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Research Review 2011/12

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UCL Institute of Child Health Street Ormond and Great Trust Hospital NHS Foundation Children for Design Manager UCL Institute Great Ormond Street Hospital Fourth floor of Child Health 40 Bernard Street 30 Guilford Street London WC1N 1LE London WC1N 1EH E [email protected] 020 7242 9789 www.ucl.ac.uk/ich Great Ormond Street Bengali Hospital for Children NHS Foundation Trust Great Ormond Street English London WC1N 3JH Translations, large print, Braille or audio versions of this report are available upon 020 7405 9200 request from the address above. www.gosh.nhs.uk French Traductions disponibles sur demande à l’adresse ci-dessus. Des versions en gros caractères, en braille ou audio sont également disponibles sur demande. Polish Tłumaczenia są do uzyskania na żądanie pod podanym powyżej adresem. Dokumenty w formacie dużym drukiem, brajlem lub audio są także do uzyskania na żądanie. Punjabi Somali Turjubaan ayaa cinwaanka kor ku qoran laga heli karaa markii la soo codsado. Daabacad far waa-wayn, farta indhoolaha Braille ama hab la dhegaysto ayaa xittaa la heli karaa markii la soo codsado. Tamil Written by Adam Kamenetzky. Designed and produced by Doulla Theodorou. Photography by Adam Laycock and Candice-Joelle Wordley. Printed by Hill and Garwood, using vegetable-based inks on Heaven 42. Turkish Thank you to everyone who was Talep edilirse yukarıdaki adresten 2011/12 Research Review interviewed for, or gave permission for çevirileri tedarik edilebilir. Talep edilirse, their picture to be used in this review, iri harflerle, Braille (görme engelliler için) as well as the many members of the veya sesli şekilde de tedarik edilebilir. UCL Institute of Child Health and Great Ormond Street Hospital staff Urdu who helped during its production. Research Please visit www.ucl.ac.uk/ich/research-ich or www.gosh.nhs.uk/research-and-innovation for an online version of this review. ReviewThe child first 2011/12 and always Sean is 10 years old and has a condition called nephrotic Zhen is three years old and is syndrome, which causes his staying on Lion Ward while he kidneys to fail. Earlier this is treated for B-cell lymphoma. year, Sean had both kidneys He has been here for two months, removed and was donated but recently got to go home for the a kidney by his dad. day to celebrate his third birthday! Contents 03 Director’s report 07 Chief Executive’s report 09 Division of Research and Innovation report Research 13 15 16 19 T-time for life-saving Beating leukaemia with the Building a foundation Pioneering treatments that use transplants best of modern biomedicine for healthy lungs the body’s own ability to heal Dr Waseem Qasim Professor Persis Amrolia Dr Sooky Lum Dr Paolo De Coppi 20 23 24 27 From an unknown disorder to Finding genes, Bringing neuromuscular Giving communities a voice fundamental advances in science transforming therapies therapies to market in international healthcare Dr Shamima Rahman Professor Phil Beales Professor Francesco Muntoni Dr David Osrin People 30 Working with UCL Business PLC 31 Awards, honours and prizes 34 Grants and donations 38 Senior academic staff 43 Administration Cover: Jessica, age 12, is a regular visitor on Island Day Unit. She has a port wine stain and is currently having laser treatment to reduce its intensity. Research Review 2011/12 01 Director’s report The UCL Institute of Child Health (ICH) is Europe’s largest academic centre for research and education in children’s health and disease. As part of University College London’s (UCL) Faculty of Population Health Services, our research stands to improve the lives of children and families across the UK and around the world. The ICH became a founding member of courses in cell and gene therapy and child the Faculty of Population Health Sciences and adolescent mental health. It was with in 2011, created as part of a restructuring great joy and anticipation that we opened of the UCL School of Life and Medical a completely refurbished Wolfson Centre Sciences. This emphasises the Institute’s in Mecklenburgh Square. As our first ever strength in epidemiology through its dedicated teaching and conference room Medical Research Council (MRC) Centre resource, the centre offers numerous of Epidemiology for Child Health (Head: meeting rooms with audio-visual facilities, Professor Carol Dezateux), and the UCL a coffee bar, a student common room, Institute for Global Health (Head: Professor and two computer cluster rooms for the Anthony Costello). Excitingly, the faculty taught course students. This superb new brings together the entire life course of facility was made possible by funding from human health, from conception to birth Great Ormond Street Hospital Children’s (the Institute for Women’s Health), from Charity and the Child Health Research newborn to adolescence (the ICH), and Appeal Trust. from young adulthood to old age (the Institute of Epidemiology and Health Care). In this time of financial stringency, it becomes Our basic science and clinical research, ever more challenging to obtain significant which continues to be the ICH’s largest area