Stem-Cell Gene Therapy Shown to Restore Immunity for Youths with Immunodeficiency Disorder
Marie de Vera | | Dec 06, 2015 07:41 AM EST |
(Photo : gettyimages.com) Stem cell therapy may be just what many young patients with immunodeficiency disorder need.
A new research has revealed that older children and young adults with X-linked severe combined immunodeficiency (SCID-X1) can undergo gene therapy to improve their condition.
According to an UPI article, the National Institute of Allergy and Infectious Diseases reports a process which includes the extraction, genetic correction and reinsertion of a patient's bone marrow to aid in improving their immune systems overtime.
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The treatment begins by extracting stem cells from the patient's bone marrow. Scientists then use a lentiviral vector to deliver a normal IL2RG gene to the cells.
After a low-dose chemo, the cells were then implanted back into the patient, which assist the stem cells establish themselves and start making new blood cells.
SCID-X1 is a rare inherited disorder of the immune system caused by inactivating mutations in the IL2RG gene, which is vital in creating protein cells required for a strong and healthy immune system. Almost exclusively the disease occurs in males and a person born with this gene disorder is extremely susceptible to persistent and recurrent infections caused by a certain bacteria and organisms that usually cause very serious or life-threatening illnesses.
Two initial participants were given therapy and one successful administration from the specialized treatment was observed out of the two participants. The individual is reported to be continuously improving, 3 years after receiving the gene therapy.
The other patient died of a pre-existing lung condition despite making improvements in immunity, two years after receiving the treatment, according to Medical Daily.
The agency is currently monitoring three other individuals between the ages of seven years old and 24 who are showing significant improvement after going through the bone marrow treatment three to six months ago. They are still being supervised as the study pushes through on recruiting new patients between ages 2 and 20.
In the coming 57th Annual Meeting and Exposition taking place in Orlando, Florida on Sunday, an in-depth discussion from the NIAID's findings will be provided.
Tagsstem cell, genes, immunodeficiency, SCID-X1, Theraphy, immune system, Research, Chemo, Bone Marrow, NIAID, stem cell therapy Immunodeficiency Disorder
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