Funded National Call Projects
GOSH Charity has a passion and commitment to support paediatric research nationally. Each year we invite researchers across the country to apply for funding for their child health research projects through the National Call. We review these applications and fund the highest quality research most likely to have life-changing benefits to children.
GOSH Charity National Call 2023-24
We received a total of 80 outline applications for the 7th GOSH Charity National Call for research project grants.
The following 12 projects were awarded grants, totalling £2,798,706.21.
| Lead Investigator | Institution | Grant Title | Total |
|---|---|---|---|
| Dr Gabriel Galea | UCL Great Ormond Street Institute of Child Health | Improving prevention and patient outcomes for Terminal Myelocystocele | £249,614.00 |
| Professor Jane Sowden | UCL Great Ormond Street Institute of Child Health | Norrie disease therapy targeting endothelial cells | £249,857.07 |
| Dr Gabriele Lignani | UCL Institute of Neurology | Gene therapy for ARID1B-related disorder | £236,001.31 |
| Professor Philippa Mills | UCL Great Ormond Street Institute of Child Health | Improved diagnosis and monitoring of treatment for patients with epilepsy caused by mutations in ALDH7A1 | £160,297.14 |
| Professor Brian Bigger | University of Edinburgh | Improving Ex Vivo Stem Cell Gene Therapy for Mucopolysaccharidosis Types II and IIIB | £250,000.00 |
| Professor Francesco Saverio Tedesco | University College London | An Advanced Multi-Organoid Platform for Therapy Modelling in X-Linked Myotubular Myopathy | £248,939.22 |
| Dr Beth Payne | University College London | Defining mechanisms and clonal evolution to leukaemia associated with germline CEBPA mutations | £249,195.60 |
| Dr Melissa Bowerman | Keele University | Assessing novel mutant-selective PDE4D PROTACs to restore molecular and functional pathologies in a mouse model of ACRDYS 2 | £249,631.08 |
| Dr Frederik van Delft | Newcastle University | Therapeutic reversal of glucocorticoid resistance in haematological malignancies | £247,480.00 |
| Dr Amy Richardson | University College London | Targeting inhibition to treat seizures in focal cortical dysplasia | £176,289.01 |
| Dr Claire Jackson | University of Southampton | Uplifting genetic detection of rare lung diseases; a novel diagnostic pipeline utilising RNA-seq analysis | £232,842.00 |
| Professor Manju Kurian | UCL Great Ormond Street Institute of Child Health | Advancing gene therapy for Beta Propeller Protein Associated Neurodegeneration (BPAN) | £248,559.78 |
GOSH Charity and Sparks National Call 2022-23
We received a total of 88 outline applications for the 6th joint GOSH Charity and Sparks National Call for research project grants.
The following 10 projects were awarded grants, totalling £2,208,420.86.
| Lead Investigator | Institution | Grant Title | Total |
|---|---|---|---|
| Professor Waseem Qasim | UCL Great Ormond Street Institute of Child Health | Base editing for gentle repair of fragile DNA disorders | £247,202.38 |
| Dr David Carmichael | King’s College London | Developing layer fMRI as a new biomarker of epilepsy in children for treatment optimisation | £248,649.44 |
| Professor Stephen Eyre | University of Manchester | Linking genetics to T-regulatory cell levels and function in JIA: evidence for a clinical trial | £191,422.88 |
| Dr Carles Gaston-Massuet | Queen Mary University of London | Understanding the role of cellular senescence in RASopathies | £249,917.99 |
| Dr Kate Oulton | Great Ormond Street Hospital for Children | Seen and Be Heard: Identifying the barriers/facilitators to equitable cancer care for children with Learning Disabilities/ Autism Spectrum Condition | £188,748.00 |
| Dr Haiyan Zhou | UCL Great Ormond Street Institute of Child Health | Developing RNA-based novel therapies to improve the current treatment of hereditary tyrosinemia type 1 | £246,899.20 |
| Dr James Dixon | University of Nottingham | RETTune: Self-regulated transgene expression to treat Rett Syndrome | £242,454.28 |
| Dr Cristina Dias | King’s College London | Understanding neurodevelopment in mosaic chromatin disorders using in vitro and ex vivo models of brain development | £249,877.69 |
| Professor Paul Gringras | King’s College London | Narcolepsy Revolution – innovative home-based diagnostic testing for Narcolepsy | £112,890.00 |
| Professor Robert McFarland | Newcastle University | Delineating the neuropathophysiological mechanisms underpinning severe drug-resistant epilepsy in Alpers’ syndrome | £230,359.00 |
GOSH Charity and Sparks National Call 2021-22
We received a total of 124 outline applications for the 5th joint GOSH Charity and Sparks National Call for research project grants.
