Gene Therapy Holds Promise for Blood Disorders

Researchers at the University of Delaware have made a step forward in gene therapy by engineering microparticles that deliver gene-regulating material to hematopoietic stem and progenitor cells, which live deep in bone marrow and direct the formation of blood cells.

In a paper published in the journal Science Advances on Nov. 7, 2018, Chen-Yuan Kao, a doctoral student in chemical engineering, and Eleftherios T. (Terry) Papoutsakis, Unidel Eugene du Pont chair of Chemical and Biomolecular Engineering, describe how they used megakaryocytic microparticles to deliver plasmid DNAs and small RNAs to hematopoietic stem cells.

Some previously developed methods to target these stem cells deliver genetic material with help from a virus, risking side effects to the patient, Papoutsakis said. Instead, the research team developed a method that takes advantage of tiny particles that already float in the bloodstream called megakaryocytic microparticles. The researchers found that they could load these microparticles with gene-regulating material and that they would infiltrate only the desired stem cells because of distinctive properties on the surface of the microparticles.

According to the university, with more development, this technology could be useful in treating inherited blood disorders such as sickle cell anemia, which causes abnormally shaped red blood cells, and thalassemia, which disrupts the production of the blood protein hemoglobin. The methods developed by the researchers could also be used to deliver personalized medicine because the microparticles can be individually generated and stored frozen for each patient, said Papoutsakis.

“A lot of researchers are trying to deliver DNA, nucleic acids, or drugs to target hematopoietic stem cells,” said Papoutsakis in a university press release. “This is the right cell to target because it gives rise to all blood cells.”

In theory, altering those cells would allow for the prevention of the genetic defect for most or all of the patient’s life, as stated by the university. Currently, Papoutsakis is collaborating with Emily Day, an expert in nanomedicine and assistant professor of biomedical engineering at UD, to explore more ways to deliver this material.

Source: University of Delaware