The Future of Gene-Editing
CRISPR, What is it and What How Does it Work?
CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats and it is a bacterial defense system that is used for genome editing. It is an ongoing development that is being worked on by scientists all around the world. Its systems can be programmed to target specific genetic code and to edit DNA at precise locations. Systems that are available now, such as CRISPR-Cas13, are able to target specific RNA for unique characteristics that can be used for sensitive diagnostic tools. The spacer sequences in CRISPR are transcribed into short RNA sequences that are capable of guiding the CRISPR system into matching sequences of DNA. When a target DNA is found, an enzyme produced by the CRISPR system called Cas9, binds to the DNA and cuts it, shutting the targeted gene off. Using modified versions of Cas9, researchers can activate gene expression without cutting the DNA, allowing researchers to study the gene’s function.
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How Does CRISPR Compare to Other Methods of Genetic Engineering?
CRISPR-Cas9 is an efficient and customizable alternative to other existing genome editing tools since the system is capable of cutting a DNA strand by itself; allowing CRISPR to be independent unlike other tools that require to be paired with other enzymes. CRISPR can also be easily matched with tailor-made guides of RNA sequences that are designed to lead them to their DNA targets; tens of thousands of these sequences have already been made and are available to the research community. CRISPR-Cas 9 can also be used to target multiple genes simultaneously when compared to the other tools available, which could only target one. However, CRISPR is still under evaluation for safe and effective use on humans.
How Can CRISPR Help?
With CRISPR, researchers can permanently modify genes in living cells and organisms. Research suggests that CRISPR-Cas9 can be used to target and modify mutations in the human genome in an effort to treat genetic disease. CRISPR genome editing also allows scientists to quickly create animal models and cell models; which could be used to accelerate research into diseases such as cancer and mental illness.
The Future of CRISPR in curing Genetic Diseases
Scientists at Harvard and MIT discovered that a cure for obesity can be found through a tiny tweak in your DNA to cause the metabolism to burn up excess fat. The pounds were thought to have come from overeating and lack of exercise but genetics also play a major role, especially the variant of the FTO gene, also known as the obesity gene, which can be found within people with higher BMIs. The researchers have found that the FTO gene turns on two other genes that stop fat being burned up and plan to turn off these genes through CRISPR by snipping out the code and replacing it with the correct sequence.
Genetically Editing Man's Best Friend
Chinese scientists are using gene editing to produce customized dogs and were successful when they created a beagle with twice the amount of muscle mass. The ability to change the dog's muscle mass leads the way for endless possibilities as dogs have similar metabolism, physiology, and anatomy as humans. With genome editing, scientists are able to disable genes or rearrange their DNA to change the composition of organisms, specifically canines in this scenario.
Layla's Story
Layla was a one year old that was dying from acute lymphoblastic leukemia after all other treatments had failed and in a desperate attempt to save their daughter's life, her parents asked for any other treatments possible, gene editing was given as a last alternative. Within a month, the cancerous cells within her bone marrow had been killed off and it is suspected that she is fully cured as there is no sign of the cancer returning.
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Join the Conversation about CRISPR
On August 22-25, 2018, there will be a panel discussion, two poster sessions, and six oral sessions about CRISPR with the topics of DNA damage and repair, CRISPR biology and function, CRISPR tools, CRISPR screening, disease correction, gene drive, plant and agriculture, and other applications.