Enabling next generation hematopoietic-based therapeutics and manufacturing

Several human disorders such blood disorders or immunodeficiency’s are life-long diseases for which patients will require life-long treatment that are not curative in nature. However, recent advances in the field of gene therapy and gene editing has now open the possibility of curing such disorders. However, current limitations in these technologies limit the extent to which they could be applied at large in both developed and under developed countries. The current proposal offers to start developing new technologies that could one day make treatment of these diseases accessible even to moderately developed countries. We propose herein, 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. Following a different strategy, we will also test whether stem cells could be used to produce precursor cells of blood and immune cells using cell engineering techniques. The development of these new cell products will be of significant benefit to Canadians and patients worldwide that requires frequent treatments to overcome
the symptoms associated with their disorders.

PINEAULT, Nicolas

Canadian Blood Services

Intramural Research Grant Program

Ontario

$300,000