Funded studentships

Developing Prenatal Nanoparticle-Mediated Gene Editing to Prevent Sickle Cell Disease Before Birth

Sickle cell disease (SCD) remains a major global health burden, with limited curative options and significant morbidity beginning early in life. Advances in non-invasive prenatal testing now offer a unique opportunity to intervene before the onset of irreversible disease. This project aims to engineer next-generation ionisable lipid nanoparticles (LNPs) capable of delivering gene-editing tools directly to fetal haematopoietic stem cells for in-utero correction of SCD. Recent laboratory work has revealed that erythroid and myeloid cells use distinct nanoparticle uptake pathways, underscoring the need for rational, cell-specific LNP design. Building on these insights, we will optimise nanoparticle composition, endocytic engagement, and targeting ligands to maximise editing efficiency while minimising toxicity. These systems will be assessed in fetal liver–derived stem cells and advanced erythroid models to evaluate biocompatibility, internalisation, and gene correction. By combining biomaterials engineering with prenatal therapeutics, this project advances the development of a scalable curative strategy capable of preventing SCD before birth.

Apply now:

https://eb4bm.org/project/developing-prenatal-nanoparticle-mediated-gene-editing-to-prevent-sickle-cell-disease-before-birth/

Dr Elizabeth Blackburn

Contact details

Research

Research

Shangaris Group

Research

Shangaris Group

Based in