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May 8, 2024CellsOpen Access

CRISPR therapies show promise for monogenic blood disorders and muscular dystrophy despite in vivo delivery challenges.

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Why the study?

Hematological and neuromuscular genetic disorders benefit from genome editing but face distinct challenges in clinical translation.

Design

Review

Key result

CRISPR-based gene therapies demonstrate transformative potential for treating monogenic blood disorders and muscular dystrophy, though clinical translation faces challenges like in vivo delivery.

Authors

MLMarine LaurentMGMarine GeoffroyGPGiulia Pavani

Discussion

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Overview

Should not yet change clinical practice; leaves open optimization of in vivo delivery for broader translation.

Structured PICO

P
Population
Patients and preclinical models with monogenic blood disorders (e.g., severe sickle cell disease, transfusion-dependent β-thalassemia, primary immunodeficiencies) and neuromuscular disorders (e.g., Duchenne muscular dystrophy)
I
Intervention
CRISPR-based gene therapies (e.g., CRISPR/Cas9)

This review summarizes the progress and challenges of CRISPR-based gene therapies for hematological and neuromuscular disorders, emphasizing recent clinical milestones and ongoing hurdles in delivery and safety.

Limitations

  • In vivo delivery methods associated with high viral vector doses
  • Safety and immunological concerns
  • Long-term efficacy
  • Potential genotoxicity
  • Adverse immune reactions

Cite This Study

Laurent et al. (2024) conducted a review in Inherited hematological and neuromuscular genetic disorders. CRISPR-based gene therapies was evaluated. CRISPR-based gene therapies demonstrate transformative potential for treating monogenic blood disorders and muscular dystrophy, though clinical translation faces challenges like in vivo delivery.

synapsesocial.com/papers/6a12c2ea83732aa7db9e4232https://doi.org/10.3390/cells13100800
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