Home Department: Biology
Supported by: Bio-X
Mentor: Matthew Porteus, Pediatrics

Sickle cell disease (SCD) is a genetic disease affecting millions worldwide, in which mutant hemoglobin causes blood cells to become very rigid and hamper blood flow or even cell destruction. Joab will be looking at ways to optimize gene editing in hematopoietic stem cells (HSPCs) to combat SCD since gene edited HSPCs may be a potential cure for sickle cell disease. Joab's summer project, therefore, will entail testing one hypothesis that might increase the frequency of gene correction as a step towards potentially curing SCD.

Poster presented at the Stanford Bio-X Interdisciplinary Initiatives Symposium on August 26, 2015:

Optimizing HR of HBB Locus Using CRISPR/Cas9 and RAAV6 in HPSCs

Joab Camarena¹, Daniel Dever¹, Ayal Hendel¹, Matthew Porteus¹
[Department of Pediatrics¹, Stanford University]