research grants from the research councils of concentration, is now focused ever more and major research charities. Congratulations on translation into improvements in children’s are due, therefore, to the many Institute staff health, not only for individuals and families, who achieved significant awards during the but also at the population level. year. These include Professor Pete Scambler (Molecular Medicine Unit), who was awarded The Institute welcomed new senior £1.21 million by the British Heart Foundation academics during the year. Dr Paul for a programme grant entitled Tbx1 and Gissen came from Birmingham University cardiovascular morphogenesis: genetic to develop a research programme in networks and tissue interactions; Professor metabolic paediatrics, as a joint appointment Faraneh Vargha-Khadem (Developmental with UCL’s MRC Laboratory of Molecular Cognitive Neurosciences Unit) who obtained and Cell Biology. Dr Manju Kurian also £1.43 million for an MRC programme grant joined the ICH from Birmingham to develop Investigation of hypoxia/ischaemia and her work on the genetic basis of children’s patterns of neuropathology associated with movement disorders. Dr Nadia Micali memory impairment, from infancy through arrived from the Institute of Psychiatry as adolescence; and Dr Paul Gissen (Clinical holder of a prestigious National Institute of and Molecular Genetics Unit) who was Health Research Clinician Scientist award, awarded a Wellcome Trust Senior Clinical to develop her research programme into Fellowship worth £1.29 million for his eating disorders in young people. project The role of two key intracellular trafficking genes, VPS33B and VIPAR, Education plays an ever more important in development and disease. Jack, 13, has been visiting Great role in the life of the ICH, as a key part Ormond Street Hospital fairly of our mission to train the next generation Professor Phil Beales of the Molecular frequently since February, due to of child health professionals. Professor Medicine Unit was elected a Fellow of the problems with his airway and lungs. Christine Kinnon was appointed Vice- Academy of Medical Sciences (FMedSci), He is very impressed with the new Dean for Education within the Faculty in recognition of his contributions to human ENRC Cardiac Day Care Unit on of Population Health Sciences, and the genetics and birth defects research. Walrus Ward, especially because of all the new TVs which he can watch Institute launched two new Masters level sports on. He loves football and is a huge Manchester United fan! Director’s report Research Review 2011/12 03 Director’s report continued Professor Anthony Costello (UCL Institute people from low-, middle-, and high-income for Global Health) was awarded the James countries were published by Russell Viner Spence Medal, the highest honour of the and colleagues (both studies in the Lancet) . Royal College of Paediatrics and Child Health. Among our staff who achieved This will be my last report as Director of senior promotion at UCL, Jane Sowden the ICH: I step down in September 2012, became Professor of Developmental after almost 10 years in the post. It has Biology and Genetics for her research been an enormous privilege to lead this into the genetics and stem cell therapy of outstanding Institute over a formative period eye disorders, and Russell Viner became in its existence. With the invaluable help of Professor of Adolescent Health for his Great Ormond Street Hospital Children’s work on diabetes, obesity, and risk-taking Charity, we have been able to greatly improve behaviours in young people. Promoted to the estate, converting the Institute’s main Reader were Khalid Hussain, a paediatric building from only two floors of laboratories endocrinologist investigating disorders in 2003, to its current six-floor laboratory of the pancreas; Shamima Rahman, a structure. We have extended the range metabolic paediatrician specialising in of research and teaching to encompass mitochondrial disorders; and Alastair general and adolescent paediatrics, Sutcliffe, a paediatrician studying health alongside our rare diseases research. outcomes of children conceived by assisted reproductive technologies. At the same time, we have strengthened the Institute’s close and productive I am pleased to report a continuing excellent relationship with Great Ormond Street performance by members of the Institute in Hospital. Perhaps most important of all, publishing the outcomes of their research we have brought a new generation of young in the highest ranking journals. In 2011, I clinical and non-clinical investigators into instigated Paper of the Month as part of team leader positions, ensuring that the my regular news bulletin for staff, and this highest quality of child health research has generated considerable competition will continue in future. The past 10 years among authors! To mention just a few of have seen the full integration of the Institute the landmark studies in 2011: new gene within UCL, enabling the ICH to go forward discoveries were reported by Phil Beales as part of a world-class, multi-faculty and colleagues for 3MC syndrome (Nature university.
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