Genetics and Genomic Medicine
- Understanding lipid metabolic flux in Gaucher and Parkinson's diseases, Supervisors: Wendy Heywood and Jenny Hallqvist
- Epigenetic modifiers of a Mendelian disease and implications for gene therapy. Supervisors: Dagan Jenkins and Louise Gregory
- Ciliary programming of fibrotic disease responses. Supervisors: Dagan Jenkins and Louise Gregory
- Developing brain-targeting lipid nanoparticles for translational RNA applications: proof of concept in creatine transporter deficiency. Supervisors: Julien Baruteau and Naresh Hanchate
- Towards personalised medicine for rare inherited brain diseases by identifying and manipulating variant RNA transcripts. Supervisors: Prof Sara Mole and Dr Chris Minnis
- Neuroimaging and genomics in primary mitochondrial diseases. Supervisors: Prof Shamima Rahman and Dr Konrad Wagstyl
- Rewiring astrocyte metabolism to treat Mental Health disease. Supervisors: Julien Baruteau, Manju Kuriane.
- Developing nucleic acid therapy for paediatric neurological diseases Supervisors: Haiyan Zhou, Mina Ryten, Alice Davidson and Jinhong Meng
- Mitochondrial Disease Gene Discovery using the 100,000 Genomes Dataset Supervisors: Shamima Rahman, Damian Smedley, Nandaki Keshavan
Developmental Biology and Cancer
- Investigating the role of MLL protein in the epigenetic regulation of paediatric diffuse midline glioma. Supervisors: David Michod and Juan Pedro Martinez-Barbera
- Human models for the investigation of proteins associated with birth defects affecting face and long bone skeleton and cognitive development. Supervisors: Patrizia Ferretti, Oliver Gardner and Simon Eaton
- Understanding the function of senescent cells in genetic models of paediatric low-grade brain tumours. Supervisors: Juan Pedro Martinez-Barbera and Laura Donovan
- Dissecting the protumourigenic activities of senescent cells in the development and relapse of paediatric diffuse midline glioma. Supervisors: Juan Pedro Martinez-Barbera and David Michod
- Building a vascularized human alveolar epithelium in vitro. Supervisors: Paolo De Coppi and Federica Michielin
- Development of a platform for somatic cell reprogramming to iPSCs in microfluidic environment. Supervisors: Jane Sowden, Paolo De Coppi and Marco Pellegrini
- Developing AAV gene therapy for Polycystic Kidney Disease. Supervisors: Dr Jennie Chandler and Professor David Long
- Lymphatics in kidney growth and regeneration. Supervisors: Professor David Long and Dr Daniyal Jafree
- Skull base malformations in craniofacial disorders. Supervisors: Erwin Pauws, Tengyang Qiu
- Generation of a Single-Cell Atlas of Human Amniotic Fluid in Congenital Diaphragmatic Hernia: Developing Advanced Disease Models and Drug Testing Tools. Supervisors: Mattia Gerli, Simon Eaton
- Validation of Human-Derived Neuroblastoma Organoids and Stem Cells for Fluorescence-Guided Surgery Targeting Neuroblastoma Antigens. Supervisors: Stefano Giuliani, Paolo de Coppi
- Improving Molecular Diagnosis of Childhood TumourS Supervisors: Thomas Jacques, Darren Hargrave
- Investigating senolytic therapy in a novel mouse model of airway stenosiS Supervisors: Dr Rob Hynds, J.P. Martinez-Barbera
- Re-purposing anti-fibrotic therapies in airway epidermolysis bullos Supervisros: Dr Rob Hynds, Dr David Pearce, Prof Rachel Chambers
- Prenatal Gene Therapy for Alpha Thalassemia Using Virus-Like Particles. Supervisors: Stavros Loukogeorgakis, Alessia Cavazza, Dominic Scaglioni
- Targeting metabolic pathways to rescue hindbrain hypoplasia in Down Syndrome. Supervisors: Paula Alexandre, Nicholas Greene
- Understanding the biology of advanced childhood brain tumours. Supervisors: Thomas Jacques, Darren Hargrave
- Developing artificial mini-kidneys to treat childhood urinary tract disorders. Supervisors: Professor David Long, Dr Karen Price, Dr Gideon Pomeranz
Infection, Immunity and Inflammation
- Using gene editing to treat a rare inflammatory disorder called Deficiency of adenosine deaminase type 2 (DADA2). Supervisors: Despina Eleftheriou and Paul Brogan
