Gene editing in blood stem cells

Rare childhood diseases known as bone marrow failure syndromes can be fatal due to the loss of vital blood production. These conditions present a promising opportunity for treatment using advanced Cas9-based gene editing techniques. Our research aims to develop mRNA-based methods to deliver gene editing tools to hematopoietic stem cells (HSCs), the source of all blood cells, to potentially cure these disorders.
This research project will help improve our use of mRNA delivery methods (similar to the technology used in COVID vaccines) for gene editing in HSCs.

mRNA-based gene editing offers several compelling advantages for treating bone marrow failure syndromes. This approach allows for efficient delivery of editing tools, as mRNA can be easily introduced into stem cells outside the body. Unlike some alternative methods, mRNA exhibits low toxicity and doesn’t stimulate the immune system, making it well-tolerated by sensitive stem cells. Our preliminary data is particularly encouraging, showing that mRNA-based editors perform over ten times better than their DNA-based counterparts in terms of editing efficiency. Perhaps most importantly, while the mRNA itself is only temporarily present in the cells, the genetic corrections it facilitates are permanent. This means that a single treatment could potentially offer lifelong benefits to patients, addressing the root cause of their condition at the genetic level.
Research Activities: Students will be involved in: Designing and optimizing mRNA-based gene editing tools; Culturing and manipulating HSCs; Assessing gene editing efficiency and cell viability; Analyzing the long-term effects of gene editing in cell culture models

This project is ideal for students with a strong interest in molecular biology, stem cell research, and genetic therapies. Participants will gain hands-on experience with cutting-edge techniques in gene editing and stem cell manipulation, contributing to the development of potentially life-saving treatments for rare childhood diseases.