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Will CRISPR Bring Hope or Turmoil?

Written by Monica Savaglia
Posted August 6, 2019

Gene editing. For some that’s a scary term, and for others it’s a beacon of hope.

With most technology, there are promises of it benefiting the future, but at the same time there’s always a lot to consider when dealing with a breakthrough technology. That’s why people are treading lightly on the topic of gene editing.

In December 2018, a Chinese scientist made a very bold claim when he admitted to secretly editing the genes of embryos. Not only that, but he claimed that he implanted gene-edited embryos into seven couples who were undergoing IVF, and two of those couples became pregnant from IVF. One of those couples ended up giving birth to twins. He Jiankui, the Chinese geneticist who led the gene-editing trial, said those twins were given natural resistance to the human immunodeficiency virus (HIV) infection.

The breakthrough technology, CRISPR-Cas9, has been seen as a useful tool for the future to help or even cure patients. It has created a way to operate on DNA by either adding a much-needed gene or disabling one that’s causing the problem.

However, the technology requires a lot of trials and tests before it is considered safe and ethical to use to edit the genes of embryos. The tool is still fairly new. Of course, in the future it could be possible to safely follow through with this type of procedure. But right now, it’s too soon. There’s still a lot to learn from the technology.

On July 26, the World Health Organization, the leading authority in global public health, released a statement that discussed its qualms with the practice of gene editing in humans. Dr. Tedros Adhanom Ghebreyesus, WHO Director-General, said, “It would be irresponsible at this time for anyone to proceed with clinical applications of human germline genome editing.” (Germline gene editing is where the modified DNA is passed on from one generation to the next.)

The WHO has requested that regulatory and ethics authorities in all countries put a stop to and deny approvals of gene-editing experiments that would lead to babies being born with gene-edited DNA.

With that being said, CRISPR-Cas9 is starting to be used in adults to treat deadly diseases. The most important thing to recognize is that the patient would be the only one affected by the gene-editing procedure and that it wouldn’t be passed to future generations. At the moment, that’s the biggest issue.

Like I mentioned earlier, the technology does aim to help out adult patients. And those patients are optimistic about the possible outcomes.

CRISPR Human Trials Are Happening Right Now

The first-ever human study in the U.S. will use CRISPR inside the body.

Up to 18 people aged three and up will be part of this study. The study’s goal is to treat a heritable eye disorder called Leber congenital amaurosis (LCA) that leads to complete loss of vision. It’s the leading cause of inherited blindness in children. 

The pharmaceutical companies Editas Medicine and Allergan will be attempting to address a variant of LCA called LCA 10. These companies plan to use CRISPR to supply a healthy version of a gene the patients lack through subretinal injection.

Leber is one of the most common causes of inherited childhood blindness. It occurs in about two to three of every 100,000 births. Researchers believe that with CRISPR, they have the opportunity to cure or treat this and other genetic diseases that currently have no treatments.

This isn’t the only groundbreaking trial that’s happening with CRISPR. Recently, doctors in the U.S. have used CRISPR to treat a patient with a genetic disorder.

Globally, sickle cell affects millions of people. There are about 100,000 people in the U.S. who have the disease. The disease is brought on by a genetic defect that causes bone marrow to produce a defective protein that makes blood cells that are sickle-shaped — they are hard and sticky. Those sickle-shaped cells get stuck inside blood vessels and are unable to carry oxygen normally. 

With this specific study, doctors have decided to use cells from the patients’ own bone marrow that have been genetically modified with CRISPR to help produce a protein that’s only made by fetuses and babies a short time after their birth. This protein will hopefully take the defective protein’s place and allow the patients to live normally with a non-defective protein. 

Boston’s Vertex Pharmaceuticals (NASDAQ: VRTX) will be leading the study with CRISPR Therapeutics (NASDAQ: CRSP).

Dr. David Altshuler, executive vice president, global research, and chief scientific officer at Vertex, said, “It’s exciting to see that we might be on the cusp of a highly effective therapy for patients with sickle cell.”

Only time will tell if these studies become successful. I’m really hoping they do. The day we have the ability to alter genes so people don’t have to live their lives in pain and medical debt will be a good day.

I think there is a lot that gene editing can offer for the future, and it’ll definitely pay off for both the companies and patients involved. 

Until next time,

Monica Savaglia Signature Park Avenue Digest

Monica Savaglia
Editorial Director, Park Avenue Digest

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