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Literature DB >> 31389773

CRISPR for Neuromuscular Disorders: Gene Editing and Beyond.

Courtney S Young^1,2,3, April D Pyle^2,3,4, Melissa J Spencer^1,2,3.

Abstract

This is a review describing advances in CRISPR/Cas-mediated therapies for neuromuscular disorders (NMDs). We explore both CRISPR-mediated editing and dead Cas approaches as potential therapeutic strategies for multiple NMDs. Last, therapeutic considerations, including delivery and off-target effects, are also discussed.

Entities: Disease

Keywords: CRISPR/Cas; gene editing; gene therapy; muscular dystrophy; neuromuscular disorders

Mesh：

Year: 2019 PMID： 31389773 PMCID： PMC6863376 DOI： 10.1152/physiol.00012.2019

Source DB: PubMed Journal: Physiology (Bethesda) ISSN： 1548-9221

138 in total

1. Genome-wide analysis reveals specificities of Cpf1 endonucleases in human cells.

Authors: Daesik Kim; Jungeun Kim; Junho K Hur; Kyung Wook Been; Sun-Heui Yoon; Jin-Soo Kim
Journal: Nat Biotechnol Date: 2016-06-06 Impact factor: 54.908

2. Applications of CRISPR technologies in research and beyond.

Authors: Rodolphe Barrangou; Jennifer A Doudna
Journal: Nat Biotechnol Date: 2016-09-08 Impact factor: 54.908

3. ERBB3 and NGFR mark a distinct skeletal muscle progenitor cell in human development and hPSCs.

Authors: Michael R Hicks; Julia Hiserodt; Katrina Paras; Wakana Fujiwara; Ascia Eskin; Majib Jan; Haibin Xi; Courtney S Young; Denis Evseenko; Stanley F Nelson; Melissa J Spencer; Ben Van Handel; April D Pyle
Journal: Nat Cell Biol Date: 2017-12-18 Impact factor: 28.824

4. In Vivo Genome Editing Restores Dystrophin Expression and Cardiac Function in Dystrophic Mice.

Authors: Mona El Refaey; Li Xu; Yandi Gao; Benjamin D Canan; T M Ayodele Adesanya; Sarah C Warner; Keiko Akagi; David E Symer; Peter J Mohler; Jianjie Ma; Paul M L Janssen; Renzhi Han
Journal: Circ Res Date: 2017-08-08 Impact factor: 17.367

Review 5. Pathogenic mechanisms of myotonic dystrophy.

Authors: Johanna E Lee; Thomas A Cooper
Journal: Biochem Soc Trans Date: 2009-12 Impact factor: 5.407

6. Identification of Novel Antisense-Mediated Exon Skipping Targets in DYSF for Therapeutic Treatment of Dysferlinopathy.

Authors: Joshua J A Lee; Rika Maruyama; William Duddy; Hidetoshi Sakurai; Toshifumi Yokota
Journal: Mol Ther Nucleic Acids Date: 2018-10-11 Impact factor: 8.886

7. Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy.

Authors: Christopher E Nelson; Yaoying Wu; Matthew P Gemberling; Matthew L Oliver; Matthew A Waller; Joel D Bohning; Jacqueline N Robinson-Hamm; Karen Bulaklak; Ruth M Castellanos Rivera; Joel H Collier; Aravind Asokan; Charles A Gersbach
Journal: Nat Med Date: 2019-02-18 Impact factor: 53.440

8. In Situ Modification of Tissue Stem and Progenitor Cell Genomes.

Authors: Jill M Goldstein; Mohammadsharif Tabebordbar; Kexian Zhu; Leo D Wang; Kathleen A Messemer; Bryan Peacker; Sara Ashrafi Kakhki; Meryem Gonzalez-Celeiro; Yulia Shwartz; Jason K W Cheng; Ru Xiao; Trisha Barungi; Charles Albright; Ya-Chieh Hsu; Luk H Vandenberghe; Amy J Wagers
Journal: Cell Rep Date: 2019-04-23 Impact factor: 9.423

9. Nucleotide-resolution DNA double-strand break mapping by next-generation sequencing.

Authors: Nicola Crosetto; Abhishek Mitra; Maria Joao Silva; Magda Bienko; Norbert Dojer; Qi Wang; Elif Karaca; Roberto Chiarle; Magdalena Skrzypczak; Krzysztof Ginalski; Philippe Pasero; Maga Rowicka; Ivan Dikic
Journal: Nat Methods Date: 2013-03-17 Impact factor: 28.547

10. CRISPR/Cas9 delivery with one single adenoviral vector devoid of all viral genes.

Authors: Eric Ehrke-Schulz; Maren Schiwon; Theo Leitner; Stephan Dávid; Thorsten Bergmann; Jing Liu; Anja Ehrhardt
Journal: Sci Rep Date: 2017-12-07 Impact factor: 4.379

6 in total

1. Comparison of dystrophin expression following gene editing and gene replacement in an aged preclinical DMD animal model.

Authors: Niclas E Bengtsson; Julie M Crudele; Jordan M Klaiman; Christine L Halbert; Stephen D Hauschka; Jeffrey S Chamberlain
Journal: Mol Ther Date: 2022-02-08 Impact factor: 12.910

2. Toward the correction of muscular dystrophy by gene editing.

Authors: Eric N Olson
Journal: Proc Natl Acad Sci U S A Date: 2021-04-30 Impact factor: 11.205

Review 3. CRISPR-Cas9: A Preclinical and Clinical Perspective for the Treatment of Human Diseases.

Authors: Garima Sharma; Ashish Ranjan Sharma; Manojit Bhattacharya; Sang-Soo Lee; Chiranjib Chakraborty
Journal: Mol Ther Date: 2020-09-20 Impact factor: 11.454

4. Dystrophin Gene-Editing Stability Is Dependent on Dystrophin Levels in Skeletal but Not Cardiac Muscles.

Authors: Niclas E Bengtsson; Hichem Tasfaout; Stephen D Hauschka; Jeffrey S Chamberlain
Journal: Mol Ther Date: 2020-11-05 Impact factor: 11.454

5. Targeting Gys1 with AAV-SaCas9 Decreases Pathogenic Polyglucosan Bodies and Neuroinflammation in Adult Polyglucosan Body and Lafora Disease Mouse Models.

Authors: Emrah Gumusgoz; Dikran R Guisso; Sahba Kasiri; Jun Wu; Matthew Dear; Brandy Verhalen; Silvia Nitschke; Sharmistha Mitra; Felix Nitschke; Berge A Minassian
Journal: Neurotherapeutics Date: 2021-04-08 Impact factor: 7.620

Review 6. Molecular and cellular basis of genetically inherited skeletal muscle disorders.

Authors: James J Dowling; Conrad C Weihl; Melissa J Spencer
Journal: Nat Rev Mol Cell Biol Date: 2021-07-13 Impact factor: 94.444

6 in total