The following 8 projects were awarded grants, totalling £1,705,597.14.
| Lead Investigator | Institution | Grant Title | Total |
|---|---|---|---|
| Professor Judith Breuer | UCL Great Ormond Street Institute of Child Health | Repurposing antiviral drugs for serious viral infections including SARS-CoV-2 | £147,667.40 |
| Professor Suzanne Turner | University of Cambridge | Improving global treatment outcomes of children diagnosed with Burkitt lymphoma | £249,123.00 |
| Professor Claire Booth | UCL Great Ormond Street Institute of Child Health | Gene editing for XIAP deficiency: preclinical development | £195,526.36 |
| Dr Alessia Cavazza | UCL Great Ormond Street Institute of Child Health | Evaluating the efficacy of hematopoietic stem cell gene editing in treating Wiskott-Aldrich Syndrome | £242,469.69 |
| Mr Richard Hewitt | UCL Great Ormond Street Institute of Child Health | Improved delivery methods for airway cell and gene therapy in epidermolysis bullosa | £188,710.32 |
| Professor Marta Korbonits | Queen Mary University of London | Discovering the molecular pathology and treatment of a novel paediatric disease | £245,422.16 |
| Dr Gabriele Lignani | UCL Institute of Neurology | SINEUP-Nav: an innovative approach to cure Dravet Syndrome | £209,623.52 |
| Dr Emanuel Rognoni | Queen Mary University of London | Exploring the role and therapeutic potential of dermal fibroblasts in Junctional Epidermolysis Bullosa | £227,054.69 |
GOSH Charity and Sparks National Call 2020-21
We received a total of 126 outline applications for the 4th joint GOSH Charity and Sparks National Call for research project grants.
The following 11 projects were awarded grants, totalling £2,504,880.76.
| Lead Investigator | Institution | Grant Title | Total |
|---|---|---|---|
| Prof Mina Ryten | UCL Great Ormond Street Institute of Child Health | Leveraging transcriptomics to improve the diagnostic rate and understanding of neurometabolic disorders | £222,826.37 |
| Prof Matthew Wood | University of Oxford | uORF-mediated SCN1A up-regulation for the treatment of Dravet syndrome | £234,677.00 |
| Dr Christian Babbs | University of Oxford | Reversing Zeta-Globin Transcriptional Silencing: Towards Embryonic Globin Induction in Patients With Severe Alpha-Thalassemia | £241,915.74 |
| Prof David Rubinsztein | University of Cambridge | Identification of pathways that protect against Beta-propeller protein-associated neurodegeneration | £248,780.00 |
| Prof Paolo de Coppi | UCL Great Ormond Street Institute of Child Health | Dissecting the role of mechanical lung compression in Congenital Diaphragmatic Hernia on a single cell level. | £229,305.04 |
| Prof Christina Liossi | University of Southampton | Development and Validation of a Paediatric Breakthrough Pain Assessment Tool | £224,650.00 |
| Dr Paula Alexandre | UCL Great Ormond Street Institute of Child Health | Investigate the underlying causes of cerebellar developmental disorders | £249,375.48 |
| Dr Siddharth Banka | University of Manchester | Building iPSC-derived models to identify treatments for KMT2 chromatin disorders | £248,605.00 |
| Dr Beth Payne | University College London | Targeting amino acid metabolism as a novel treatment avenue for Diamond-Blackfan anaemia | £249,650.29 |
| Prof Patrizia Ferretti | UCL Great Ormond Street Institute of Child Health | Generating human models of acrodysostosis for testing mechanisms and pharmacological intervention | £245,028.40 |
| Dr Hayley Crawford | University of Warwick | A clinical checklist of causes of poor behavioural outcomes in children with moderate-profound intellectual disability and complex needs | £110,067.44 |
GOSH Charity and Sparks National Call 2019-20
We received a total of 82 outline applications for the 3rd joint GOSH Charity and Sparks National Call for research project grants.
