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Emerging Strategies for Genome Editing in the Brain
1Innovative Genomics Institute, University of California, Berkeley, California 94720, United States; California Institute for Quantitative Biosciences, University of California, Berkeley, California 94720, United States; ORCID https://orcid.org/0000-0003-2937-4703.
Trends in Molecular Medicine
|August 15, 2018
Summary
Researchers delivered genome-editing enzymes using ribonucleoprotein complexes to successfully treat a mouse model of fragile X syndrome (FXS). This approach overcomes challenges in delivering gene-editing tools for potential genetic disease therapies.
Area of Science:
- Molecular Biology
- Genetics
- Biotechnology
Background:
- Genome editing holds great therapeutic potential for genetic disorders.
- Efficient delivery of gene-editing tools to target cells remains a significant challenge.
- Fragile X syndrome (FXS) is a genetic disorder with limited treatment options.
Purpose of the Study:
- To investigate the efficacy of delivering pre-assembled genome-editing enzymes as ribonucleoprotein (RNP) complexes.
- To determine if RNP delivery can rescue a mouse model of fragile X syndrome (FXS).
Main Methods:
- Utilized pre-assembled CRISPR-Cas9 ribonucleoprotein (RNP) complexes for delivery.
- Administered RNPs to a mouse model exhibiting symptoms of fragile X syndrome (FXS).
Main Results:
- Successful rescue of the fragile X syndrome (FXS) mouse model was achieved.
- Demonstrated the feasibility of using RNP complexes for in vivo genome-editing delivery.
Conclusions:
- Ribonucleoprotein complex delivery is a viable strategy for in vivo genome editing.
- This method shows promise for treating genetic disorders like fragile X syndrome (FXS).
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