In vivo gene delivery into hCD34+ cells in a humanized mouse model.


In vivo targeted gene delivery to hematopoietic stem cells (HSCs) would mean a big step forward in the field of gene therapy. This would imply that the risk of cell differentiation and loss of homing/-engraftment is reduced, as there is no need for purification of the target cell. In vivo gene delivery also bypasses the issue that no precise markers that… (More)
DOI: 10.1007/978-1-61779-095-9_15


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