CRISPR is a molecular tool that can make precise changes to a gene's DNA. An acronym for "clustered regularly interspaced short palindromic repeats," the tool was discovered in single-celled organisms, where it acts as an immune system to attack invasive viruses. Scientists have since used it to target and manipulate precise sections of DNA—the genetic blueprints for living things.
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One of the most significant advances in modern medical science, CRISPR is a molecular tool for making precise changes to a gene's DNA. The technology has already been approved for treating sickle-cell disease.
The system uses RNA sequences to guide enzymes like Cas9 to specific spots in DNA, where they cut or modify genes. Unlike Cas9, the Cpf1 enzyme makes staggered cuts, which helps insert new DNA more cleanly.
Jennifer Doudna's research uncovered how bacteria use the tool as an immune defense by cutting viral DNA. She and Emmanuelle Charpentier received the 2020 Nobel Prize in chemistry for showing how this mechanism could be engineered to target and edit genes in living cells.
Biochemists from UIC found that persistent Cas9 binding to double-strand breaks impacts DNA repair efficiency and the effectiveness of the gene editing tool. RNA polymerase collisions also dislodge Cas9, which affects the efficiency of the tool.
CRISPR is a revolutionary gene-editing technology already making a real-world impact in helping to treat several diseases and conditions. This simulation provides a microscopic view to help easily visualize the complex science.
Victoria Gray was the first patient to receive a CRISPR-based therapy, which eliminated her symptoms and restored her health. The therapy's initial cost of $2.2M has kept it out of reach of many patients.
The gene editing tool is a modern version of the genetic modification that has been done on crops for centuries to create plants with preferred traits. Other applications of CRISPR include creating plants with higher yields and better resistance to climate challenges, as well as foods with improved shelf life and nutrition.
The clinical trials are designed to determine whether experimental CRISPR-related therapies are safe and effective. The majority of trials involve treatments for blood disorders, including beta thalassemia.
Environmental factors like X-ray exposure may break a strand of DNA, but cells can use a sister strand to patch the damage. Scientists exploit this system by introducing custom DNA templates, which cells use instead to make repairs.
When viruses called phages inject DNA into bacteria, some bacterial species store bits of this DNA in their own genomes. This allows them to recognize and destroy the same virus if it attacks again.
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