Home Department: Biology
Mentor: Matthew Porteus (Pediatrics - Stem Cell Transplantation)

“Inducible EPOR Signaling as a Means to Enrich for Edited Erythrocytes in vivo”

Erythropoietin regulates the proliferation and differentiation of erythroid precursor cells, and is mediated by the erythropoietin receptor (EPOR). Clinical genetics studies have revealed the existence of a mutant EPOR where affected individuals present with elevated levels of red blood cells (RBCs) but minimal adverse effects. The Porteus lab has postulated that by introducing this EPOR mutation into edited hematopoietic stem cells (HSCs), they can bias a heterogeneous population of edited HSCs towards producing a greater proportion of edited RBCs; bypassing a major therapeutic bottleneck in gene therapy. By understanding the structure of EPOR, and using previous work in the Porteus lab Kiran has hypothesized that he can go one step further and create a chimeric, small-molecule sensitive EPOR that brings therapeutic control over the proportion of edited, differentiated RBCs.