Schematic representations of site-directed gene-editing strategies for

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Download scientific diagram | Schematic representations of site-directed gene-editing strategies for the therapeutic correction of DMD mutations. A. Non-edited DMD genotype. Out-of-frame deletion results in incorrect splicing of the pre-mRNA and no functional dystrophin protein is expressed. B. Oligonucleotide-mediated gene correction. A point mutation resulting in a premature stop codon or a splice site alteration is corrected with a short homologous oligonucleotide utilising cellular HR mechanisms. C. Gene editing with site-specific nucleases by non-homologous end joining. An engineered nuclease is used to generate a DSB in a downstream exon from a frame-shifting deletion. The cellular NHEJ mechanisms repair the DSB generating a mixture of INDELs at the break point resulting in restoration of the reading frame in a subsection of the edited chromosomes. D. Gene editing and repair using site-specific nucleases and a homologous recombination repair matrix. An engineered nuclease is used to generate a DSB, and a repair matrix containing either the deleted exons (as shown in figure) or the wild-type versions of exons containing missense or nonsense mutations, flanked by arms of homology is co-introduced leading to gene correction or a knock-in of an exonic cDNA block by cellular HR mechanisms and full-length mRNA and dystrophin protein expression. from publication: New developments in the use of gene therapy to treat Duchenne muscular dystrophy | Introduction: Duchenne muscular dystrophy (DMD) is a lethal X-linked inherited disorder characterised by progressive muscle weakness, wasting and degeneration. Although the gene affected in DMD was identified over 25 years ago, there is still no effective treatment. Areas | Duchenne Muscular Dystrophy, Exons and Muscular Dystrophy | ResearchGate, the professional network for scientists.

Linda POPPLEWELL, Royal Holloway, University of London, Egham, RHUL, Department of Biological Sciences

Susan Jarmin's research works Royal Holloway, University of London, Egham (RHUL) and other places

PDF) New developments in the use of gene therapy to treat Duchenne muscular dystrophy

Susan Jarmin's research works Royal Holloway, University of London, Egham (RHUL) and other places

Schematic representations of site-directed gene-editing strategies for

Linda POPPLEWELL, Royal Holloway, University of London, Egham, RHUL, Department of Biological Sciences

Full article: New developments in the use of gene therapy to treat Duchenne muscular dystrophy

A) The SOD-1 (G93A)PS mice (n = 8) have a delayed onset of weight

A) The SOD-1 (G93A)PS mice (n = 8) have a delayed onset of weight

PDF) New developments in the use of gene therapy to treat Duchenne muscular dystrophy

Linda POPPLEWELL, Royal Holloway, University of London, Egham, RHUL, Department of Biological Sciences

Full article: New developments in the use of gene therapy to treat Duchenne muscular dystrophy

A) The SOD-1 (G93A)PS mice (n = 8) have a delayed onset of weight

Hanna KYMÄLÄINEN, PhD Biological Sciences, Royal Holloway, University of London, Egham, RHUL

Linda POPPLEWELL, Royal Holloway, University of London, Egham, RHUL, Department of Biological Sciences