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AIDS virus used in gene therapy to fix 'bubble baby' disease - New Delhi Times - India's Only International Newspaper

May 11, 2021
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A gene therapy that makes use of an unlikely helper, the AIDS virus, gave a working immune system to 48 babies and toddlers who were born without one, doctors reported Tuesday.
Results show that all but two of the 50 children who were given the experimental therapy in a study now have healthy germ-fighting abilities.
“We’re taking what otherwise would have been a fatal disease” and healing most of these children with a single treatment, said study leader Dr. Donald Kohn of UCLA Mattel Children’s Hospital.
“They’re basically ‘free range’ going to school, doing normal things,” without the worry that any infection could become life-threatening, he said. ....

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AIDS virus used to fix 'bubble baby' disease through gene therapy

AIDS virus used in gene therapy to fix bubble baby disease
The one-time treatment involves using a disabled version of the AIDS virus to deliver a gene the kids lack to form an immune system.
Author: MARILYNN MARCHIONE AP Chief Medical Writer
Published: 6:20 AM EDT May 11, 2021
Updated: 6:20 AM EDT May 11, 2021
WASHINGTON A gene therapy that makes use of an unlikely helper, the AIDS virus, gave a working immune system to 48 babies and toddlers who were born without one, doctors reported Tuesday.
Results show that all but two of the 50 children who were given the experimental therapy in a study now have healthy germ-fighting abilities. ....

United States , United Kingdom , City Of , Donald Kohn , Kim Carter , Los Angeles , Josselyn Kish , Las Vegas , Stephen Gottschalk , California Institute For Regenerative Medicine , Us National Institutes Of Health , American Society Of Gene Cell Therapy , Jude Children Research Hospital In Memphis , Associated Press , Family Photos , Great Ormond Street Hospital , Jude Children , Research Hospital , New England Journal , American Society , Cell Therapy , California Institute , Regenerative Medicine , London Based Orchard , ஒன்றுபட்டது மாநிலங்களில் , ஒன்றுபட்டது கிஂக்டம் ,

Gene therapy uses AIDS virus to help children born with 'bubble baby disease'

Gene therapy uses AIDS virus to help children born with 'bubble baby disease'
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Gene Therapy Fixes Rare Type of Deadly Immune Deficiency

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A novel gene therapy for severe combined immunodeficiency from adenosine deaminase deficiency (ADA-SCID) restored immune cell counts without death or need for enzyme replacement therapy, researchers reported.
Survival without needing to re-initiate pegylated ADA enzyme replacement therapy, or have a rescue allogeneic hematopoietic stem cell transplantation (AHCT), was 95% to 97% at last follow-up at 24 or 36 months across three studies pooled together by Donald B. Kohn, MD, of the University of California Los Angeles, and colleagues.
Those rates compare favorably with the current standard of care, AHCT, for which overall survival of 65% to 88% and event-free survival of 56% has been reported, they stated in the ....

United States , Stephen Gottschalk , Donaldb Kohn , University Of California Los Angeles , American Society Of Gene , Jude Children Research Hospital In Memphis , California Los Angeles , New England Journal , American Society , Cell Therapy , Jude Children , Research Hospital , England Journal , Englj Med , ஒன்றுபட்டது மாநிலங்களில் , ஸ்டீபன் கொதிடஸ்சல்க் , பல்கலைக்கழகம் ஆஃப் கலிஃபோர்னியா லாஸ் ஏஞ்சல்ஸ் , அமெரிக்கன் சமூகம் ஆஃப் கீந் , ஜூட் குழந்தைகள் ஆராய்ச்சி மருத்துவமனை இல் மெம்பிஸ் , கலிஃபோர்னியா லாஸ் ஏஞ்சல்ஸ் , புதியது இங்கிலாந்து இதழ் , அமெரிக்கன் சமூகம் , செல் சிகிச்சை , ஜூட் குழந்தைகள் , ஆராய்ச்சி மருத்துவமனை , இங்கிலாந்து இதழ் ,

AIDS virus used in gene therapy to fix 'bubble baby' disease

AIDS virus used in gene therapy to fix bubble baby disease
MARILYNN MARCHIONE, AP Chief Medical Writer
May 11, 2021
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1of3This January 2020 photo provided by the family shows Josselyn Kish of Las Vegas. As a baby, Josselyn suffered rashes, painful shingles and frequent diarrhea, said her mother, Kim Carter. “Day care was calling me a couple times a week to come get her because she was always getting fevers.” After the gene therapy, “she was better right away,” Carter said. Now, “she rarely, rarely gets sick at all” and has been able to recover whenever she has. (Family photo via AP)Kim Carter/APShow MoreShow Less ....

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