MULTIPLEXED-TARGETED ADENOVIRUS VECTORS AND THEIR USE

Abstract: the present application provides compositions and methods for increasing safety, selectivity, and efficacy of transduction of human cells, including human long-term repopulating and hematopoietic stem cells (lt-hsc) and human cancer cells, by adenovirus vectors through multiplexed targeting of virus attachment and internalization receptors. multiplexing targeting ad vector receptor specificities through the combination of i) restricting fiber-specific attachment receptors, ii) deleting the rgd amino acid motif from the penton base protein, and iii) inserting into ad penton base protein of peptides that lack the rgd amino acid motif and enable vector interaction with integrin classes expressed on target cells, allows for the improvement of safety, selectivity, and efficacy of vector-mediated transduction of human cells in vitro and after intravenous vector administration in vivo.

Inventor(s): Dmitry M. SHAYAKHMETOV, Nelson C. DI PAOLO

CPC Classification: A61K48/0041 (Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy)

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