CRISPR Research News and Information

Introduction

CRISPR (pronounced "crisper") stands for Clustered Regularly Interspaced Short Palindromic Repeats, which are the hallmark of a bacterial defense system that forms the basis for CRISPR-Cas9 genome editing technology. Emmanuelle Charpentier is the co-inventor of CRISPR. With Dr. Doudna, Dr. Charpentier was involved in the biochemical characterization of guide RNA and Cas9 enzyme-mediated DNA cleavage. ("Cleave" is the scientific term for cut or break apart.)

Main Document

Three major types of CRISPR-Cas systems are at the top of the classification hierarchy. The three types are readily distinguishable by the presence of three unique signature genes:

• Cas3 in type I systems
• Cas9 in type II systems
• Cas10 in type III systems

The essence of CRISPR is simple: it's a way of finding a specific bit of DNA inside a cell. After that, the next step in CRISPR gene editing is usually to alter that piece of DNA. However, CRISPR has also been adapted to do other things, such as turning genes on or off without altering their sequence.

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CRISPR-Cas9 edits genes by cutting DNA and then letting natural DNA repair processes take over. The system consists of the Cas9 enzyme and a guide RNA. The technology allows precise edits to the genome and has swept through laboratories worldwide since its inception in the 2010s. It has countless applications: researchers hope to use it to alter human genes to eliminate diseases, create hardier plants, wipe out pathogens, and more. Researchers regularly use CRISPR to alter genes in plant, bacteria, and animal models. CRISPR technology has been applied in the food and farming industries to engineer probiotic cultures and to immunize industrial cultures (for yogurt, for instance) against infections. It is also used in crops to enhance yield, drought tolerance, and nutritional value.

In the field of genome engineering, the term "CRISPR" or "CRISPR-Cas9" is often used loosely to refer to the various CRISPR-Cas9 and -CPF1 and other systems that can be programmed to target specific stretches of genetic code and to edit DNA at precise locations, as well as for other purposes, such as for new diagnostic tools.

CRISPR genome editing allows scientists to quickly create cell and animal models, which researchers can use to accelerate research into diseases such as cancer and mental illness. In addition, CRISPR is now being developed as a rapid diagnostic tool. Scientists are studying CRISPR for many conditions, including high cholesterol, HIV, and Huntington's disease.

Ethical Concerns

The ethical implications of using gene editing on human beings are perhaps the greatest concern of this branch of technology, but it is not the only concern. While CRISPR can cure some diseases, studies have shown that it could lead to mutations that lead to others down the line. If genetic edits are made to embryos or egg or sperm cells, these changes will be inherited by all future generations. This is one of the greatest ethical concerns of this gene editing: any edits will have a ripple effect and be passed down from generation to generation. Eventually, the entire human species could bear the marks of genetic editing.

Amy and twins Lulu and Nana are three children who were the first genetically engineered humans in history. Known publicly only by these pseudonyms, embryos' genomes were edited using CRISPR technology by scientist He Jiankui to prevent them from contracting HIV from their fathers. The children are now toddlers, and as they grow up, the scientific community faces a complex dilemma: how to care for their well-being and any fallout from the experiment while respecting their private lives.

The Future of CRISPR

As a gene-editing tool, CRISPR-Cas9 has revolutionized biomedical research and may soon enable medical breakthroughs in a way few biological innovations have before. With CRISPR technology, gene editing could be an amazing option for treating neurodegenerative, mental illnesses, and possibly even psychiatric disorders. In addition, CRISPR gene editing has been used to more than double the lifespan of mice engineered to have premature aging disease progeria, also greatly improving their health. The results far surpassed expectations.