Dana-Farber-Cancer Institute Inc.

Children's Medical Center Corporation

Alan Engelman

Joseph G Sodroski

Judy Lieberman

Nan Yan

Richard Lu

Wolfgang Hofmann

Described herein are methods and compositions for the inhibition of retroviral integration and replication. The methods and compositions inhibit the activity of one or more components of the SET complex or base excision repair enzymes and induce autointegration of retroviral double-stranded nucleic acid.

Compositions and methods for inhibition of retroviruses

The invention is directed to a lentiviral vector system that can be used for episomal replication of a desired gene. The vector system contains a first vector containing a lentiviral gag gene encoding a lentiviral gag protein, a second vector containing an env gene encoding a functional envelope protein, and a lentiviral pol gene encoding a lentiviral pol protein. The pol protein is at least integrase, but that the integrase has been modified so that it is not capable of integration. This pol gene can be on the first or second vectors or on a third vector. The lentiviral gag and pol and the envelope protein are not on a single vector, and these vectors do not contain nucleotides to effectively package lentiviral RNA. The system also contains another vector having a nucleic acid sequence encoding a target molecule operably linked to a component of an episomal replicon and a lentiviral packaging sequence.

Episomally replicating lentiviral vectors

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Brookline, MA, United States of America

Alan Engelman