
Liana Lareau
BioengineeringLiana Lareau is an Assistant Professor in the Department of Bioengineering. She earned her undergraduate degree from the Massachusetts Institute of Technology and her PhD from UC Berkeley.
Spark Award Project
Engineering Poison Exons to Treat Repeat Expansion Diseases
This project focuses on developing kill switches to deactivate disease-causing genes, particularly focusing on challenging conditions like Huntington’s disease. Standard CRISPR approaches for these conditions have limitations. Instead, the project proposes a novel method utilizing machine learning and CRISPR editing to insert ‘poison exons’ into disease genes. These exons, when incorporated into mRNA, eliminate toxic proteins responsible for the disease. This approach offers a versatile platform for targeting various genetic diseases, particularly repeat expansion disorders.
Liana Lareau’s Story
Targeting Genetic Diseases with CRISPR: Engineering ‘Poison Exons’ to Turn Off Harmful Genes
By: Niki Borghei
Liana Lareau, Assistant Professor in the Department of Bioengineering, is leading a groundbreaking approach to treat some of the most challenging genetic diseases, including Huntington’s disease, using advanced CRISPR gene editing. These diseases, known as repeat expansion diseases, are caused by abnormal repeats in the DNA sequence, and currently, there are no effective treatments available. The hope is that CRISPR, which has shown great promise in editing genes, can offer a solution. However, targeting these problematic genes with CRISPR alone has proven to be difficult and ineffective.
To overcome this challenge, her Spark Award project envisions a new method that combines machine learning and CRISPR editing to introduce what are called “poison exons” into the disease-causing gene. Exons are segments of a gene that are usually spliced together to make mRNA, which is then used to create proteins. In this case, the poison exons are engineered to disrupt the production of toxic mRNA and proteins, which are responsible for causing the disease. Once inserted into the gene, these poison exons essentially act as “kill switches,” turning off the harmful gene expression.
This innovative method is not only a promising approach for treating repeat expansion diseases but also offers a flexible platform that can be adapted to target other similar genetic diseases. By using poison exons, researchers hope to offer a more effective way of treating conditions that were previously thought to be untreatable, bringing new hope to patients with these debilitating genetic disorders.