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Making gene editing a therapeutic reality.

Irina Conboy1, Niren Murthy1, Jessy Etienne1

  • 1Bioengineering, UC Berkeley, Berkeley, CA, 94720, USA.

F1000Research
|January 8, 2019
PubMed
Summary
This summary is machine-generated.

CRISPR-Cas9 gene editing faces challenges in clinical application. This review explores solutions and trials for genetic diseases like muscular dystrophies, focusing on editing strategies for recessive and dominant conditions.

Keywords:
AAVAlpha1 Antitrypsin deficiencyCRISPRClinical trialsDMDDNA damageHDRMyotonic DystrophyNHEJnanoparticles

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Area of Science:

  • Biotechnology
  • Genetics
  • Medical Science

Background:

  • CRISPR-Cas9 gene editing holds therapeutic promise but faces significant hurdles for clinical translation.
  • Genetic diseases, including muscular dystrophies, represent key targets for genome editing interventions.

Purpose of the Study:

  • To review current challenges in translating CRISPR-Cas9 gene editing into therapies.
  • To outline strategies for overcoming these bottlenecks.
  • To examine the scope of clinical trials and disease-specific gene editing approaches for genetic myopathies.

Main Methods:

  • Literature review of current research on CRISPR-Cas9 technology.
  • Analysis of ongoing clinical trials for gene editing therapies.
  • Discussion of gene editing principles for recessive and dominant genetic disorders.

Main Results:

  • Identified key bottlenecks in CRISPR-Cas9 clinical application.
  • Highlighted novel strategies to enhance safety and efficacy.
  • Summarized the landscape of CRISPR-Cas9 clinical trials, particularly for genetic myopathies.
  • Differentiated gene editing approaches for recessive versus dominant diseases.

Conclusions:

  • Overcoming current limitations is crucial for realizing CRISPR-Cas9's therapeutic potential.
  • Clinical trials are advancing, showing promise for treating genetic diseases.
  • Targeted gene editing strategies are being developed for various genetic conditions, including Duchenne and myotonic dystrophies.