Adeno-Associated Virus Vectors—a Target of Cellular and Humoral Immunity—are Expanding Their Reach Toward Hematopoietic Stem Cell Modification and Immunotherapies

Abstract excerpt

gene therapy of monogenic diseases rely on adeno-associated virus (AAV) vectors. Advances in gene editing technologies and vector engineering have expanded the spectrum of target cells and, thus, diseases that can be addressed. Consequently, AAV vectors are now being explored to modify cells of the hematopoietic system, including hematopoietic stem and progenitor cells (HSPCs), to develop novel strategies to...

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