CRISPR-Cas9; the future of medicine

Ben Ortega, Editor

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It seems like every day, scientists are discovering new things and coming up with new technologies to propel the human race into the future. Relatively recently, a genome-editing tool called CRISPR-Cas9 was discovered, adapted, and adopted into the scientific world.

There are many gene editing techniques readily available to scientists, but CRISPR-Cas9 stands out thanks to its enhanced speed, affordability, simplicity, versatility, and precision compared to other techniques and technologies. CRISPR has generated great excitement in the scientific world and has massive potential to serve us in many aspects of our lives. It is a great tool for geneticists and other scientists to alter or delete unwanted sections of a DNA sequence and add more desirable ones.

“Operationally, you design a stretch of 20 [nucleotide] base pairs that match a gene that you want to edit,” said George Church, a genetics professor at Harvard Medical School, explaining how CRISPR works. “Then the RNA plus the protein [Cas9] will cut — like a pair of scissors — the DNA at that site, and ideally nowhere else,” he explained.

CRISPR-Cas9 has two molecular components. An enzyme called Cas9 that is used to cut specific sections of a DNA sequence and a 20 base long piece of predesigned guide RNA that acts as a form of transport for the Cas9 enzyme to have it cut the desired part of the DNA sequence. The two molecules combined are capable of severing any predetermined section in a DNA strand, creating an extremely functional partnership.

CRISPR-Cas9 has the potential to revolutionize genetic science and medicine altogether. It has been used to cure embryonic genetic diseases, cure HIV in different organisms, target cancer sources in organisms, delay cancerous growth, edit Huntington’s disease out of mice, and prevent genetic and communicable diseases. In the foreseeable future, scientists expect to be able to successfully treat cancer, hepatitis, cystic fibrosis, sickle cell anemia, heart disease, mental diseases, and other complex diseases. It also has other potential uses including altering crops, plants, and animals for desirable traits, creating more efficient biofuel, creating vaccines, and terminating harmful diseases altogether by destroying their reproductive capabilities.

Even though CRISPR is groundbreaking, it is not perfect. The technology has not been approved for human trials yet, mostly because it is not completely successful all the time. It does have its limitations, and errors, potentially devastating if they were to occur in a human genome, do happen. “I think the biggest limitation of CRISPR is it is not a hundred percent efficient,” said Church.

Although human trials aren’t currently an option, scientists are optimistic as to what CRISPR can do for the human race, but limits must be set. There is very little ethical debate on CRISPR usage on somatic cells (non reproductive cells), but t

A computerized diagram of the Cas9 gene-editing enzyme demonstrates it interacting with and RNA guide and its target DNA. (Photo via McGovern Institute of Brain Research at MIT)

here are restrictions on editing germ cells (reproductive cells), the germline, and embryos.

David Baltimore, a Nobel Prize winner for medicine, other scientists, and ethicists said that germline editing raises the possibility of unintended consequences for future generations “because there are limits to our knowledge of human genetics, gene-environment interactions, and the pathways of disease (including the interplay between one disease and other conditions or diseases in the same patient).”

Regardless of its current limitations, CRISPR-Cas9 is an exciting piece of technology at our disposal with great potential and many usages. Maybe soon we will all be enhanced and live longer, healthier lives thanks to CRISPR-Cas9.