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Landmark study shows gene therapy a successful treatment for some with 'bubble boy' syndrome

TORONTO
A new landmark study examining treatment for a debilitating genetic disease known colloquially as bubble boy syndrome has found that gene therapy may provide a solution.
There are several types of severe combined immunodeficiency due to adenosine deaminase (SCID or ADA-SCID), but “bubble boy syndrome” refers to when affected children must be extra cautious and protected from germs and infections as their immune systems do not function properly.
The study followed 50 patients, 20 from the U.K. and 30 in the U.S. – including eight patients from Canada who had been referred by specialists to take part of the study south of the border. ....

United States , University Of California Los Angeles , City Of , United Kingdom , Donald Kohn , Etienne Blais , Anne Pham Huy , Pierre Blais , University Of California , Orchard Therapeutics Ltd , New England Journal , California Los Angeles , Eastern Ontario , Orchard Therapeutics , ஒன்றுபட்டது மாநிலங்களில் , பல்கலைக்கழகம் ஆஃப் கலிஃபோர்னியா லாஸ் ஏஞ்சல்ஸ் , நகரம் ஆஃப் , ஒன்றுபட்டது கிஂக்டம் , டொனால்ட் கோன் , அன்னே ஃப்யாம் ஹுய் , பியர் பிளேஸ் , பல்கலைக்கழகம் ஆஃப் கலிஃபோர்னியா , ஆர்சர்ட் சிகிச்சை லிமிடெட் , புதியது இங்கிலாந்து இதழ் , கலிஃபோர்னியா லாஸ் ஏஞ்சல்ஸ் , கிழக்கு ஆஂடேரியொ ,