Gene modification strategies using AO-mediated exon skipping and CRISPR/Cas9 as potential therapies for Duchenne muscular dystrophy patients

Gene modification strategies using AO-mediated exon skipping and CRISPR/Cas9 as potential therapies for Duchenne muscular dystrophy patients

Duchenne muscular dystrophy (DMD) is an X-linked genetic disease affecting 1 in 5000 young males worldwide annually. Patients experience muscle weakness and loss of ambulation at an early age, with ∼75% reduced life expectancy. Recently developed genetic editing strategies aim to convert severe DMD...

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Bibliographic Details
Main Authors: Marthe Helene Solberg, Maryam Shariatzadeh, Samantha L Wilson
Format: Article
Language:English
Published: Wiley 2020-12-01
Series:Engineering Biology
Subjects:
patient diagnosis
cellular biophysics
diseases
muscle
cardiology
microorganisms
molecular biophysics
genetics
neurophysiology
biochemistry
gene therapy
genomics
gene modification strategies
ao-mediated exon skipping
duchenne muscular dystrophy patients
genetic disease
patients experience muscle weakness
reduced life expectancy
developed genetic editing strategies aim
severe dmd phenotypes
milder disease course
antisense oligonucleotide-mediated exon
dystrophin protein expression
potential therapeutic benefits
gene-editing strategies
therapeutic benefit
long-term efficacy
exon skipping drugs
adeno-associated viral-delivered crispr-cas9 gene editing
adeno-associated viral-delivered clustered regularly interspaced short palindromic repeat
Online Access:https://digital-library.theiet.org/content/journals/10.1049/enb.2020.0017

Internet

https://digital-library.theiet.org/content/journals/10.1049/enb.2020.0017

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