On Oct. 1, 2019, the National Institutes of Health (NIH) awarded a $3.5 million grant to Georgia Institute of Technology researchers Phil Santangeloﾠ and James Dahlman to develop an mRNA-based drug to correct hematopoietic stem cells.

Hematopoietic stem cells are stem cells that give rise to other blood cells. By creating an mRNA-based drug that safely edits HSPCs, the project aims to advance gene editing as a viable therapeutic approach for blood disorders, such as sickle cell disease.

As co-principal investigators, they aim to iteratively evolve new drug delivery nanoparticles that target HSPCs in vivo, reduce mRNA immunogenicity (reduce the immune response to make this type of treatment more effective), and further analyze gene editing. They will work with Francois Villinger, a professor at the University of Louisiana Lafeyette. The team will combine a DNA barcoded nanoparticle technology to screen thousands of nanoparticles in vivo, synthesize mRNA-based drugs with low immunogenicity and cell type-specific expression, and utilize a customized bioinformatics pipeline that facilitates ‘big data’ nanoparticle experiments to more quickly identify treatment options.

The project is being funded by the NIH Somatic Cell Genome Editing program. Making changes to a patient’s DNA can have powerful implications for the treatment of disease. To tap into this immense potential, NIH recently awarded 24 Somatic Cell Genome Editing grants — totaling approximately $89 million over the next four years — to support research aimed at improving methods to edit the human genome.

A genome is an individual’s complete set of DNA, the molecules that carry genetic information. Many diseases are caused by DNA changes that can be inherited from parents or can happen during a person’s lifetime. In the past decade, scientists have developed techniques to edit DNA in order to treat disease. The newly awarded grants focus on editing DNA in somatic cells, which are cells in the body other than reproductive cells. Since somatic cells do not pass DNA to the next generation, genome editing changes in somatic cells cannot be inherited.