Home Health Gene therapy for 8 children to treat a rare serious metabolic disease: they are doing well

Gene therapy for 8 children to treat a rare serious metabolic disease: they are doing well

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Strengthened by the long experience on other genetic diseases, the researchers of the SR-Tiget they managed to effectively correct the genetic defect responsible for the syndrome, so much so that, two years after the treatment, all the children involved in the study are well and have reached important milestones in their development.

“The positive effects of the therapy were seen soon – explains Maria Ester Bernardo, head of the San Raffaele pediatric bone marrow transplantation unit. – Their cells quickly began to produce large quantities of the enzyme, which cleaned up organs. and tissues from accumulated toxic metabolites. We observed the progressive acquisition of new motor and cognitive skills typical of their age, as well as excellent growth in height and a reduction of other typical symptoms of the syndrome such as joint stiffness and corneal opacity “.

But “we will have to continue to observe these children to verify that the positive effects continue over time”. The path, underlines Alessandro Aiuti, deputy director of SR-Tiget and full professor of Pediatrics at the Vita-Salute San Raffaele University, who coordinated the study, “is still long but it is encouraging that the development times of these therapies are shortening. thanks to the experience accumulated over the years “.

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