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Research

Bringing Research to Life focuses on the research undertaken in partnership between Great Ormond Street Hospital (GOSH) and the UCL Institute of Child Health.

Our researchers aim to use their discoveries to help the young patients at GOSH as swiftly as possible. 

Find out more about the new research projects for cancer, rare diseases and gene therapy that they are currently working on.

Cancer

Cancer is the leading cause of death in children aged one to 14 in the UK. Even for those who overcome the disease, the side effects of harsh cancer therapy treatments can be very damaging in the long term.

  • Medulloblastoma Research documentsDr Jonathan Ham is researching how medulloblastoma cells work by studying the molecular biology of the disease at Great Ormond Street Hospital.
  • Neuroblastoma John Anderson (3A)Professor John Anderson's laboratory at Great Ormond Street Hospital is developing an innovative new treatment for children with neuroblastoma.
  • Acute lymphoblastic leukaemia Persis AmroliaProfessor Persis Amrolia is leading a new clinical trial at Great Ormond Street Hospital which focuses on acute lymphoblastic leukaemia.
  • Drug redeployment Jake The drug redeployment programme at Great Ormond Street Hospital aims to fast track the process of developing new leukaemia drugs.

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Rare diseases

Rare diseases are defined as those that affect less than one in 2,000 people. However, collectively, rare diseases are not rare. One in 17 people are affected by them. Seventy five per cent of rare diseases affect children, and there is a great need to understand and develop treatments for them.

  • Sanfilippo disease TobyDr Brian Bigger is developing the first ever treatment for Sanfilippo disease at Great Ormond Street Hospital.
  • Ciliopathies Phil Beales - BRTL researcherResearchers at Great Ormond Street Hospital are developing new treatments for ciliopathies – conditions affecting more than one in 200 children from birth.
  • Epilepsy and vitamin B6 Dr Philippa MillsAt Great Ormond Street Hospital, Dr Philippa Mills and her team are researching whether certain types of epilepsy can be treated with vitamin B6.

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Gene therapy

Researchers at GOSH are working on a life-saving technique called 'gene therapy' – the precise insertion of a working gene into a patient's DNA to replace or correct a faulty, disease-causing gene.

  • Gene therapy programme Bobby Gaspar and Adrian Thrasher (3A)Great Ormond Street Hospital and the UCL Institute of Child Health's gene therapy programme will help even more children with complex genetic conditions.