DUBAI, 11th February, 2022 (WAM) As part of the UK Pavilion at Expo 2020 Dubai’s How will we Thrive? week program, Prof Claire Booth, Mahboubian Professor in Gene Therapy at UCL Great Ormond Institute of Child Health (UCL GOS ICH) and Consultant in Paediatric Immunology, Great Ormond Street Hospital (GOSH), was invited to give a virtual seminar to discuss her world-leading research in gene therapy.
GOSH has been at the cutting edge of the gene therapy field for more than 20 years, and much.
Gene Therapy for ADA-SCID Still Benefitting Patients after Two to Three Years
May 11, 2021
“I remember asking the doctor if my daughter was going to die,” said Chelsea Oakley, mother of Cora Oakley, a newborn who was diagnosed with ADA-SCID when she was just seven days old, in April 2017. “His response was, ‘I hope not.’ It was the darkest day of my life.”
Cora was the last child to enroll in a clinical trial of a gene therapy for ADA-SCID, or severe combined immunodeficiency due to adenosine deaminase deficiency. After receiving her own corrected cells in September 2017, Cora spent a month in the bone marrow transplant unit close to her family’s home. Observing the other young patients, the Oakleys witnessed what her daughter would have gone through if the experimental gene therapy hadn’t been an option.
Credit: Ann Johansson/UCLA Broad Stem Cell Research Center
An experimental form of gene therapy developed by a team of researchers from UCLA and Great Ormond Street Hospital in London has successfully treated 48 of 50 children born with a rare and deadly inherited disorder that leaves them without an immune system.
Severe combined immunodeficiency due to adenosine deaminase deficiency, or ADA-SCID, is caused by mutations in the ADA gene that creates the enzyme adenosine deaminase, which is essential to a functioning immune system. For children with the condition, even day-to-day activities like going to school or playing with friends can lead to dangerous, life-threatening infections. If untreated, ADA-SCID can be fatal within the first two years of life.
Researchers find gene therapy as potential cure to children born without immune system ANI | Updated: May 11, 2021 18:44 IST
California [US], May 11 (ANI): Researchers have developed a gene therapy that successfully treated 48 out of 50 children with a form of severe combined immunodeficiency that leaves them without an immune system.
The study led by an international team of researchers at Great Ormond Street Hospital (GOSH), and the University of California, Los Angeles (UCLA) - was published in the New England Journal of Medicine.
Severe combined immunodeficiency due to adenosine deaminase deficiency, also known as ADA-SCID, is a rare, life-threatening disease that prevents children from living a normal life. It is caused by mutations in the gene that creates the enzyme adenosine deaminase, which is essential to a functioning immune system.
Researchers have hailed a significant breakthrough in the treatment of a form of immunodeficiency (Lynne Cameron/PA)
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Researchers have made a “significant breakthrough” with a gene therapy that treats children with a form of severe combined immunodeficiency which leaves them without an immune system.
Severe combined immunodeficiency due to adenosine deaminase deficiency, also known as ADA-SCID, is a rare, life-threatening disease that prevents children from living a normal life.