Emirates News Agency – UAE pediatric care experts discuss recent findings in research and gene therapy for rare diseases

22nd June, 2022 (WAM) — Researchers from the Zayed Center for Research into Rare Disease in Children in London recently shared their cutting-edge work during two podcasts.

Waseem Qasim, professor of cell and gene therapy at the Institute of Child Health and Great Ormond Street Hospital, was featured on Crowd Science, a BBC World Service podcast, in an episode titled ‘How Far Does Editing of genes could she go?”.

Professor Qasim, whose team at the Zayed Center for Research focuses on developing science and technology to make treatments for rare and hard-to-treat leukaemias, explained how gene editing is currently being used and what might be possible in the future.

“We can now engineer immune cells to attack leukemia more specifically. We can collect cells from the patient or a healthy donor, take them to a lab, select the immune cells, and as various pieces of technology have merged – or converged – it is now possible to reprogram these cells and introduce new genes into them to give them new properties,” Qasim noted.

“We try to rearm these cells and give them receptors on their surface, which can select – very specifically – for target cells that they recognize as dangerous or abnormal.”

Dr Amy McTague, Academic Clinical Consultant, Principal Investigator at UCL GOS Institute of Child Health, focuses her research on early onset epilepsies. She was featured on the educational podcast “Epilepsy Sparks Insights”, hosted by Torie Robinson.

Dr. McTague explained how her work helps babies and families with the diagnosis and treatment of babies with genetic epilepsy. She described her team’s work in an international genomics collaboration called the Gene Step Study, which includes SickKids Toronto, Melbourne Children’s Hospital and Boston Children’s Hospital. It is focused on rapid testing for babies and their parents and has had “real impacts for families”.

She continued: “Doing [the research project] internationally has been very exciting – and quite challenging too! But we hope to show that even across different healthcare systems, having the diagnosis earlier in the patient journey can make a big difference in the patient and parent experience as well as in the management of epilepsy.” .

Looking to the future, Dr. McTague shared excitement about tailored treatments that address genetic abnormalities rather than treating seizures or movement disorders. Early diagnosis means patients can be moved more quickly to the appropriate pathway for personalized treatment, maximizing outcomes and life chances for children.

The Zayed Center for Research is a partnership between Great Ormond Street Hospital (GOSH), University College London and Great Ormond Street Hospital Children’s Charity, and was made possible by a generous donation of £60 million in 2014 by HH Sheikha Fatima bint Mubarak, President of the General Women’s Union, President of the Supreme Council for Maternity and Childhood and Supreme President of the Family Development Foundation.

All research conducted at Great Ormond Street Hospital is supported by support from the NIHR GOSH Biomedical Research Center.

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