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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
Summary
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.
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.
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