McPhee; R Jude Samulski; Christopher M Walker
Journal: N Engl J Med Date: 2010-10-07 Impact factor: 91.245

2. Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy.

Authors: Chengzu Long; Leonela Amoasii; Alex A Mireault; John R McAnally; Hui Li; Efrain Sanchez-Ortiz; Samadrita Bhattacharyya; John M Shelton; Rhonda Bassel-Duby; Eric N Olson
Journal: Science Date: 2015-12-31 Impact factor: 47.728

3. In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy.

Authors: Christopher E Nelson; Chady H Hakim; David G Ousterout; Pratiksha I Thakore; Eirik A Moreb; Ruth M Castellanos Rivera; Sarina Madhavan; Xiufang Pan; F Ann Ran; Winston X Yan; Aravind Asokan; Feng Zhang; Dongsheng Duan; Charles A Gersbach
Journal: Science Date: 2015-12-31 Impact factor: 47.728

4. In vivo gene editing in dystrophic mouse muscle and muscle stem cells.

Authors: Mohammadsharif Tabebordbar; Kexian Zhu; Jason K W Cheng; Wei Leong Chew; Jeffrey J Widrick; Winston X Yan; Claire Maesner; Elizabeth Y Wu; Ru Xiao; F Ann Ran; Le Cong; Feng Zhang; Luk H Vandenberghe; George M Church; Amy J Wagers
Journal: Science Date: 2015-12-31 Impact factor: 47.728

5. Evidence-based path to newborn screening for Duchenne muscular dystrophy.

Authors: Jerry R Mendell; Chris Shilling; Nancy D Leslie; Kevin M Flanigan; Roula al-Dahhak; Julie Gastier-Foster; Kelley Kneile; Diane M Dunn; Brett Duval; Alexander Aoyagi; Cindy Hamil; Maha Mahmoud; Kandice Roush; Lauren Bird; Chelsea Rankin; Heather Lilly; Natalie Street; Ram Chandrasekar; Robert B Weiss
Journal: Ann Neurol Date: 2012-03 Impact factor: 10.422

6. Corticosteroids in Duchenne muscular dystrophy: major variations in practice.

Authors: Robert C Griggs; Barbara E Herr; Allen Reha; Gary Elfring; Leone Atkinson; Valerie Cwik; Elaine McColl; Rabi Tawil; Shree Pandya; Michael P McDermott; Kate Bushby
Journal: Muscle Nerve Date: 2013-04-25 Impact factor: 3.217

7. Long-term benefits of deflazacort treatment for boys with Duchenne muscular dystrophy in their second decade.

Authors: W D Biggar; V A Harris; L Eliasoph; B Alman
Journal: Neuromuscul Disord Date: 2006-03-20 Impact factor: 4.296

8. Randomized, double-blind six-month trial of prednisone in Duchenne's muscular dystrophy.

Authors: J R Mendell; R T Moxley; R C Griggs; M H Brooke; G M Fenichel; J P Miller; W King; L Signore; S Pandya; J Florence
Journal: N Engl J Med Date: 1989-06-15 Impact factor: 91.245

9. Safety, tolerability, and pharmacokinetics of SMT C1100, a 2-arylbenzoxazole utrophin modulator, following single- and multiple-dose administration to healthy male adult volunteers.

Authors: Jon Tinsley; Neil Robinson; Kay E Davies
Journal: J Clin Pharmacol Date: 2015-02-20 Impact factor: 3.126

10. Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy.

Authors: Jerry R Mendell; Nathalie Goemans; Linda P Lowes; Lindsay N Alfano; Katherine Berry; James Shao; Edward M Kaye; Eugenio Mercuri
Journal: Ann Neurol Date: 2016-01-08 Impact factor: 10.422

18 in total

Review 1. High-Definition Medicine.

Authors: Ali Torkamani; Kristian G Andersen; Steven R Steinhubl; Eric J Topol
Journal: Cell Date: 2017-08-24 Impact factor: 41.582

9. mCAL: A New Approach for Versatile Multiplex Action of Cas9 Using One sgRNA and Loci Flanked by a Programmed Target Sequence.

Authors: Gregory C Finnigan; Jeremy Thorner
Journal: G3 (Bethesda) Date: 2016-07-07 Impact factor: 3.154

Review 10. Current and emerging treatment strategies for Duchenne muscular dystrophy.

Authors: Jean K Mah
Journal: Neuropsychiatr Dis Treat Date: 2016-07-22 Impact factor: 2.570

Duchenne muscular dystrophy: CRISPR/Cas9 treatment.

1. Dystrophin immunity in Duchenne's muscular dystrophy.

2. Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy.

3. In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy.

4. In vivo gene editing in dystrophic mouse muscle and muscle stem cells.

5. Evidence-based path to newborn screening for Duchenne muscular dystrophy.

6. Corticosteroids in Duchenne muscular dystrophy: major variations in practice.

7. Long-term benefits of deflazacort treatment for boys with Duchenne muscular dystrophy in their second decade.

8. Randomized, double-blind six-month trial of prednisone in Duchenne's muscular dystrophy.

9. Safety, tolerability, and pharmacokinetics of SMT C1100, a 2-arylbenzoxazole utrophin modulator, following single- and multiple-dose administration to healthy male adult volunteers.

10. Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy.

Review 1. High-Definition Medicine.

Review 2. Molecular Therapies for Muscular Dystrophies.

3. Genome Editing in Cardiovascular Biology.

4. CRISPR/Cas9-Mediated miR-29b Editing as a Treatment of Different Types of Muscle Atrophy in Mice.

Review 5. Current and Emerging Therapies for Duchenne Muscular Dystrophy.

Review 6. Orienting Muscle Stem Cells for Regeneration in Homeostasis, Aging, and Disease.

Review 7. Mitochondria: emerging therapeutic strategies for oocyte rescue.

Review 8. Plasticity of the Muscle Stem Cell Microenvironment.

9. mCAL: A New Approach for Versatile Multiplex Action of Cas9 Using One sgRNA and Loci Flanked by a Programmed Target Sequence.

Review 10. Current and emerging treatment strategies for Duchenne muscular dystrophy.