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Creation of a Novel Humanized Dystrophic Mouse Model of Duchenne Muscular Dystrophy and Application of a CRISPR/Cas9 Gene Editing Therapy.
Young, Courtney S; Mokhonova, Ekaterina; Quinonez, Marbella; Pyle, April D; Spencer, Melissa J.
Afiliación
  • Young CS; Molecular Biology Interdepartmental Program, University of California, Los Angeles, CA, USA.
  • Mokhonova E; Center for Duchenne Muscular Dystrophy at UCLA, University of California, Los Angeles, CA, USA.
  • Quinonez M; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, University of California, LosAngeles, CA, USA.
  • Pyle AD; Center for Duchenne Muscular Dystrophy at UCLA, University of California, Los Angeles, CA, USA.
  • Spencer MJ; Department of Neurology, David Geffen School of Medicine, University of California, Los Angeles, CA, USA.
J Neuromuscul Dis ; 4(2): 139-145, 2017.
Article en En | MEDLINE | ID: mdl-28505980
Duchenne muscular dystrophy is caused by mutations in DMD which disrupt the reading frame. Therapeutic strategies that restore DMD's reading frame, such as exon skipping and CRISPR/Cas9, need to be tested in the context of the human DMD sequence in vivo. We have developed a novel dystrophic mouse model by using CRISPR/Cas9 to delete exon 45 in the human DMD gene in hDMD mice, which places DMD out-of-frame. We have utilized this model to demonstrate that our clinically-relevant CRISPR/Cas9 platform, which targets deletion of human DMD exons 45-55, can be directly applied in vivo to restore dystrophin.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Terapia Genética / Distrofina / Distrofia Muscular de Duchenne / Modelos Animales de Enfermedad Límite: Animals / Humans Idioma: En Revista: J Neuromuscul Dis Año: 2017 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Países Bajos
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Terapia Genética / Distrofina / Distrofia Muscular de Duchenne / Modelos Animales de Enfermedad Límite: Animals / Humans Idioma: En Revista: J Neuromuscul Dis Año: 2017 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Países Bajos