Prime editing optimized RTT permits the correction of the c.8713C>T mutation in DMD gene

• Hlavní autoři: Cedric Happi Mbakam (Autor), Joel Rousseau (Autor), Yaoyao Lu (Autor), Anne Bigot (Autor), Kamel Mamchaoui (Autor), Vincent Mouly (Autor), Jacques P. Tremblay (Autor)
• Médium: Kniha
• Vydáno: Elsevier, 2022-12-01T00:00:00Z.
• Témata: article
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Duchenne muscular dystrophy is a severe debilitating genetic disease caused by different mutations in the DMD gene leading to the absence of dystrophin protein under the sarcolemma. We used CRISPR-Cas9 prime editing technology for correction of the c.8713C>T mutation in the DMD gene and tested di...