منابع مشابه
Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 System
Exonic duplications account for 10%-15% of all mutations in Duchenne muscular dystrophy (DMD), a severe hereditary neuromuscular disorder. We report a CRISPR (clustered regularly interspaced short palindromic repeat)/Cas9-based strategy to correct the most frequent (exon 2) duplication in the DMD gene by targeted deletion, and tested the efficacy of such an approach in patient-derived myogenic ...
متن کاملThe AAV-mediated and RNA-guided CRISPR/Cas9 system for gene therapy of DMD and BMD.
Mutations in the dystrophin gene (Dmd) result in Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD), which afflict many newborn boys. In 2016, Brain and Development published several interesting articles on DMD treatment with antisense oligonucleotide, kinase inhibitor, and prednisolone. Even more strikingly, three articles in the issue 6271 of Science in 2016 provide new ins...
متن کاملTransformative Learning Theory: A Possibility for Teachers’ Professional Development
Based on the transformative learning theory, it was sought to develop a procedure for helping teachers develop professionally. 13 elementary school teachers in one of the boys schools in Tehran’s district one participated in a workshop consisting of ten sessions and then interviewed. The content of the sessions was drawn from the theoretical basis of the study. Further field observations led to...
متن کاملThe CRISPR/Cas9 System and the Possibility of Genomic Edition for Cardiology
The CRISPR/Cas9 System and the Possibility of Genomic Edition for Cardiology Marcela Corso Arend,1,2 Jessica Olivaes Pereira,1,3 Melissa Medeiros Markoski1 Laboratório de Cardiologia Molecular e Celular Programa de Pós-Graduação em Ciências da Saúde (Cardiologia) Instituto de Cardiologia/ Fundação Universitária de Cardiologia IC/FUC,1 Porto Alegre, RS; Universidade do Vale do Rio dos Sinos UNIS...
متن کاملAdenoviral vectors encoding CRISPR/Cas9 multiplexes rescue dystrophin synthesis in unselected populations of DMD muscle cells
Mutations disrupting the reading frame of the ~2.4 Mb dystrophin-encoding DMD gene cause a fatal X-linked muscle-wasting disorder called Duchenne muscular dystrophy (DMD). Genome editing based on paired RNA-guided nucleases (RGNs) from CRISPR/Cas9 systems has been proposed for permanently repairing faulty DMD loci. However, such multiplexing strategies require the development and testing of del...
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ژورنال
عنوان ژورنال: Science-Business eXchange
سال: 2014
ISSN: 1945-3477
DOI: 10.1038/scibx.2014.1115