The following 11 projects were awarded grants, totalling £2,273,124.
| Lead investigator | Institution | Grant Title | Total |
|---|---|---|---|
| Dr Manavendra Pathania | University of Cambridge | Identifying drivers of aggressive childhood brain tumours that could hold the key to new treatments. | £249, 945 |
| Professor Nicholas Greene | UCL Great Ormond Street Institute of Child Health | Testing a new compound derived from cinnamon that could help lower toxic levels of ammonia and glycine in children with the rare metabolic diseases non-ketotic hyperglycinemia and Urea Cycle Disorders. | £249,741 |
| Dr Giovanni Baranello | UCL Great Ormond Street Institute of Child Health | Supporting the UK arm of a global trial understanding if the breast cancer drug tamoxifen could help children with the muscle disorder X-linked myotubular myopathy. | £232,432 (£58,108 from Myotubular trust) |
| Professor Jane Sowden | UCL Great Ormond Street Institute of Child Health | Aiming to replace the faulty gene that causes deafness in Norrie Disease, saving the hearing of boys who are born blind. | £251,332 (£62,500 from Norrie Disease Foundation) |
| Professor Paul Gissen | UCL Great Ormond Street Institute of Child Health | Aiming to tackle all symptoms of a complex multi-organ disorder, Arthrogryposis Renal Dysfunction and Cholestasis Syndrome (ARC), with two types of gene therapy at once. | £228,757 |
| Professor Andrew Wilkie | University of Oxford | Aiming to unravel why the activity of a gene called FOXD3 is affected, and whether it could offer a diagnostic tool, in some children with craniosynostosis - a condition which causes the bones in the skull to fuse too early. | £169,449 |
| Professor Nicola Dawes | Oxford Brookes University | A lifestyle weight management program for children with conditions that strip the outer protective layer of their nerves, like MS or Neuromyelitis Optica Spectrum Disorder (NMOSD). | £122,771 |
| Dr Rajvinder Karda | University College London | Driving a new approach to gene therapy for a rare, inherited and aggressive type of epilepsy called Dravet Syndrome. | £249,271 (£62,318 from Dravet Syndrome UK) |
| Dr Victor Hernandez Hernandez | Brunel University of London | Using gene therapy to the eyes and brain to correct the most common genetic mistake that leads to Bardet Biedl Syndrome – a condition that leads to blindness, learning disabilities, and weight gain (and other symptoms). | £220,191 |
| Dr Hassan Rashidi | UCL Great Ormond Street Institute of Child Health | Developing an implantable and removable ‘liver patch’ to provide liver support to patients with the metabolic condition non-ketotic hyperglycinemia. | £101,394 |
| Dr Kate Baker | University of Cambridge | Understanding more, through gene and brain activity testing, about a range of conditions that affect the chemicals that allow nerves to communicate – Synaptic Vesicle Cycling Disorders. | £197,841 |
GOSH Charity and Sparks National Call 2018-19
We received a total of 83 outline applications for the 2nd joint GOSH Charity and Sparks National Call for research project grants.
The following 12 projects were awarded grants, totalling £2,174,694.
