New platform for targeted gene editing of hematopoietic stem cells for cellular therapy

Many blood disorders are life-threatening conditions that require regular transfusion support and non-curative long-term therapies, and/or stem cell transplantation that remains risky with a high risk of complications. Recent advances in the field of gene therapy and gene editing has introduced the possibility of curing some of these disorders. While very promising, limitations in these technologies remain and limit the ability to apply this type of treatment more broadly. In this proposal, we outline our approach to developing lipid-based nanoparticle delivery to bloodforming stem cells as a novel approach to treating blood and immune disorders. We propose to change the means of delivery from complex virus-based methods to lipid-based nanoparticles. In addition, to allow for the future treatment of patients, we will test a new technology that should make possible cell-specific targeting to the cells in the bone marrow that produce all blood and immune cells, the hematopoietic stem cells. Moreover, we will perform knowledge synthesis work through the conduct of a systematic review of gene editing used in conjunction with stem cell transplantation. This will allow us to understand how gene editing can be used in specific conditions and will help design future clinical studies that will benefit patients.

PINEAULT, Nicolas

Canadian Blood Services

Postdoctoral Fellowship Program

Ontario

$120,000