"The microvesicles in the purified populations provided herein contain or are otherwise associated with the viral particles to produce a MAV. The characteristic of a MAV according to the invention is a viral particle, e.g., viral capsid, that contains a nucleic acid encoding a gene of interest, where the MAV is further surrounded by or otherwise associated with a membrane derived from a virus-producing cell. For example, the MAV is or is derived from adenovirus, including replication defective and competent vectors; lentivirus; retrovirus; herpes virus, including replication defective and competent vectors; adeno-associated virus (AAV); alphavirus, including, for example, sindbis virus, semliki forest virus, Venezuelan equine encephalitis virus,
"These viral particles are useful in a variety of applications such as, for example, gene transfer applications, including gene replacement, gene repair, and/or mRNA knockdown therapeutic applications, and vaccine applications. These MAV are useful in gene transfer applications where conventional viral vectors including, for example, conventional AAV vectors, are inefficient or incapable of infection of a target cell or tissue type.
"In some embodiments, the vector in the MAV is an adeno-associated viral (AAV) vector. The AAV capsids are incorporated into microvesicles that can be purified from the supernatant of the producer cell line. These microvesicles are fully competent for transduction of cells in culture as well as in vivo. In vivo delivery of the vector is markedly enhanced with the associated microvesicles and extended to multiple tissues compared to AAV capsids of those same serotypes as assessed by in vivo bioluminescence imaging.
"The purified populations of microvesicles and compositions containing these purified populations are useful in a variety of therapeutic indications. The ability of vector producer cells to insert viral particles inside microvesicles or the vector's association with microvesicles components (on the surface and/or interior) offer many opportunities for gene therapy applications. First, the viral vector may be co-delivered with therapeutic proteins, mRNA or microRNA inside the microvesicle. The microvesicles are useful for enhancing the potency and bioavailability of a poorly soluble anti-inflammatory drug (see e.g., Mol Ther. 2010 September; 18(9):1606-14. Epub 2010 Jun. 22). Second, as other viral particles, e.g. HIV, can be packaged in or otherwise associated with microvesicles (see e.g., PNAS,
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