- Grants since 2016
- 66
- Awarded, last 5 years (where amounts are published)
- £3.9M
- Funded works, last 5 years
- 50
- Funded works, all time
- 517
Grants
2022 · grant · 2022-23
RETTune: Self-regulated transgene expression to treat Rett Syndrome
James Dixon · University of Nottingham
2022 · grant · 2022-23
Developing RNA-based novel therapies to improve the current treatment of hereditary tyrosinemia type 1
Haiyan Zhou · University College London
2022 · grant · 2022-23
Developing layer fMRI as a new biomarker of epilepsy in children for treatment optimisation
David Carmichael · King's College London
2022 · grant · 2022-23
Linking genetics to T-regulatory cell levels and function in JIA: evidence for a clinical trial
Stephen Eyre · University of Manchester
2022 · grant · 2022-23
Understanding neurodevelopment in mosaic chromatin disorders using in vitro and ex vivo models of brain development
Cristina Dias · King's College London
2022 · grant · 2022-23
Seen and Be Heard: Identifying the barriers/facilitators to equitable cancer care for children with Learning Disabilities/ Autism Spectrum Condition
Kate Oulton · Great Ormond Street Hospital
2022 · grant · 2022-23
Narcolepsy Revolution – innovative home-based diagnostic testing for Narcolepsy
Paul Gringras · King's College London
2022 · grant · 2022-23
Delineating the neuropathophysiological mechanisms underpinning severe drug-resistant epilepsy in Alpers’ syndrome
Robert Mcfarland · Newcastle University
2022 · grant · 2022-23
Understanding the role of cellular senescence in RASopathies
Carles Gaston-Massuet · Queen Mary University of London
2022 · grant · 2022-23
Base editing for gentle repair of fragile DNA disorders
Waseem Qasim · University College London
2021 · grant · 2021-22
Gene editing for XIAP deficiency: preclinical development
Claire Booth · University College London
2021 · grant · 2021-22
Improving global treatment outcomes of children diagnosed with Burkitt lymphoma
Suzanne Turner · University of Cambridge
2021 · grant · 2021-22
Improved delivery methods for airway cell and gene therapy in epidermolysis bullosa
Richard Hewitt · University College London
2021 · grant · 2021-22
Discovering the molecular pathology and treatment of a novel paediatric disease
Marta Korbonits · Queen Mary University of London
2021 · grant · 2021-22
Evaluating the efficacy of hematopoietic stem cell gene editing in treating Wiskott-Aldrich Syndrome
Alessia Cavazza · University College London
2021 · grant · 2021-22
Repurposing antiviral drugs for serious viral infections including SARS-CoV-2
Judith Breuer · University College London
2021 · grant · 2021-22
Exploring the role and therapeutic potential of dermal fibroblasts in Junctional Epidermolysis Bullosa
Emanuel Rognoni · Queen Mary University of London
2021 · grant · 2021-22
SINEUP-Nav: an innovative approach to cure Dravet Syndrome
Gabriele Lignani · UCL Queen Square Institute of Neurology
2020 · grant · 2020-21
Targeting amino acid metabolism as a novel treatment avenue for Diamond-Blackfan anaemia
Beth Payne · University College London
2020 · grant · 2020-21
Dissecting the role of mechanical lung compression in Congenital Diaphragmatic Hernia on a single cell level.
Paolo De Coppi · University College London
2020 · grant · 2020-21
Reversing Zeta-Globin Transcriptional Silencing: Towards Embryonic Globin Induction in Patients With Severe Alpha-Thalassemia
Christian Babbs · University of Oxford
2020 · grant · 2020-21
A clinical checklist of causes of poor behavioural outcomes in children with moderate-profound intellectual disability and complex needs
Hayley Crawford · University of Warwick
2020 · grant · 2020-21
Leveraging transcriptomics to improve the diagnostic rate and understanding of neurometabolic disorders
Mina Ryten · University College London
2020 · grant · 2020-21
Investigate the underlying causes of cerebellar developmental disorders
Paula Alexandre · University College London
2020 · grant · 2020-21
uORF-mediated SCN1A up-regulation for the treatment of Dravet syndrome
Matthew Wood · University of Oxford
2020 · grant · 2020-21
Building iPSC-derived models to identify treatments for KMT2 chromatin disorders
Siddharth Banka · University of Manchester
2020 · grant · 2020-21
Identification of pathways that protect against Beta-propeller protein-associated neurodegeneration
David Rubinsztein · University of Cambridge
2020 · grant · 2020-21
Development and Validation of a Paediatric Breakthrough Pain Assessment Tool
Christina Liossi · University of Southampton
2020 · grant · 2020-21
Generating human models of acrodysostosis for testing mechanisms and pharmacological intervention
Patrizia Ferretti · University College London
2019 · grant · 2019-20
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.
Andrew Wilkie · University of Oxford
2019 · grant · 2019-20
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.
Paul Gissen · University College London
2019 · grant · 2019-20
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).
Victor Hernandez Hernandez · Brunel University of London
2019 · grant · 2019-20
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).
Nicola Dawes · Oxford Brookes University
2019 · grant · 2019-20
Developing an implantable and removable ‘liver patch’ to provide liver support to patients with the metabolic condition non-ketotic hyperglycinemia.
Hassan Rashidi · University College London
2019 · grant · 2019-20
Identifying drivers of aggressive childhood brain tumours that could hold the key to new treatments.
Manavendra Pathania · University of Cambridge
2019 · grant · 2019-20
Driving a new approach to gene therapy for a rare, inherited and aggressive type of epilepsy called Dravet Syndrome.
Rajvinder Karda · University College London
2019 · grant · 2019-20
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.
Kate Baker · University of Cambridge
2019 · grant · 2019-20
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.
Nicholas Greene · University College London
2019 · grant · 2019-20
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.
Giovanni Baranello · University College London
2019 · grant · 2019-20
Aiming to replace the faulty gene that causes deafness in Norrie Disease, saving the hearing of boys who are born blind.
Jane Sowden · University College London