The Uab Research Foundation (the University of Alabama at Birmingham)

Other

Texas Tech University System

John Christopher Kappes

Xiaoyun Wu

Christie G Brouillette

Zhengrong Yang

Ina L Urbatsch

The present invention is directed to modified CFTR proteins or fragments thereof that contain single or multiple amino acid mutations to improve the structural stability of such CFTR proteins and/or fragments. Specifically, the modified CFTR proteins or fragment thereof differ from the wild-type human CFTR protein or fragment thereof by the presence of four or more mutations selected from V150D, M470V, S492P, F494N, S495P, A534P, I539T, G550E, G551D, R553Q, R555K, Q637R, S1255L, K1334G, S1359A, E1371Q, H1402S, Q1411D, and any combination thereof, such that the stability of the polypeptide is increased relative to that of the wild-type human CFTR polypeptide or fragment thereof.

Modified cystic fibrosis transmembrane conductance regulator (CFTR) polypeptides with increased stability and uses thereof

Trans-lentiviral gene transfer vectors, vector systems, vector particles, and methods for transduction of primary nondividing cells using the same, are described. Further described are modifications to the gene transfer vectors that improve transduction efficiency and/or gene expression, e.g., by incorporating the cis-acting sequences, PPT-CTS and/or WPRE, and derivatives or analogs thereof.

Trans-lentiviral vector particles and transduction of eukaryotic cells therewith

The present invention provides a composition of matter, comprising: DNA encoding a viral Vpx protein fused to DNA encoding a protein. In another embodiment of the present invention, there is provided a composition of matter, comprising: DNA encoding a viral Vpr protein fused to DNA encoding a protein. The present invention further provides DNA, vectors and methods for expressing a lentiviral pol gene in trans, independent of the lentiviral gag-pol. A gene transduction element is optionally delivered to a lentiviral vector according to the present invention. Also provided are various methods of delivering a virus inhibitory molecule to a target in an animal. Further provided is a pharmaceutical composition.

Fusion protein delivery system and uses thereof

Methods and reagents for the capture of primary HIV are provided. A cell line expressing CCR5, CXCR4 and CD4 receptors binds and is infected by primary HIV. The cell line contains a marker gene sequence, the marker gene sequence expressed in near linear quantities over at least two orders of magnitude in response to HIV infection. Primary HIV is amplified to create a primary virus stock through insertion of an amplicon gene into the receptor expressing cell line. HIV amplification occurs rapidly and is operative with noninfectious HIV through amplification in the presence of an infectivity complement. The present invention is useful in determining host HIV titer, drug sensitivity, HIV amplification, gene sequencing and co-receptor utilization.

Cell-based assay for immunodeficiency virus infectivity and sensitivity

The present invention provides a composition of matter, comprising: DNA encoding a viral Vpx protein fused to DNA encoding a virus inhibitory protein. In another embodiment of the present invention, there is provided a composition of matter, comprising: DNA encoding a viral Vpr protein fused to DNA encoding a virus inhibitory protein. Also provided are various methods of delivering a virus inhibitory molecule to a target in an animal. Further provided is a pharmaceutical composition.

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Birmingham, AL, United States of America

John Christopher Kappes