CRISPR-Cas is a powerful molecular tool for changing the code of life. Jennifer Doudna and her team focus on developing novel CRISPR-Cas technologies and using them to study molecular mechanisms of disease.
Their goal is to establish first-of-their-kind approaches to treat disease by cutting out or modifying harmful DNA. Current research in Doudna’s lab focuses on discovering and characterizing novel CRISPR-Cas-related proteins, developing genome-editing tools for use in vitro and in vivo, and using CRISPR technology to better understand the genetics of certain cancers like glioblastoma. New discoveries in this field continue at a rapid pace, revealing a technology that has widespread applications.
Disease Areas
- Cancer
- Infectious Diseases
- Neurodegenerative Diseases
Areas of Expertise
- CRISPR and Genome Editing
- Biochemistry and Structural Biology
Lab Focus
- Elucidating the mechanisms underlying CRISPR–Cas immunity in bacterial cells, especially the functions of the various Cas proteins discovered so far.
- Developing and applying novel CRISPR-based tools toward applications in biotechnology, diagnostics, neurodegenerative diseases, and other diseases.
- Understanding the mechanisms controlling translation—the reading of messenger RNAs (mRNAs) into proteins—with a focus on factors that regulate mRNA accessibility and on the inhibition of pathogens’ RNA translation by interferon during infection.
Research Impact
Doudna and her colleagues rocked the research world in 2012 by describing a simple way of editing the DNA of any organism using an RNA-guided protein found in bacteria. This technology, called CRISPR-Cas9, has opened the floodgates of possibility for human and non-human applications of gene editing, including assisting researchers in the fight against HIV, sickle cell disease, and muscular dystrophy. While it was possible to modify cells’ genomes prior to CRISPR-Cas9, this platform has revolutionized genetic engineering by its simplicity, versatility, and precision. Doudna’s lab continues to refine the technology by investigating the mechanisms of action of various bacterial Cas proteins, with an eye to adapting their findings toward specific applications in biotechnology or medicine.