Imagine if you could isolate the segment of human DNA that is directly responsible for causing a genetic disorder such as down syndrome, cystic fibrosis, or Tay-Sach’s disease, and ‘fix’ it. Believe it or not, the science is here. It may still be in its earliest stages, but researchers feel such tasks are definitely possible, thanks to procedures such as CRISPR gene editing.
What is CRISPR Gene Editing?
CRISPR is an acronym for Clustered Regularly Interspaced Short Palindromic Repeats. In layman’s terms, it’s the short repetitions of base sequences found in an organism’s DNA. In certain bacteria, these repeat sequences were found to have ‘spacers’ in between. When the bacteria became infected by a virus, it released a protein that effectively ‘cut’ the strand, protecting itself from further infection. This protein, called a nuclease, was later nicknamed ‘CAS,’ which means it’s CRISPR-Associated.
Once scientists discovered this breakthrough system of fending off infection, they began building upon it. As a result, they now have early forms of the tools needed to effectively ‘edit’ the human genome. Once these tools have been tested and perfected, it may be possible to eradicate some of humanity’s most painful and debilitating diseases.
What Is CRISPR/Cas?
When you hear about a study that involves CRISPR/Cas research, this means it used CRISPR gene editing to manipulate a Cas nuclease. In 2012, researchers discovered they could guide a Cas nuclease to any DNA strand using ribonucleic acid, causing dissection of the strand. Using this new technology, they are now able to sever a DNA strand in a desired location. Better — they can insert new DNA into the strand at that location — effectively removing and replacing genes that cause mutation, even in human cells.
How Could CRISPR/Cas Help Cancer Patients?
Gene-editing research is already being used to help patients recover from certain forms of cancer. In these procedures, doctors harvest T-cells from the patient and genetically modify them to contain a Chimeric Antigen Receptor, or CAR. Once infused back into the patient, the special CAR-T cells hunt down and kill existing cancer cells.
However, with the advancement of CRISPR/Cas technology, doctors could harvest cells from healthy patients, modify them, and store them to treat future patients. CRISPR/CAS could also destroy long sequences of mutated DNA and replace them with healthy sequences, creating positive outcomes for cancer patients. And it’s entirely customizable, which makes it easier to target specific locations along the strand of infected DNA.
What Does the Future Hold for CRISPR/Cas Technology?
Despite the many wonderful possibilities that await us with the advancement of CRISPR gene editing, the process does still have limitations. Sadly, at the current time, it’s not 100-percent effective. This means there’s no guarantee it will work every time. There are also limitations in how much CRISPR/Cas material can be delivered to target cells at one time. This procedure can also result in off-target applications, which could potentially be detrimental to the patient.
Still, cures for some of humanity’s worst ailments could be just around the corner. And it’s all thanks to the dedicated scientists who are working to improve the efficiency and effectiveness of CRISPR gene editing.