The latest development of ADA-SCID gene therapy in 90% of children with continued health after ten years of treatment

A few days ago, researchers at the University of California, Los Angeles reported on "Blood" related data on experimental gene therapy for severe combined immunodeficiency (ADA-SCID) children.

Severe combined immunodeficiency (ADA-SCID) is a rare and life-threatening genetic disease caused by mutations in the adenosine deaminase (ADA) gene

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During the period 2009-2012, a total of 10 children received the therapy, and the current results show that 9 of these children continue to be in stable condition and do not require enzyme replacement or bone marrow transplantation to support their immune system in the following years

At the same time, researchers have observed that the therapy has significant differences in the immune system among different individuals.

In terms of safety, studies have found that treatment in the stem cells of some children can disrupt genes related to cell growth.

In August of this year, the Kohn team and the research team of Great Ormond Street Hospital in London jointly reported the treatment results of the therapy in 50 ADA-SCID children.

In addition, it is worth mentioning that in 2016 the European Union approved the world’s first gene therapy for the treatment of ADA-deficiency severe combined immunodeficiency-the autologous stem cell in vitro gene therapy Strimvelis (currently developed by GSK, Telethon and Ospedale San Raffaele).

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"A decade after gene therapy, children born with deadly immune disorder remain healthy"