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Updated: Aug 20, 2025

CRISPR/Cas9 Technology in Restoring Dystrophin Expression in iPSC-Derived Muscle Progenitors

CRISPR/Cas9 Technology in Restoring Dystrophin Expression in iPSC-Derived Muscle Progenitors

Published on: September 14, 2019

Duchenne Muscular Dystrophy Gene Therapy.

Fawzy A Saad^1,2, Jasen F Saad², Gabriele Siciliano³

¹Department of Biology, Padua University School of Medicine, Via Trieste 75, Padova 35121, Italy.

Current Gene Therapy

|November 22, 2022

Summary

This summary is machine-generated.

Gene therapy offers new hope for Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD), aiming to correct the underlying genetic cause in the Dystrophin gene. Emerging strategies like gene transfer and editing show promise for treating these rare neuromuscular disorders.

Keywords:

CRISPR/Cas gene editing Duchene muscular dystrophy adeno-associated virus exon skipping gene drugs gene therapy genetic drugs genomic drugs.microdystrophin pharmacophore

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Purification and Transplantation of Myogenic Progenitor Cell Derived Exosomes to Improve Cardiac Function in Duchenne Muscular Dystrophic Mice

Purification and Transplantation of Myogenic Progenitor Cell Derived Exosomes to Improve Cardiac Function in Duchenne Muscular Dystrophic Mice

Published on: April 10, 2019

Area of Science:

Neuromuscular Disorders
Human Genetics
Molecular Biology

Background:

Duchenne and Becker muscular dystrophies are X-linked recessive diseases impacting skeletal and cardiac muscles.
Current treatments for these conditions are palliative, managing symptoms rather than addressing the root genetic cause within the Dystrophin gene.
Affected individuals, primarily boys, inherit mutations from carrier mothers due to their single X chromosome.

Approach:

Investigating gene therapies including gene transfer, exon skipping, and gene editing to target the genetic defect.
Utilizing engineered adeno-associated virus (AAV) vectors for targeted delivery of therapeutic payloads to specific organs like muscle.
Exploring exosome-mediated delivery of gene editing tools and therapeutic agents to tissues such as the brain and muscle.
Developing strategies to camouflage viral vectors (AAV) and lipid nanoparticles to evade immune system recognition for safer delivery.

Key Points:

Gene reprogramming enables the engineering of AAV vectors for precise organ-specific cargo delivery.
Exosomes can be directed for tissue-specific delivery of gene editing components.
Methods to mask viral capsids and nanoparticles can mitigate immune responses.
These advancements offer potential avenues for treating previously incurable genetic neuromuscular diseases.

Conclusions:

Gene therapy presents a promising frontier for Duchenne and Becker muscular dystrophies, moving beyond symptomatic treatment.
Targeted delivery systems and immune evasion techniques are critical for effective in vivo gene therapy.
This review highlights the evolving landscape of gene therapy for rare genetic diseases, offering hope for patients with DMD and BMD.