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CRISPR/Cas9-Mediated Genome Editing for Huntington's Disease
Gabriel Vachey1, Nicole Déglon2
1Laboratory of Neurotherapies and Neuromodulation (LNCM), Neuroscience Research Center (CRN), Lausanne University Hospital (CHUV), Lausanne, Switzerland.
Methods in Molecular Biology (Clifton, N.J.)
|June 2, 2018
Summary
This study explores using viral gene transfer for central nervous system genome editing to treat Huntington's disease. Protocols cover CRISPR/Cas9 vector design, cloning, and efficiency assessment for potential therapeutic applications.
Area of Science:
- Neuroscience
- Molecular Biology
- Genetics
Background:
- Huntington's disease is a fatal neurodegenerative disorder.
- Current treatments are largely symptomatic.
- Genome editing offers a potential disease-modifying strategy.
Purpose of the Study:
- To describe viral-mediated gene transfer for central nervous system genome editing in Huntington's disease.
- To provide protocols for designing genome editing strategies.
- To detail the cloning of CRISPR/Cas9 elements into lentiviral vectors.
Main Methods:
- Design of CRISPR/Cas9-based genome editing strategies.
- Cloning of CRISPR/Cas9 components into lentiviral vectors for central nervous system delivery.
- Assessment of gene editing efficiency and off-target effects.
Main Results:
- Protocols for designing and implementing genome editing strategies are presented.
- Methods for cloning CRISPR/Cas9 into lentiviral vectors are detailed.
- Techniques for assessing cleavage efficiency and potential adverse effects are outlined.
Conclusions:
- Viral-mediated gene transfer provides a viable method for central nervous system genome editing.
- The described protocols facilitate the development of CRISPR/Cas9-based therapies for Huntington's disease.
- Further research is warranted to optimize safety and efficacy.