Abstract: Compositions and methods are provided for modulating adeno-associated virus (AAV) infection. For example, compositions and methods are provided for enhancing permissiveness of a target cell to AAV infection (e.g., by increasing levels of AAVR (KIAA0319L) in the cell), for reducing permissiveness of a target cell to AAV infection (e.g., by reducing levels of AAVR in the cell), and for nucleic acid delivery (e.g., by (i) increasing permissiveness of a target cell to AAV infection, e.g., by increasing the amount of AAVR in the cell; and (ii) contacting the target cell with an AAV particle that includes a nucleic acid of interest). Also provided are screening methods and kits for practicing the methods of the disclosure.

Abstract: Compositions and methods are provided for modulating adeno-associated virus (AAV) infection. For example, compositions and methods are provided for enhancing permissiveness of a target cell to AAV infection (e.g., by increasing levels of AAVR (KIAA0319L) in the cell), for reducing permissiveness of a target cell to AAV infection (e.g., by reducing levels of AAVR in the cell), and for nucleic acid delivery (e.g., by (i) increasing permissiveness of a target cell to AAV infection, e.g., by increasing the amount of AAVR in the cell; and (ii) contacting the target cell with an AAV particle that includes a nucleic acid of interest). Also provided are screening methods and kits for practicing the methods of the disclosure.

Abstract: Methods and assays for treating a subject with a filovirus infection using an agent that inhibits Niemann-Pick CI (NPCI), VPSII, VPSI6, VPSI8, VPS33A, VPS39, VPS41, BLOCISI, BLOCIS2, GNPTAB, PIKFYVE, ARHGAP23 or FIG4. Methods for screening for an agent that treats and/or prevents infection of a subject with a filovirus, where the methods comprise determining whether the agent inhibits one or more of Niemann-Pick CI (NPCI), VPSII, VPSI6, VPSI8, VPS33A, VPS39, VPS41. BLOCISI, BLOCIS2, GNPTAB, PIKFYVE, ARHGAP23 or FIG4, wherein an agent that inhibits one or more of NPCI, VPSII, VPSI6, VPSI8, VPS33A, VPS39, VPS41, BLOCISI, BLOCIS2, GNPTAB, PIKFYVE, ARHGAP23 or FIG4 is a candidate for treating and/or preventing an infection with a filovirus and wherein an agent that does not inhibit NPCI, VPSII, VPSI6, VPSI8, VPS33A. VPS39, VPS41, BLOCISI, BLOCIS2, GNPTAB, PIKFYVE, ARHGAP23 or FIG4 is not a candidate for treating and/or preventing an infection with a filovirus.

Abstract: Methods and assays for treating a subject with a filovirus infection using an agent that inhibits Niemann-Pick CI (NPCI), VPSII, VPSI6, VPSI8, VPS33A, VPS39, VPS41, BLOCISI, BLOCIS2, GNPTAB, PIKFYVE, ARHGAP23 or FIG4. Methods for screening for an agent that treats and/or prevents infection of a subject with a filovirus, where the methods comprise determining whether the agent inhibits one or more of Niemann-Pick CI (NPCI), VPSII, VPSI6, VPSI8, VPS33A, VPS39, VPS41. BLOCISI, BLOCIS2, GNPTAB, PIKFYVE, ARHGAP23 or FIG4, wherein an agent that inhibits one or more of NPCI, VPSII, VPSI6, VPSI8, VPS33A, VPS39, VPS41, BLOCISI, BLOCIS2, GNPTAB, PIKFYVE, ARHGAP23 or FIG4 is a candidate for treating and/or preventing an infection with a filovirus and wherein an agent that does not inhibit NPCI, VPSII, VPSI6, VPSI8, VPS33A. VPS39, VPS41, BLOCISI, BLOCIS2, GNPTAB, PIKFYVE, ARHGAP23 or FIG4 is not a candidate for treating and/or preventing an infection with a filovirus.

Abstract: Described is a replication-competent virus capable of replication and having lytic capacity in target cells. The virus comprises in the genome thereof, at least one DNA sequence coding for a silencing factor functional in reducing expression of a target gene in the target cells, operably linked to one or more expression control sequences, functional in the target cells. The use thereof in the preparation of a medicament and the use thereof in a method for lysing target cells expressing a virus inhibitory factor are also described.