| Lead investigator | Host institution | Total | Project title |
|---|---|---|---|
| Professor Dimitri Kullman | UCL Institute of Neurology | Gene therapy for epilepsy & focal cortical dysplasia | £190,404.38 |
| Dr Susan Campbell | Sheffield Hallam University | eiF2B bodies in association with the human disease Leukoencephalopathy with Vanishing White Matter -a novel diagnostic tool | £189,024 |
| Professor John Anderson | UCL Great Ormond Street Institute of Child Health | Development of B7-H3 targeting chimeric antigen receptors for cellular therapy of childhood solid cancers | £212,589.30 |
| Dr Katie Gallagher | Homerton University Hospital NHS Foundation Trust | The attitudes of health professionals involved in preterm delivery towards the treatment of extremely preterm infants | £23,209 |
| Dr David Carmichael | King's College London | Realising the potential of 7T MRI as a clinical tool for paediatric neuroimaging | £224,981.90 |
| Dr Matthias Zilbauer | University of Cambridge | Investigating intestinal epithelial cell biology in paediatric Inflammatory Bowel Disease | £238,250 |
| Professor Henry Houlden | UCL Institute of Neurology | AMPA Receptor Mutations are important causes of epilepsy, autism spectrum and developmental disorders | £196,734.36 |
| Dr Sara Benedetti | UCL Great Ormond Street Institute of Child Health | Targeting the blood-brain barrier endothelium to increase hematopoietic stem cell engraftment for lysosomal storage disease gene and cell therapy | £96,085.94* |
| Dr Owen Williams | UCL Great Ormond Street Institute of Child Health | Pre-clinical evaluation of mebendazole in AML therapy | £232,782.24 |
| Dr Rajvinder Karda | University College London | AAV gene targeting of long non-coding RNA in a mouse model of Dravet Syndrome | £75,725.17** |
| Professor Grant Stewart | University of Birmingham | Investigating the pathogenic impact of mutations in TONSL as the genetic cause of a novel skeletal disorder in children | £246,993 |
| Dr Jasper de Boer | UCL Great Ormond Street Institute of Child Health | Exploiting therapeutic targets in paediatric high grade gliomas with the H3K27M mutation | £247,914.21 |
*co-funded with Krabbe UK
**co-funded with Dravet Syndrome UK
GOSH Charity and Sparks National Call 2017-18
We received a total of 142 outline applications for the 1st joint GOSH Charity and Sparks National Call for research project grants.
The following 14 projects were awarded grants, totalling £2,156,225.
| Lead investigator | Host institution | Total | Project title |
|---|---|---|---|
| Dr Ming Lim | Oxford University Hospitals | UK Multicentre Study of Children with Opsoclonus Myoclonus Syndrome (UMSCOM) Study | £45,898 |
| Dr Heidi Fuller | Keele University | Therapy development for children with motor neuron disease | £192,945 |
| Professor Mary Rutherford | King's College London | Investigating early brain development in Down Syndrome: new therapeutic windows for intervention | £203,084 |
| Professor Tessa Crompton | UCL Great Ormond Street Institute of Child Health | Induction of tolerance by thymic epithelial cell transplantation | £146,243 |
| Professor Andrew Copp | UCL Great Ormond Street Institute of Child Health | Chiari II brain malformation: association with spina bifida and response to fetal surgery | £200,139 |
| Professor Celia Moss | Birmingham Children's Hospital | BPSU study: The incidence, management and early outcome of congential ichthyosis | £33,151 |
| Dr Alessia Cavazza | UCL Great Ormond Street Institute of Child Health | Targeted gene correction for the treatment of Severe Combined Immunodeficiency caused by mutations in the IL7R gene | £41,589 |
| Professor Christina Liossi | University of Southampton | End-of-Life pain management by carers and healthcare professionals in infants, children and young people in out of hospital settings | £214,821 |
| Professor Michael Duchen | University College London | From the biology of EPG5 to the pathophysiology of Vici syndrome | £194,675 |
| Professor Chris O'Callaghan | UCL Great Ormond Street Institute of Child Health | Restoring ciliary function in Primary Cilia Dyskinesia | £186,044 |
| Dr Thomas O'Brien | Liverpool John Moores University | An intelligent ultrasound-based diagnostic tool to decouple neural and structural contributions to reduced joint range of motion in cerebral palsy | £148,573 |
| Professor Giulio Cossu | University of Manchester | A pre-clinical investigation on cell pharmacodynamics after intra-arterial systemic delivery in a rat model of Duchenne Muscular Dystrophy | £235,664 |
| Dr Karin Straathof | UCL Great Ormond Street Institute of Child Health | T-cell immunotherapy for childhood brain tumour DIPG | £201,324 |
| Dr Sylwia Ammoun | Plymouth University | Investigation and targeting cellular prion protein PrPC in Neurofibromatosis type II related tumours schwannomas, meningiomas and spinal ependymomas | £112,071 |