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An international collaboration between Great Ormond Street Hospital, the UCL GOS Institute for Child Health and Harvard Medical School has shown that the beneficial effects of gene therapy can be seen decades after the transplanted blood stem cells has been cleared by the body.
The research team monitored five patients who were successfully cured of SCID-X1 using gene therapy at GOSH. For 3-18 years patients blood was regularly analysed to detect which cell types and biomarker chemicals were present in their blood. The results showed that even though the stem cells transplanted as part of gene therapy had been cleared by the patients, the all-important corrected immune cells, called T-cells, were still forming.
Gene therapy s cellular benefits remain decades after treatment ANI | Updated: Mar 17, 2021 23:28 IST
Washington [US], March 17 (ANI): During an international collaboration recently between Great Ormond Street Hospital, the UCL GOS Institute for Child Health, and Harvard Medical School, it was found that the beneficial effects of gene therapy can be seen decades after the transplanted blood stem cells have been cleared by the body.
The research team monitored five patients who were successfully cured of SCID-X1 using gene therapy at GOSH. For 3-18 years patients blood was regularly analysed to detect which cell types and biomarker chemicals were present in their blood. The results showed that even though the stem cells transplanted as part of gene therapy had been cleared by the patients, the all-important corrected immune cells, called T-cells, were still forming.
Beneficial effects of gene therapy can be seen in SCID-X1 patients decades after treatment
An international collaboration between Great Ormond Street Hospital, the UCL GOS Institute for Child Health and Harvard Medical School has shown that the beneficial effects of gene therapy can be seen decades after the transplanted blood stem cells has been cleared by the body.
The research team monitored five patients who were successfully cured of SCID-X1 using gene therapy at GOSH. For 3-18 years patients blood was regularly analyzed to detect which cell types and biomarker chemicals were present in their blood. The results showed that even though the stem cells transplanted as part of gene therapy had been cleared by the patients, the all-important corrected immune cells, called T-cells, were still forming.
World-leading research institutions join forces to find better treatments for pediatric diseases
Ahead of Rare Disease Day (28 February), four leading children s research institutions on three continents are joining forces to decipher pediatric illnesses, including rare diseases, and find better treatments.
The four pediatric hospitals Boston Children s Hospital; UCL Great Ormond Street Institute for Child Health and Great Ormond Street Hospital (London); the Murdoch Children s Research Institute with The Royal Children s Hospital (Melbourne); and The Hospital for Sick Children (SickKids) in Toronto are working together to evaluate genomic data, clinical data from patients, and scientific and medical expertise to accelerate discovery and therapeutic development.
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Ahead of Rare Disease Day (28 February), four leading children s research institutions on three continents are joining forces to decipher paediatric illnesses, including rare diseases, and find better treatments.
The four paediatric hospitals Boston Children s Hospital; UCL Great Ormond Street Institute for Child Health and Great Ormond Street Hospital (London); the Murdoch Children s Research Institute with The Royal Children s Hospital (Melbourne); and The Hospital for Sick Children (SickKids) in Toronto are working together to evaluate genomic data, clinical data from patients, and scientific and medical expertise to accelerate discovery and therapeutic development.
The partnership, known as the International Precision Child Health Partnership (IPCHiP), is the first major global collaboration around genomics and child health. The founding partners anticipate that additional institutions will join the collaboration in the future.