One of the broadest research areas aiding drug development involves gene editing. Gene editing, or genome editing, encompasses a set of technologies empowering scientists to modify an organism’s DNA. These technologies enable adding, removing, or altering genetic material at specific locations within a person’s genome. One such technology causing a major upheaval in biomedical research involves CRISPR. This blog post will delve into how CRISPR works and how it is currently used to revolutionize and further medical advancements.
What is CRISPR and How Does It Work?
CRISPR, which stands for Clustered Regularly Interspaced Short Palindromic Repeats, is a hallmark of a bacterial defense system that serves as the foundation for CRISPR/Cas9 (CRISPR-associated protein 9) genome editing technology. This system allows researchers to permanently modify genes in living cells and organisms by targeting specific stretches of genetic code and editing DNA at precise locations. It uses a Cas9 that acts as molecular scissors to cut DNA. Once the DNA is cut, a change in DNA sequence can occur at and around the cute site by harnessing the cell’s natural repair pathways. New DNA fragments can be added to use as a template for when the break is repaired, thereby introducing a healthy or modified sequence. CRISPR genome editing additionally enables the rapid creation of cell and animal models, accelerating research into diseases like mental illness and cancer.
Aside from modifying and replacing, CRISPR can also be used to knock out a gene entirely. It does this by disrupting its function or causing a cell to stop making the protein. Alternatively, it is possible to modify gene expression through epigenetic editing, which can impact the effective level of a gene. Newer approaches are even more specific with techniques like prime editing and base editing that allow scientists to change single letters in the genetic code.
In the future, this technology can potentially treat genetic causes of disease by making it possible to correct mutations at precise locations in the human genome. CRISPR/Cas9 can also help improve agriculture, develop new proposals for adapting to and putting an end to climate change, and study crucial biological principles by having the ability to edit the DNA of almost any organism.
How CRISPR is Currently Used
Diseases/disorders: The most crucial benefit of CRISPR/Cas9 is the potential to treat genetic disorders caused by a single gene mutation, such as cystic fibrosis (CF) and Duchenne’s muscular dystrophy (DMD). In addition, in late 2023, the first CRISPR-based medicine was approved for treating sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). CRISPR is also currently being explored to help with diabetes, cardiovascular disease, and cancer therapies, among many others.
Agriculture: Gene editing is not a foreign concept to agriculture. Farmers have long sought desirable qualities in their crops and livestock, qualities made more easily attainable through selective breeding, cross-pollination, and mutation breeding. Only recently has CRISPR and gene editing technology been added as a tool for improving agriculture. CRISPR allows the genomes of plants and animals to be altered with unparalleled precision to generate desired characteristics. For example, 2022 saw the first appearance of CRISPR-edited foods on grocery shelves. These foods included longer-lasting bananas, tomatoes with higher GABA content, and milder-flavored leafy greens. There has also been gene editing to produce high crop yields and grow bigger fish.
The Ethical Concerns of CRISPR
Ethical concerns emerge when genome editing techniques like CRISPR-Cas9 are employed to modify human genetic material. Although most modifications do not occur in egg and sperm cells (collectively called germline cells), changes made to genes in these cells or embryos can potentially be passed down to future generations. This kind of genome editing creates ethical challenges concerning whether it is acceptable to enhance normal human traits (i.e., height, intelligence) using this technology. Long-term consequences are largely unknown, making the procedure incredibly unsafe. Because of these concerns and the risk of generating a non-therapeutic genetic enhancement, editing germline cells and embryos is currently illegal in the United States and various other countries.
Conclusion
CRISPR has revolutionized genetic research and holds immense promise for addressing pressing challenges in medicine, agriculture, and beyond. Its precision, efficiency, and versatility have paved the way for groundbreaking discoveries and innovations. However, as with any powerful technology, ethical considerations must be carefully weighed to ensure responsible and equitable use. With ongoing advancements and thoughtful guidance, CRISPR continues to shape the future of science and medicine and offers unprecedented opportunities for improving lives worldwide.