- Exploring New Treatments for Airway Inflammation by Targeting CD44. Supervisors: Dr Claire Smith and Dr Tereza Masonou
- Predicting and preventing spontaneous preterm birth. Supervisors: Mona Bajaj-Elliott and Patricia Hunter
- Characterising infectious illnesses in children through integrated host/microbe metagenomics studies. Supervisors: Judith Breuer and Sofia Morfopoulou and Cristina Venturin
- Targeting the blood-brain barrier to increase hematopoietic stem cell engraftment for neurometabolic disorders. Supervisors: Sara Benedetti, Giorgia Santilli, Bethan Critchley
- Using Genomics to Detect Multi-Drug Resistant Organisms that are a Threat to Child Health. Supervisors: Louis Grandjean, James Hatcher, Ben Sobkowiak
- Development of new gene editing delivery systems that cross the blood brain barrier for the treatment of Krabbe Disease and other lysosomal storage disorders. Supervisors: Sara Benedetti, Alessia Cavazza, Asma Naseem
- Why the drugs don’t work: using nanopore sequencing to decode immune cells in immunocompromised babies with severe RSV bronchiolitis to find new therapies
Population, Policy and Practice
- Health and development outcomes for children with kidney disease: a population-based cohort using novel linkage between health and education records. Supervisors: Katie Harron and Kate Lewis
- Price Promotions and Children’s Health: Evidence to inform policy. Supervisor: Oliver Mytton and Mario Cortina Borja
- Outcomes for childhood cancer survivors and their siblings: a national data linkage study using ECHILD. Supervisors: Katie Harron and Ruth Blackburn
- Novel approaches to imaging the developing brain in children with intellectual disabilities of genetic origin. Supervisors: Prof David Skuse and Prof Chris Clark
- Paternal health and family courts: a study using whole-of-England, linked administrative data. Supervisors: Ruth Gilbert and Matthew Jay
- Have disease modifying treatments in cystic fibrosis reduced or exacerbated health inequalities? Supervisors: Gwyneth Davies and Ofran Almossawi
- Health outcomes of children with rare or complex conditions and their families: a longitudinal cohort study using linked primary and secondary healthcare data in England. Supervisors: Ania Zylbersztejn and Joachim Tan
- Child-to-adult BMI trajectories in British birth cohorts: life course risk factors and adult health impacts. Supervisors: Leah Li and Simon Russell
- National Child Measurement Programme: using statistical and machine learning approaches to inform public health practice. Supervisors: Oliver Mytton, Mario Cortina Borja
- Climate change and double burden of malnutrition in Brazilian children. Supervisors: Leah Li, Mengyun Liu
Developmental Neurosciences
- Developing 3D patient-derived neuronal models to elucidate neurodevelopmental defects in Spinal Muscular Atrophy. Supervisors: Giovanni Baranello and Serena Barral
- Unravelling the role of SMN in neurodevelopment. Supervisors: Giovanni Baranello and Naresh Hanchate
- Adherence to Ketogenic diet therapy for children and young people with epilepsy: how can we do better? Supervisors: Prof Rob Horne and Dr Natasha Schoeler
- Do novel gene therapies influence motor, social and thinking skills in young children with spinal muscular atrophy? Supervisors: Michelle de Haan, Giovanni Baranello
- Understanding and Exploiting Stem Cell Regenerative Potential in Muscle Health and Disease. Supervisors: Francesco Muntoni, Massimo Ganassi
- Predictors of functional outcome in children born without the corpus callosum. Supervisors: Michelle de Haan, Rhonda Booth
- Identification of EEG biomarker for genetic early onset epilepsy syndromes. Supervisors: Amy McTague, Ronit Pressler
- Towards intraoperative visualisation of brain connectivity in epilepsy patients. Supervisors: Jon Clayden, Martin Tisdall
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