Science and medicine are incredibly amazing. Thanks to modern science and medicine, we now have lifespans far beyond our Dark Age predecessors, living until ripe old age and enjoying the fruits of our labour well into retirement.

One incredible aspect of science is gene editing, whereby scientists can edit the human genome. CRISPR, short for Clustered Regularly Interspaced Short Palindromic Repeats, is a gene-editing technology. For those wondering more about CRISPR, there are bsn to fnp online programs that let people get into the field and learn more about its practical application.

This helpful article will explore the CRISPR revolution and explain what it means for humanity.

What is CRISPR?

We’ve explained above what the acronym stands for. CRISPR allows scientists to modify the DNA of living organisms, including plants, animals, and humans. It acts like a pair of molecular scissors that can precisely target and cut specific DNA sequences, enabling researchers to change an organism’s genetic code.

What is Gene Editing?

Gene editing, also known as genome editing, refers to technologies that allow scientists to make specific changes to an organism, such as an animal, plant, or human’s DNA. These changes can involve adding, removing, or altering DNA sequences at particular locations in the genome. This technology is used in research labs to understand diseases better and is also being explored for therapeutic applications. Gene editing tools precisely cut DNA at a specific location. Once cut, scientists can remove, insert, or replace the DNA at that location for various results.

The DNA from only a single cell in your body contains your entire ‘genome’, which is all the genetic instructions for you to grow, develop, and function as a healthy human being. These genetic instructions are encoded in around 6 billion letters of DNA across 46 chromosomes that we inherit from our parents; we get 23 from each parent.

Instead of the 26 letters in the English alphabet, DNA consists of four letters, A, C, G, and T. In some cases, even a single letter alteration, mutation, or variant can cause the entire set of instructions to be misread, which can change the molecules your body produces and the way your body functions, resulting in genetic disorders and diseases.

Gene editing means altering one of those genetic instructions. This can include adding or deleting a gene, switching a gene on or off, or correcting a damaging variant that is causing the gene to be misread by the body. Correcting damaging variants via gene therapy could be the key to curing some of the hardest-to-treat diseases known to humanity.

How was CRISPR Discovered?

In 2012, researchers demonstrated that RNAs could be constructed to guide a Cas nuclease (Cas9 was the first used) to any DNA sequence in a genome. The so-called “guide RNA” can also be made so that it is specific to only that one sequence, improving the chances that the DNA will be cut only at that site and nowhere else in the genome. Further testing revealed that the system works quite well in all types of cells, including human cells.

Implications of CRISPR for Medicine

With CRISPR/Cas techniques, it’s easy to disrupt a targeted gene. In addition, if a DNA template is added to the mix, scientists can insert a new sequence at the precise spot in the gene that is desired. The method has profoundly changed biomedical research, as it greatly reduces the time and cost of developing animal models with specific genomic changes in mind.

Scientists now routinely use the CRISPR/Cas system for this purpose in mice in research. And for human diseases with a known mutation, such as cystic fibrosis or sickle cell anemia, it’s theoretically possible to insert DNA that corrects the mutation and cures the disease. There are clinical applications in human trials now, including groundbreaking research for engineering T cells outside of the body for CAR-T cancer therapy and for editing retinal cells for Leber’s congenital amaurosis 10, an inherited type of blindness.

CRISPR Breakthroughs

A team of scientists is now using CRISPR to develop a therapeutic approach that is direly needed for kids born with severe inherited immune disorders. At the moment, the main avenue for treatment for these disorders is a bone marrow transplant from a healthy donor. While this can provide a functional immune system, the transplants come with major side effects, and patients face lifelong immunosuppression treatment to stop their bodies from rejecting the donor cells. In contrast, gene editing via CRISPR could cure the condition.

The same team of researchers is ambitiously targeting more than 300 genetic conditions, including sickle cell anaemia and β-thalassaemia, and a large number of immunodeficiency and inflammatory disorders that primarily affect children, such as chronic granulomatous disease, several types of severe combined immunodeficiency, haemophagocytic lymphohistiocytosis, and various antibody deficiencies like X-linked hyper-IgM and agammaglobulinemia.

As research continues, and scientists learn more about CRISPR and gene editing, we’re likely to see rapid advancements in treatment and the possible cure of a multitude of genetic disorders and diseases, improving the quality of life for people suffering from these conditions, and possibly even cures.

The CRISPR revolution in science gives us the power of gene editing, and this breakthrough has real, practical applications for treating a large number of genetic disorders and diseases.