Scientific researchers are currently working on creating a new AI technology that can edit people’s DNA, opening up new possibilities for combating illness and diseases more quickly.
First described in a research paper published in April by startup company Profluent, the A.I. technology, known as OpenCRISPR, uses large language models to train the system to identify unnatural proteins. By using A.I., researchers are now able to create gene editing systems from scratch rather than rely on natural gene editors.
OpenCRISPR builds from the CRISPR method; short for “Clustered Regularly Interspaced Short Palindromic Repeats,” CRISPR-Cas9 is a genome editing tool first used in 2012 by Dr. Jennifer Doudna and Dr. Emmanuelle Charpentier.
The technology edits DNA by cutting proteins up and relying on natural repairing processes to manipulate the gene. The CAS9 enzyme functions as the “molecular scissors” while the guide RNA informs the enzyme on where the edit needs to be made in the DNA.
CRISPR has previously been utilized in creating Casegevy; approved in late 2023, the gene therapy treatment modifies stem cells to create healthy red blood cells.
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“It’s phenomenal that the first CRISPR-based treatments for genetic diseases such as sickle cell disease are already changing the lives of patients, but there remains an urgent need to accelerate the development of this technology for thousands of other currently incurable diseases,” said Hilary Eaton, Chief Business Officer at Profluent, per a statement. “Our intention with OpenCRISPR is to partner with cutting-edge research institutions and drug developers with a powerful and practical way to safely expedite the development of new CRISPR genetic therapies.”
The usage of OpenCRISPR and new A.I. advancements comes as Cold Spring Harbor Laboratory also revealed the creation of a virtual laboratory. Created by Cold Spring Harbor Laboratory (CSHL) Assistant Professor Peter Koo, the tool, known as CREME, elaborates on and builds off of the CRISPR interference, essentially creating an A.I. form of the latter.
By creating virtual genomes, the tool lets researchers conduct virtual experiments to analyze changes made to gene activity, opening up doors for those without a lab to undergo their own studies.
“In reality, CRISPRi is incredibly challenging to perform in the laboratory. And you’re limited by the number of perturbations and the scale,” said Koo per a press release. “But since we’re doing all our perturbations [virtually], we can push the boundaries. And the scale of experiments that we performed is unprecedented—hundreds of thousands of perturbation experiments.”
