High-Capacity Adenoviral Vectors Permit Robust and Versatile Testing of <i>DMD</i> Gene Repair Tools and Strategies in Human Cells

High-Capacity Adenoviral Vectors Permit Robust and Versatile Testing of <i>DMD</i> Gene Repair Tools and Strategies in Human Cells

Duchenne muscular dystrophy (DMD) is a fatal X-linked muscle wasting disorder arising from mutations in the ~2.4 Mb dystrophin-encoding <i>DMD</i> gene. RNA-guided CRISPR-Cas9 nucleases (RGNs) are opening new DMD therapeutic routes whose bottlenecks include delivering sizable RGN complex...

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Bibliographic Details
Main Authors: Marcella Brescia, Josephine M. Janssen, Jin Liu, Manuel A. F. V. Gonçalves
Format: Article
Language:English
Published: MDPI AG 2020-04-01
Series:Cells
Subjects:
gene editing
gene repair
CRISPR-Cas9
multiplexing
high-specificity nucleases
high-capacity adenoviral vectors
Online Access:https://www.mdpi.com/2073-4409/9/4/869

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https://www.mdpi.com/2073-4409/9/4/869

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