UAE pediatric care experts discuss recent research discoveries, gene therapy for rare diseases

UAE pediatric care experts discuss recent research discoveries, gene therapy for rare diseases.

LONDON, TGO: Researchers based at the Zayed Centre 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 could gene editing go?”.

Professor Qasim, whose team at the Zayed Centre for Research focuses on developing the 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 leukaemia more specifically. We can collect cells from the patient or a healthy donor, take them to a laboratory, pick out the immune cells and as various bits of technology have merged – or converged – it is now possible to reprogram those cells and introduce new genes into them to give them new properties,” Qasim noted.

“We are trying to re-arm these cells and give them receptors on their surface, which can pick out – very specifically – target cells that they recognize as being dangerous or abnormal.”

Dr. Amy McTague, Clinical Consultant Academic, Principal Research Fellow 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 discussed how her work helps babies and families when it comes to the diagnosis and treatment of babies who suffer from genetic epilepsy. She described her team’s work as part of an international genomic 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 as well! But we’re hoping to show that even across different healthcare systems having the diagnosis earlier in the patient journey can make a big difference to the patient and parents’ experience as well as the management of the epilepsy”.

In terms of the future, Dr. McTague shared the excitement around tailored treatments which address genetic abnormalities rather than treating the seizures or movement disorder. Early diagnosis means patients can be moved into the appropriate pathway for a tailored treatment more quickly, maximising the outcomes and life chances of children.

The Zayed Centre 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 thanks to a generous ₤60 million gift in 2014 from H.H. Sheikha Fatima bint Mubarak, Chairwoman of the General Women’s Union, President of the Supreme Council for Motherhood and Childhood, and Supreme Chairwoman of the Family Development Foundation.

All research at Great Ormond Street Hospital is underpinned by support from the NIHR GOSH Biomedical Research Centre.