DNA Gene Editing CRISPR
Medicine

The Innovation Of CRISPR Is Coming

New advancements in genomic editing technology that once promised to revolutionize the medical industry have finally been approved and are already transforming how we diagnose and treat diseases. Known as “CRISPR”, this DNA altering technique provides proof that tweaking the code of life is indeed possible, and that prompting life-changing results through its one-time treatment has now become a reality.

CRISPR, which stands for clustered regularly interspaced short palindromic repeats, is a revolutionary new technology that can be used to detect specific genes along segments of DNA, then alter this DNA to change how the genes behave. Furthermore, it can turn on and off the functionality of genes, without altering their sequence.

This one-time, intravenous technique promises huge advancements genome editing as well as our ability to diagnose and treat a wide range of diseases. The diagnostic properties of CRISPR also provide a deeper insight into how and why diseases develop, which will further open the doors to new remedies and cures.

The Innovation Of CRISPR Is Coming

First CRISPR Treatments FDA Approved

Casgevy became the first publicly available CRISPR treatment in the world when it was approved for use in the UK on Nov 16th 2023. In the U.S.A, the FDA approved version of the treatments, known as Exa-cel and made by Vertex and CRISPR Therapeutics, was made available on December 8th. Less than two weeks later on the 20th Dec., a third CRISPR treatment, known as lovo-cel and manufactured by Bluebird, was also approved.

These approvals were subsequently followed by the successful treatment of patients suffering from the genetic defect, sickle cell disease.

Sickle cell disease, is a condition that causes red blood cells to become sticky and crescent-shaped, which in turn leads to the cells clumping together, reducing their capacity to carry oxygen, and resulting in excruciating bouts of pain for the patient. Over time sickle cell disease progressively damages organs and eventually leads to death.

The only truly effective treatment to cure sickle cell is to perform a bone marrow transplant; an extremely invasive and painful procedure that still only offers between 42%-60% success rate.

Jimi Olaghere, one of the patients who received the new one-time medication, had previously been rushed to the ER on such regular occasion that the hospital had reserved a bed for him. Following the treatment however, Olaghere told reporters that the pain his sickle cell used to cause had almost gone completely, and that his, “quality of life has soared to new heights.”

Casgevy, represents one of the first CRISPR-based gene editors, and works by correcting problems in stem cells isolated from the patient’s bone marrow. It is then injected back into the body where the stem cells promote the production of healthy bloody cells that can deliver sufficient amounts of oxygen throughout the body once again.

CRISPR, or CRISPR-Cas9, was first unveiled in the journal Science in 2012, by Jennifer Doudna, Emmanuelle Charpentier, Martin Jinek, Krzysztof Chylinski, Ines Fonfara and Michael Hauer. Prior to this first paper, similar technologies such as zinc finger nucleases and TALENs (transcription activator-like effector nucleases) did exist, however CRISPR promised a technique that would be far more efficient and much cheaper; 11 years after the initial discovery, these claims have finally been proven correct by real-world cases.

Jennifer Doudna, a biochemist at the University of California, Berkeley, who shared the Nobel Prize in Chemistry in 2020 for her role in the development of the gene-editing technique.

“It’s the start of the era of CRISPR medicine. I think it suggests that we’re on the edge of real transformation in medicine,”

The CRISPR concept works by essentially slicing the DNA sequence at specific genetic markers, then inserting, or deleting, into other sequences. This can be used to switch on or off certain genes, and to correct defective ones; effectively changing an organism’s DNA to fight off infection or treat disease at its source.

There are however, a couple of downsides that may prevent Casgevy seeing widespread use. The cost of treatment is listed at 2.2million dollars per patient, and although it’s a one-time treatment, it still requires a lengthy hospital stay of up to 4-6 weeks.

There are also concerns that the slicing technique, which cuts both strands of DNA could cause adverse mutations, which could activate in cancer triggering genes. Fortunately, research looking into techniques that can slice single strands of DNA has already been underway since 2016. Scientists have been working to reengineer the scissor tool and reduce the size of the components to make the slicing technique more precise.