Schematic representations of site-directed gene-editing strategies for
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Description
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
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
BMD and DMD exceptions to the reading frame rule.
Susan Jarmin's research works Royal Holloway, University of London, Egham (RHUL) and other places
Full article: New developments in the use of gene therapy to treat Duchenne muscular dystrophy
PDF) 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) Genotyping of the SOD-1 (G93A)PS mouse reveals a characteristic
Susan Jarmin's research works Royal Holloway, University of London, Egham (RHUL) and other places
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