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Related Concept Videos

CRISPR01:59

CRISPR

52.5K
Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced...
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CRISPR/Cas9 Genome Editing01:28

CRISPR/Cas9 Genome Editing

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The CRISPR-Cas system serves as a bacterial defense mechanism against invading genetic elements such as viruses and plasmids, forming the foundation for its adaptation as a powerful genome-editing tool. Originally discovered in prokaryotes, this system has been repurposed to revolutionize genetic engineering across a wide range of organisms, including plants, animals, and humans. The core component, Cas9, is an endonuclease derived from Streptococcus pyogenes, capable of introducing...
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What is Genetic Engineering?00:49

What is Genetic Engineering?

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Overview
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Satellite Stem Cells and Muscular Dystrophy01:21

Satellite Stem Cells and Muscular Dystrophy

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Satellite stem cells or myosatellite cells are quiescent stem cells that Alexander Mauro first identified in 1961. These cells are located between the sarcolemma, the plasma membrane of muscle fibers, and the basal lamina, the connective tissue sheath covering it. These mononucleated cells are activated in response to muscle injury, can transform into myoblasts, and may form or repair muscle fibers. Myosatellite cells can provide additional myonuclei for muscle regeneration or return to a...
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Precision modification of heart failure signaling by CRISPR-Cas9 base editing.

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Related Experiment Video

Updated: Aug 2, 2025

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

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CRISPR-Editing Therapy for Duchenne Muscular Dystrophy.

Francesco Chemello1, Eric N Olson2,3, Rhonda Bassel-Duby2,3

  • 1Department of Biology, University of Padova, Padova, Italy.

Human Gene Therapy
|April 15, 2023
PubMed
Summary

CRISPR-Cas9 gene editing shows promise for treating Duchenne muscular dystrophy (DMD) by correcting genetic mutations. However, challenges in delivery and immune response must be overcome for clinical application.

Keywords:
AAV vectorsCRISPR-Cas9Duchenne muscular dystrophygene editing

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Direct Reprogramming of Human Fibroblasts into Myoblasts to Investigate Therapies for Neuromuscular Disorders
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Last Updated: Aug 2, 2025

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Area of Science:

  • Biotechnology
  • Genetics
  • Neuromuscular Disorders

Background:

  • Duchenne muscular dystrophy (DMD) is a fatal genetic disorder caused by dystrophin gene mutations.
  • Current treatments do not offer a cure for DMD.
  • Dystrophin protein is crucial for muscle structure and function.

Conclusions:

  • CRISPR-Cas9 gene editing represents a promising therapeutic avenue for Duchenne muscular dystrophy.
  • Further research is needed to address delivery, efficacy, and immunogenicity for successful clinical translation.
  • Overcoming current challenges is critical for realizing the potential of gene editing in treating DMD.