Health and Me

Rachel V Thomas

New gene-editing tech corrects Canada teen’s DNA, cures rare disease in world-first

New gene-editing tech corrects Canada teen’s DNA, cures rare disease in world-first

In a world first, doctors have corrected the DNA of a teen from British Columbia, Canada, using new gene-editing technology under clinical trial, and cured him of his rare disease, raising hopes for scores of patients with such inherited diseases around the globe.

A new paper published recently in the New England Journal of Medicine described Ty Sperle, 19, as the first person to be cured of chronic granulomatous disease (CGD) -- a genetic condition where the body’s white blood cells do not work properly, increasing his risk of infectious disease -- by a treatment known as “prime editing”.

Ty was diagnosed with the condition at the age of five and has since been on pills, antibiotics, and antifungals. Yet, skin and soft-tissue infections, as well as an invasive bacterial infection in the lung, were quite frequent in his life.

“We have an immune system that I like to think of as a suit of armor that protects us, and because of Ty’s issue, he really had a big hole in that suit of armor,” Dr. Stuart Turvey, a pediatric immunologist at B.C. Children’s Hospital was quoted as saying in Global News.

"And so different bacterial or fungal infections could sneak in really at any time and cause serious or even life-threatening infections. So, it’s a tough disease to live with. People with this disease don’t live long, healthy lives," he added.

<h2>The Landmark Gene-Editing Technology </h2>

Ty became the first person to take part in the clinical trial, set up by US-based Prime Medicine, where doctors utilized the technology to correct an error in the DNA of patients affected by CGD. 

In the trial, Ty's blood stem cells were drawn and enriched before being treated with the gene editing product.

"The product contains the necessary components to target and correct the mutation in the gene responsible for the condition, NCF1," the B.C. Children’s Hospital shared in a statement. 

Ty was discharged after spending 24 days in hospital care. Follow-ups revealed that his immune system’s antimicrobial activity remained even durable six months later. 

“When we got the call from Dr. Turvey, I was very excited as there were no cure options for me at that time,” Ty said. 

“It was nerve-wracking for me as I was the first patient to volunteer for this procedure, and it seemed I was the only one, so it was scary," he said.

"I am thrilled with the result because I am cured of CGD and can live my life fully.” 

CGD is an inherited disease that severely impacts the immune system and affects approximately one in 200,000 children. 

Turvey, who treated Ty for more than a decade, said that the rare genetic disease leaves patients susceptible to infections that can turn serious, even fatal. 

People with the condition have a dramatically reduced ability to fight illnesses and are extremely vulnerable to inflammatory conditions and bacterial and fungal infections. 

While other treatments, such as hematopoietic stem cell transplantation, or a bone marrow transplant, can help patients with CGD, they work only in the case of an optimal healthy donor.

Health Conditions A-Z

health

BC teen

gene editing

immunity

white blood cells

immune system

Genetic Disorders

chronic granulomatous disease

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In a world first, doctors have corrected the DNA of a teen from British Columbia, Canada, using new gene-editing technology under clinical trial, and cured him of his rare disease, raising hopes for scores of patients with such inherited diseases around the globe. A new paper published recently in the New England Journal of Medicine described Ty Sperle, 19, as the first person to be cured of chronic granulomatous disease (CGD) -- a genetic condition where the body’s white blood cells do not work properly, increasing his risk of infectious disease -- by a treatment known as “prime editing”. Ty was diagnosed with the condition at the age of five and has since been on pills, antibiotics, and antifungals. Yet, skin and soft-tissue infections, as well as an invasive bacterial infection in the lung, were quite frequent in his life. “We have an immune system that I like to think of as a suit of armor that protects us, and because of Ty’s issue, he really had a big hole in that suit of armor,” Dr. Stuart Turvey, a pediatric immunologist at B.C. Children’s Hospital was quoted as saying in Global News. "And so different bacterial or fungal infections could sneak in really at any time and cause serious or even life-threatening infections. So, it’s a tough disease to live with. People with this disease don’t live long, healthy lives," he added.The Landmark Gene-Editing Technology Ty became the first person to take part in the clinical trial, set up by US-based Prime Medicine, where doctors utilized the technology to correct an error in the DNA of patients affected by CGD. In the trial, Ty's blood stem cells were drawn and enriched before being treated with the gene editing product. "The product contains the necessary components to target and correct the mutation in the gene responsible for the condition, NCF1," the B.C. Children’s Hospital shared in a statement. Ty was discharged after spending 24 days in hospital care. Follow-ups revealed that his immune system’s antimicrobial activity remained even durable six months later.  “When we got the call from Dr. Turvey, I was very excited as there were no cure options for me at that time,” Ty said. “It was nerve-wracking for me as I was the first patient to volunteer for this procedure, and it seemed I was the only one, so it was scary," he said."I am thrilled with the result because I am cured of CGD and can live my life fully.”  What Is CGD? CGD is an inherited disease that severely impacts the immune system and affects approximately one in 200,000 children.  Turvey, who treated Ty for more than a decade, said that the rare genetic disease leaves patients susceptible to infections that can turn serious, even fatal. People with the condition have a dramatically reduced ability to fight illnesses and are extremely vulnerable to inflammatory conditions and bacterial and fungal infections. While other treatments, such as hematopoietic stem cell transplantation, or a bone marrow transplant, can help patients with CGD, they work only in the case of an optimal healthy donor.