In a collaborative study, researchers from The Jackson Laboratory, MIT’s Broad Institute, and Yale University have unveiled an AI-powered approach to understanding DNA sequences that regulate gene activity. This research focuses on refining gene editing techniques through the study of DNA regions known as cis-regulatory elements (CREs). Acting as "on-off switches," CREs are responsible for controlling the activation of specific genes within precise cell types, a process which has proven challenging to decode until now.
Deciphering DNA “Grammar” with AI
The research team employed AI models to analyze extensive DNA data, identifying patterns within CREs that either trigger or suppress gene activity in specific cells. With these findings, scientists created synthetic DNA switches tailored to activate in specific tissues, a breakthrough for targeted gene regulation.
Testing in Living Organisms
To validate these synthetic CREs, researchers conducted tests on animal models, including zebrafish embryos. Results indicated successful tissue-specific gene activation, with markers illuminating only liver cells while leaving other cells unaffected. This precise targeting highlights the potential for future therapeutic approaches that could selectively activate genes in specific tissues or organs.
Thousands of New CREs Created for Targeted Gene Control
Leveraging the AI approach, scientists generated thousands of unique CREs, each designed for controlled gene activation in particular cells. This development could pave the way for genetic treatments that require precise cell targeting, potentially offering new avenues in medical research and therapeutic interventions.
Potential Applications and Future Medical Use
Beyond advancing research, this AI tool holds promise for targeted medical treatments. By enabling gene activation in defined cell types, scientists foresee applications in treating genetic disorders or enhancing tissue repair. Lead researcher Ryan Tewhey emphasized the potential of this technology to “fine-tune gene activity in one tissue,” pointing towards treatments with minimal side effects on unintended cells.
With inputs from agencies
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