CRISPR-Cas9-Mediated ELANE Mutation Correction in Hematopoietic Stem and Progenitor Cells to Treat Severe Congenital Neutropenia
Ngoc Tung Tran, Robin Graf, Annika Wulf-Goldenberg, Maria Stecklum, Gabriele Strauß, Ralf Kühn, Christine Kocks, Klaus Rajewsky, Van Trung Chu
Topics & Concepts
Congenital NeutropeniaBiologyProgenitor cellHaematopoiesisCancer researchStem cellMutationImmunologyGeneticsGeneBlood disorders and treatmentsImmunodeficiency and Autoimmune DisordersErythrocyte Function and Pathophysiology