CRISPR, meaning clustered regularly interspaced palindromic repeats, is a very powerful tool in genetics. It disrupts the normal sequence of repeating units of base pairs in a DNA molecule (a process known as gene editing) thereby, modifying the function of the gene. It is short for CRISPR/Cas9, a specialized structure of DNA with a protein associate which is an enzyme that acts like a pair of scissors capable of cutting DNA strands. CRISPR is a specialized DNA structure bearing “spacers” between the repeating units of base pairs.
This sequence of repeated base pairs forms the genetic code for the cells of the body. The code acts as a set of rules that influences the behavior of cells. A disruption in the normal sequence of these repeated base pairs, such as during a mutation, affects the cells’ normal function, leading to an abnormality which is often reflected in genetic disorders such as Down syndrome, Edward syndrome, etc.
Scientists have not stopped researching ways to effectively treat these genetic disorders, and so far, the discovery of the CRISPR technology seems like a promising method. However, the challenge with this method is the fact that it most often results in the deletion of large portions of DNA, which renders the method not completely effective in treating genetic diseases. This challenge once again pushed scientists to go into research to discover ways to overcome it while still maintaining the efficiency of the procedure. Recently, the team of scientists at Wake Forest Institute for Regenerative Medicine (WFIRM), made a discovery that reveals a way that overcomes the challenge.
What they did
The team realized that they could not alter the procedure to avoid the deletion of large portions of DNA, so instead, they found a way to maximize small deletions and minimize large deletions. After studying a variety of human cells and observing the genes they were interested in, they found that combining either DNA polymerase I or the Klenow fragment to the protein associate, Cas9, greatly reduced large DNA deletions, and most importantly, did not affect the efficiency of the procedure – gene editing was not affected.
This discovery is important for it uncovers a way to effectively treat disorders that are most often considered uncurable because they affect the controllers of life’s basic unit – genes. Finding a way around the once-held challenge of the CRISPR technology has paved the way for therapies that would be effective to treat genetic disorders by either disrupting disease-causing genes or fixing disrupted genes.
After much research, the discovery that reveals a way around the challenge of the CRISPR technology has finally been made, thanks to lead author Baisong Lu and his team. More research can still be done to reveal more efficient ways around this obstacle.