THE CANDLE: Gene editing could be a positive force in fighting disease | News

I like to focus on the positive and there is no shortage of stories to write.

Gene editing is changing everything from basic research to medical and agricultural advances that are revolutionizing these areas of research.

If I had to name another area that is progressing just as rapidly and has similar power, it would be the rise of artificial intelligence. However, that’s a topic for another day.

A recent article in the New England Journal of Medicine offers hope for children suffering from a rare disease that essentially destroys their immune system.

The title of this article is “Lentiviral gene therapy for Artemis-deficient SCID”. Although the trial was small, the results were shocking. A child born with this version of her SCID, which represents severe combined immunodeficiency, has a mutation in a single gene on her chromosome 10, labeled DCLRE1C.

This gene is responsible for making a protein called Artemis.

Artemis is an enzyme in the nucleus called an endonuclease. The normal version of this Artemis protein is essential for V(D)J recombination that occurs during B and T cell development. This protein rearranges gene segments so that B and T cell receptors are highly diverse. Receptors allow one day to respond to a wide variety of potential pathogens.

The mutated gene does not produce a “normal” Artemis protein.

This undermines the diversity of B and T cells a child can create, leaving them vulnerable to almost anything.

Children with Artemis-SCID need a healthy, matched donor to receive a bone marrow transplant in order to survive. Still, this version of her SCID doesn’t respond as well to bone marrow transplants as other versions.

If the error could be corrected from the child’s cells, no donor would be needed and rejection would not be an issue.

This is exactly what this team of researchers did.

They harvested stem cells from the bone marrow of 10 patients aged 18 months to just over 4 years old.

The researchers used a virus to infect stem cells with the “correct” version of the DCLRE1C gene.

Using a modified virus to deliver the desired payload simply hijacks what many viruses are originally good at.

Think of them like lab-engineered Trojan horses that are used to carry the necessary genes into the cell.

Once the virus had modified the cells to carry the “good” genes, they were re-injected into the patient.

In just 12 weeks, all 10 children produced functioning B and T cells.

All 10 of them are doing well every day.

4 out of 10 regained all immunity and are believed to have been completely cured of the dreaded disease.

If you’re ready to cry tears of joy, watch this 5-minute video from the University of San Francisco research team.

This video covers the story of HT, the first Navajo boy to receive this treatment.

Before you look at it, I’ll prepare some Kleenex.

Dr. Jack Brown is Chair of the Science Department at Paris Junior College. His scientific articles are published every other Sunday.