MAR 18, 2021 3:43 AM PDT

Gene Therapy Benefits Can Last Decades

WRITTEN BY: Carmen Leitch

Genetic diseases arise from inborn errors in gene sequences, and researchers have long sought ways to correct those errors. There have been some instances in which scientists and clinicians have successfully treated a human patient with gene therapy. Researchers have now followed up on a group of gene therapy recipients and found that the benefits of their treatment have lasted for decades. The work has been reported in Nature Communications.

Image credit: CC0

These individuals had a form of severe combined immunodeficiency (SCID), which makes people highly susceptible to infection, and has been referred to as 'bubble boy disease.' The most common form of the disorder is X-linked SCID (SCID-X1), which is caused by mutations in a gene called  IL2RG. This gene plays an essential role in the development of several important types of immune cells that help fight infection: T and natural killer lymphocytes, and nonfunctional B lymphocytes. People with SCID are left without much of an immune system.

Blood stem cells are also known as hematopoietic stem cells. Normally, they produce all the cells that are found in the blood including red and white blood cells. SCID-X1 gene therapy involves collecting blood stem cells from patients. A correct version of the IL2RG gene is then carried in a non-pathogenic virus to the inside of these patient blood stem cells. Chemotherapy is used to remove some of the dysfunctional blood stem cells from the patient, and the modified cells are then returned to the patient in their place.

Scientists have monitored five recipients of this treatment for three to eighteen years afterward; blood samples were collected to determine the levels of various cell types and biomarkers. This study showed that even though the transplanted stem cells were cleared from the patients, the correct cells were still forming and the patients had been cured of their disease.

This work indicated that the 'autologous transplant' of the patient's own corrected cells generated a continuous supply of healthy immune cells, and a normal immune system.

The researchers suggested that this gene therapy has enabled the thymus, where T cells develop, to maintain a repository of the right kind of progenitor cells that can produce new T cells. More work will be needed to understand how that happens.

Sources: AAAS/Eurekalert! via University College London, Nature Communications

About the Author
BS
Experienced research scientist and technical expert with authorships on over 30 peer-reviewed publications, traveler to over 70 countries, published photographer and internationally-exhibited painter, volunteer trained in disaster-response, CPR and DV counseling.
You May Also Like
OCT 07, 2022
Earth & The Environment
United Nations Genetic Diversity Target Deadline Has Passed
OCT 07, 2022
United Nations Genetic Diversity Target Deadline Has Passed
In a recent study published in Science, an international team of researchers led by Stanford University examine how habi ...
OCT 07, 2022
Genetics & Genomics
Epigenetic Marks Can Pass Down to Multiple Generations
OCT 07, 2022
Epigenetic Marks Can Pass Down to Multiple Generations
The earliest studies of genetic inheritance were very straightforward - Gregor Mendel documented the passage of certain ...
OCT 18, 2022
Clinical & Molecular DX
New Mitochondrial Disease Identified in Identical Twins
OCT 18, 2022
New Mitochondrial Disease Identified in Identical Twins
A new mitochondrial disease has been identified after a pair of identical twins were showing an unusual symptom. Despite ...
NOV 10, 2022
Neuroscience
HUSH - A Complex That Silences Genes & Helps Control Brain Architecture
NOV 10, 2022
HUSH - A Complex That Silences Genes & Helps Control Brain Architecture
The HUSH (human silencing hub) protein complex is known to silence genes, and researchers have thought that it could be ...
NOV 10, 2022
Microbiology
TB Germ That Can Move to the Bones Resembles Ancient Strain Found in Mummies
NOV 10, 2022
TB Germ That Can Move to the Bones Resembles Ancient Strain Found in Mummies
Mycobacterium tuberculosis is the pathogenic bacterium that causes TB, which is still a major public health problem in m ...
DEC 04, 2022
Genetics & Genomics
Aging Processes may be Altered by Early-Life Experiences
DEC 04, 2022
Aging Processes may be Altered by Early-Life Experiences
The way certain genes are expressed in aging organisms seems to be significantly impacted by events that happen earlier ...
Loading Comments...