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Journal / Genetics / Exon Skiping en Miotubos Directamente…
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Genetics

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Exon Skiping en Miotubos Directamente Reprogramados obtenidos de Células Derivadas de orina humana
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Exon Skiping en Miotubos Directamente Reprogramados obtenidos de Células Derivadas de orina humana

Article DOI: 10.3791/60840-v • 06:20 min • May 7th, 2020
May 7th, 2020 •
1, 1, 1
1Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry

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En este artículo, describimos un protocolo detallado para el modelado eficiente del músculo de la distrofia muscular de Duchenne usando células derivadas de orina convertidas MYOD1para evaluar la restauración de los niveles de ARNm de distrofina y proteína según el exon.

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Genética Número 159 Distrofia muscular de Duchenne (DMD) células derivadas de la orina exonsaltación medicina de precisión músculo esquelético ensayo clínico
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