Funded Lift Off Grants: Discovery Research Projects
Lift Off Grants: Discovery Research are intended to support the first step for a new research question in an area of rare or complex paediatric disease. These grants provide early-stage research funding, intended to give early support for new research projects. This scheme supports research across the spectrum of medical conditions affecting the foetus, neonates, and children directly as well as pregnancy disorders that affect the child.
| Lead Investigator | Institution | Project Title | Total |
|---|---|---|---|
| Professor Despina Eleftheriou | UCL Great Ormond Street Institute of Child Health | Preclinical in vivo evaluation of antisense oligonucleotide therapy for STING Associated Vasculopathy with Onset in Infancy (SAVI | £86,250.00 |
| Professor Stephen Hart | UCL Great Ormond Street Institute of Child Health | A novel humanised mouse model for cystic fibrosis gene delivery research | £86,222.62 |
| Dr Colin Butler | UCL Great Ormond Street Institute of Child Health | Recombinant laminin-332 protein as a therapy for airway epidermolysis bullosa | £85,918.37 |
| Professor Haiyan Zhou | UCL Great Ormond Street Institute of Child Health | Proof-of-concept RNA-editing therapy studies in COL6-related congenital muscular dystrophy | £86,249.08 |
| Dr Giovanni Giobbe | UCL Great Ormond Street Institute of Child Health | Antral foveolar hyperplasia in vitro modelling | £85,974.67 |
| Dr Christopher Minnis | UCL Great Ormond Street Institute of Child Health | Development of SPARTA-CLN3 to accelerate therapeutic potential in juvenile CLN3 disease using a multidisciplinary approach | £74,847.82 |
| Dr Asma Naseem | UCL Great Ormond Street Institute of Child Health | A Trojan horse strategy for targeted drug delivery for the treatment of lysosomal storage disorders | £75,104.68 |
| Lead Investigator | Institution | Project Title | Total |
|---|---|---|---|
| Dr Gabriel Galea | UCL Great Ormond Street Institute of Child Health | Does Maternal Age Impact Spina Bifida Prevention By Folic Acid? | £74,608.00 |
| Prof David Long | UCL Great Ormond Street Institute of Child Health | Activating AMPK as a Therapy for Autosomal Recessive Polycystic Kidney Disease | £74,678.91 |
| Dr Sonam Gurung | UCL Great Ormond Street Institute of Child Health | Targeting claudin-1 to increase blood brain barrier permeability and enhance cerebral drug delivery | £74,825.30 |
| Dr Karin Tuschl | UCL Great Ormond Street Institute of Child Health | mRNA therapies for SLC transporter defects – SLC30A10 deficiency as proof-of-concept | £74,581.94 |
| Dr Yara Sanchez Corrales | UCL Great Ormond Street Institute of Child Health | Elucidating the cellular mechanisms of rare placental disease with machine learning | £66,902.63 |
| Dr Federica Michielin | UCL Great Ormond Street Institute of Child Health | Role of mechanical signals in alveolar differentiation | £74,518.05 |