Typesetting │ Shuicheng WenCRISPR Gene EditingIt is widely recognized as the most attention-grabbing and groundbreaking breakthrough in life sciences since the 21st century. Just eight years after its formal introduction in 2012, it received recognition from the Nobel Prize. By the end of last year, the first CRISPR-based gene-editing therapy was approved by the FDA for marketing, aimed at treating sickle cell disease and β-thalassemia, thus ushering in a new chapter in the treatment of genetic disorders.
The rapid development in the field of artificial intelligence in recent years is expected to unlock gene editors restricted by natural evolution, thereby helping to design more adaptive and powerful gene editors.。April 22, 2024, AIProtein Design CompanyProfluentAnnouncement of LaunchOpenCRISPRTMPlan, releasedThe World's First Open-Source AI-Generated Gene Editor——OpenCRISPR-1。Just like ChatGPT, which is based on large language models generating a new article, large language models can also generate entirely new proteins. And now,Profluent Showcases an AI-Designed Gene Editor from Scratch, Achieving the First Successful Precise Edit of the Human Genome.It is worth mentioning that，OpenCRISPR-1 is open source and can be used for free not only in scientific research but also for commercial purposes.This paves the way for improving the accessibility of gene-editing therapies and reducing their costs, helping to accelerate the development of treatments for thousands of currently incurable genetic diseases. It is worth noting that Profluent has open-sourced the AI-generated gene-editing tool but did not open-source the AI technology itself.Profluent is an AI protein design company established in 2022. It is dedicated to developing groundbreaking drugs and therapies by designing entirely new proteins from scratch using artificial intelligence. Its AI tools, based on large language models, are capable of reading and writing any type of protein, including enzymes, gene editors, antibodies, and the entire protein universe.Currently, the companyTotal Amount Completed$44 millionFinancing.This startup usedTrained in large-scale sequences and biological contextsLarge Language Model（LLM）, generating millions of different non-natural CRISPR-like proteins, thereby exponentially expanding almost all known CRISPR families.The company's achievement is a significant milestone in the field of gene editing, with the potential to accelerate the development of precision treatments for a variety of genetic diseases.。The details of OpenCRISPR-1 were published on the preprint platform bioRxiv, with the paper titled:Design of highly functional genome editors by modeling the universe of CRISPR-Cas sequences。Generate a variety of CRISPR-associated protein familiesSpCas9It is a CRISPR nuclease derived from Streptococcus pyogenes and is currently the most widely used gene editor, which relies on guide RNA.（gRNA）Under the guidance, the DNA double strand at the target site is cleaved in a targeted manner, thereby achieving highly efficient gene editing.And OpenCRISPR-1, which is fully generated by artificial intelligence, byComposed of Cas9-like proteins and gRNA, thisAmino Acid Sequence of Cas9-like ProteinsCompared with SpCas9, there are 400 mutations,With nearly 200 mutations compared to any other known natural CRISPR-associated protein,But it showsGene editing activity and specificity comparable to or better than SpCas9。The research team detailedGeneration and Characterization of OpenCRISPR-1. In the human HEK293T cell line, OpenCRISPR-1 delivered via plasmid,Exhibits gene editing efficiency comparable to SpCas9 and higher specificity in the editing of multiple genomic targets and off-target detection.(Significantly reduce off-target rates at some editing sites)。In addition, when combined with AI-generatedDeaminaseWhen combined, OpenCRISPR-1 is also able to functionBase Editing（Base Editing）The function shows robust A-to-G editing at a set of target sites.Generation of Gene Editing in Human CellsCurrently,Profluent Bio, Inc.Studying purified ribonucleoprotein（RNP）Specificity and Behavior of OpenCRISPR-1 in Complex Form at the Whole-Genome Level. OpenCRISPR-1 is the first version,ProfluentEncourage people to use and testOpenCRISPR-1, and provide feedback,Thereby helping to iterate and advance OpenCRISPRTMPlan.Co-founder and CEO of ProfluentAli MadaniDr. stated,Attempting to use AI-designed biological systems to edit human DNA is a scientific moonshot, and our success points to a future where AI can precisely design what's needed, creating a range of customized treatments for diseases.OpenCRISPR-1 Complex Structure, the World's First Open-Source Gene Editor Designed by Artificial IntelligenceIt should be noted that,Despite the enormous potential of AI-generated gene editors, they are unlikely to impact the pharmaceutical landscape in the short term, as they still require further demonstration of safety and efficacy in preclinical and clinical studies.Nevertheless, the rapid development of generative artificial intelligence technology based on large language models gives us a glimpse into the future,In this future, drugs and treatment methods can be quickly tailored to individuals. This breakthrough achieved by Profluent marks the beginning of a new era in gene editing,In this era, artificial intelligence is playing a central role in designing tools that may revolutionize medicine. As this technology continues to evolve, it holds the promise of bringing us closer to a world,In this world，Gene disease precision treatment is now more accessible and effective than ever before.https://www.profluent.bio/https://www.biorxiv.org/content/10.1101/2024.04.22.590591v1
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