This disclosure relates to the use of size exclusion chromatography and/or size exclusion chromatography with multi-angle light scattering technology to characterize viral particles such as adeno-associated virus and lentivirus particles. The disclosed methods are also useful for estimating the titer of viral particles, determining the integrity of the viral particles and estimating the amount of DNA encapsidated in the viral particle.

Viral vectors, lentiviral particles, and modified cells are disclosed. They encode or express a small RNA capable of targeting the KIF11 gene. In embodiments, the viral vectors and lenti viral particles further comprise and a KIF11 gene whose non-coding region has been modified such that it is resistant to activity by the small RNA.

The invention is directed to a system comprising a lentivirus vector particle which encodes at least one guide RNA sequence that is complementary to a first DNA sequence in a host cell genome, a Cas9 protein, and optionally a donor nucleic acid molecule comprising a second DNA sequence. The invention also is directed to a method of altering a DNA sequence in a host cell using such a system, where the host cell can be in a human and the altered DNA can be of the human β-globin gene. The invention also is directed to a fusion protein comprising a Cas9 protein and a cyclophilin A (CypA) protein. The invention also is directed to sequences of vectors that can be used in the system and method.

Disclosed herein are virus-like particle (VLP)-based bivalent vaccine compositions. The compositions may comprise a spherical retroviral Group-specific Antigen (“Gag”) protein core and at least two Ebola glycoproteins. The at least two Ebola glycoproteins may be located at the exterior surface of the spherical Gag protein core, such that the VLP-based vaccine presents at least two Ebola glycoprotein antigens. In one aspect, the at least two Ebola glycoproteins are a Zaire (EBOV) glycoprotein, and a Sudan (SUDV) glycoprotein.

Viral vectors, lentiviral particles, and modified cells are disclosed. They encode or express a small RNA capable of targeting the KIF11 gene. In embodiments, the viral vectors and lenti viral particles further comprise and a KIF11 gene whose non-coding region has been modified such that it is resistant to activity by the small RNA.

A lentiviral vector system for expressing a lentiviral particle is disclosed. The lentiviral vector system includes a therapeutic vector. The lentiviral vector system produces a lentiviral particle that encodes a codon-optimized PAH for upregulating PAH expression in the cells of a subject afflicted with phenylketonuria (PKU).

The present invention relates to methods of modulating the activity of CAR-T cells in the treatment of diseases, disorders and conditions by the administration of an IL-10 agent. The invention further provides engineered CAR-T cells to express additional therapeutically effective agents. The present invention further provides improved pharmaceutical and therapeutic compostions and methods relating to the use of CAR-T cell therapies in the treatment of disease in mammalian subjects.

Described herein are Bovine immunodeficiency virus gag protein (“Bgag”) recombinant virus like particles (“VLPs”) comprising one or more different types of target pathogen proteins. Also described, are compositions comprising the novel Bgag VLPs and the methods of making and using the novel Bgag VLPs.

Provided for herein are mutant VSV-G polypeptides, compositions comprising the same, and methods of using the same. Also provided for herein are polypeptides and compositions that bind to CD7 and uses thereof. Also provided for herein are polypeptides and compositions that bind to CD8 and uses thereof.

Provided for herein are mutant VSV-G polypeptides, compositions comprising the same, and methods of using the same. Also provided for herein are polypeptides and compositions that bind to CD7 and uses thereof. Also provided for herein are polypeptides and compositions that bind to CD8 and uses thereof.