Gene therapy is applied to prevent type 1 diabetes in newborns

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Gene therapy is applied to prevent type 1 diabetes in newborns
Gene therapy is applied to prevent type 1 diabetes in newborns

MedPage Today announced a new treatment for some infants in the first month of their life to potentially prevent type 1 diabetes (T1D). The treatment is for a specific genetic defect, the DR3/4 genotype. The treatment consists of introducing probiotics (live bacteria) into the infant’s digestive system. The goal is to prevent an autoimmune attack on the islet of the pancreas through a series of interactions of the bacteria in the gut. This treatment reduces the risk of the infant developing T1D.

Salynn Bolyes, contributing writer, for MedPage Today, released the article that is titled “Probiotic Tx May Lower Risk for Islet Autoimmunity. Supplements during first month of life may help kids with genetic risk for T1D.” A defect in the DR3/4 genotype presents the highest risk for an infant to develop T1D.

Ulla Uusitalo, PhD, of Morsani College of Medicine at the University of South Florida in Tampa was the lead author for the study. Uusitalo defined the major conclusion of the study to MedPage Today.

Certainly, we need more studies to confirm this, but it looks like probiotics may be protective against islet autoimmunity, but only in children with the highest risk for type 1 diabetes.

Uusitalo discussed the experiences of using probiotics in other countries.

While probiotic supplementation is considered safe in healthy infants, it is still not clear whether giving them very early in life can modify the infant gut microbiota trajectory and disease susceptibility.

In Finland, probiotics are given in supplement form to over half of infants and children in the first year of life. In Germany, probiotics are often added to baby formula, but fewer than 10% of newborns and babies in the U.S. receive probiotics.

An article in the Journal of the American Medical Association (JAMA) Pediatrics in December 2013 provided a review of study results of providing probiotics to infants to reduce colic. The NIH has provided a detailed description of the microbiome (gut bacteria) progression that leads to T1D in infants.

Probiotics have previously been studied as an approach to lessen colic, which is a painful condition in infants related to the intestines that results in extending crying. While some studies showed promise, the general uncertainty of meta-studies has confused the results. The National Institute of Health (NIH) concluded that there was evidence that showed value for probiotics to treat colic. The positive results for using probiotics to prevent development of T1D and colic may encourage other applications of probiotics in newborn infants.

Research in this area may yield substantial results in halting the development of diseases in infants by restoring the normal functioning of the immune system in the baby. As in cancer, specific actions to repair defective genes are a promising approach to actually curing a disease instead of masking symptoms. The applications will expand as the research continues.

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