1. Delivery of RNA-based molecules to human hematopoietic stem and progenitor cells for modulation of gene expression.
- Author
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Diener Y, Bosio A, and Bissels U
- Subjects
- Genetic Therapy methods, Hematopoietic Stem Cell Transplantation, Humans, MicroRNAs chemistry, MicroRNAs genetics, RNA administration & dosage, RNA chemistry, RNA, Antisense chemistry, RNA, Messenger chemistry, RNA, Messenger genetics, Transfection methods, Cell Engineering, Gene Expression, Gene Transfer Techniques, Hematopoietic Stem Cells metabolism, RNA genetics
- Abstract
Gene modulation of human hematopoietic stem and progenitor cells (HSPCs) harbors great potential for therapeutic application of these cells and presents a versatile tool in basic research to enhance our understanding of HSPC biology. However, stable genetic modification might be adverse, particularly in clinical settings. Here, we review a broad range of approaches to transient, nonviral modulation of protein expression with a focus on RNA-based methods. We compare different delivery methods and describe the usefulness of RNA molecules for overexpression as well as downregulation of proteins in HSPCs., (Copyright © 2016 ISEH - International Society for Experimental Hematology. Published by Elsevier Inc. All rights reserved.)
- Published
- 2016
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