Abstract: The present invention provides new zinc finger proteins and zinc finger nuclease (ZFNs) that find particular using in repairing the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Abstract: The present invention relates to therapeutic agents comprising miR-138, a miR-138 mimic, a SIN3A RNAi molecule, or a an anti-SIN3A RNAi molecule, and/or an anti-SIN3A antisense oligonucleotide (ASO) or other agent that suppresses SIN3A expression, a small molecule drug that interferes with SIN3A activity or whose actions mimic the biological effects of SIN3A suppression and methods of use of these therapeutic agents to treat cystic fibrosis.

Abstract: The present invention provides new zinc finger proteins and zinc finger nuclease (ZFNs) that find particular using in repairing the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Abstract: A method of co-expressing a portion of the VSV G protein gene or a truncated “stem” portion with GP64 and a retrovirus increases the titer of retroviral vectors. A truncated VSV G protein, preferably comprised of a small segment from the C-terminal portion of the ectodomain plus the transmembrane (TM) and cytoplasmic tail (CTD) domains of VSV G, co-expressed with retroviral vectors, enhances the production titers of the retroviral vectors. A preferred embodiment uses a VSV G construct that includes an N-terminal c-Myc epitope plus 42 amino acids from the C-terminal portion of the ectodomain, 20 amino acids from the predicted TM domain, and 29 amino acids from the predicted CTD of the VSV G protein.

Abstract: A method of co-expressing a portion of the VSV G protein gene or a truncated “stem” portion with GP64 and a retrovirus increases the titer of retroviral vectors. A truncated VSV G protein, preferably comprised of a small segment from the C-terminal portion of the ectodomain plus the transmembrane (TM) and cytoplasmic tail (CTD) domains of VSV G, co-expressed with retroviral vectors, enhances the production titers of the retroviral vectors. A preferred embodiment uses a VSV G construct that includes an N-terminal c-Myc epitope plus 42 amino acids from the C-terminal portion of the ectodomain, 20 amino acids from the predicted TM domain, and 29 amino acids from the predicted CTD of the VSV G protein.

Abstract: The present invention provides new zinc finger proteins and zinc finger nuclease (ZFNs) that find particular using in repairing the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Abstract: The present invention concerns the methods and compositions related to pseudotyped viral vectors. Embodiments of the invention include pseudotyping expression cassettes that include nucleic acid elements for enhancing the titer of pseudotyped viral particles. Embodiments of the invention include novel methods and compositions related to making high titer pseudotyped retroviral vector compositions. A heterologous envelope glycoprotein is typically incorporated into the virus during the budding or virus production process. Certain embodiments of the invention include pseudotyped retroviral vectors comprising a heterologous envelope glycoprotein derived from a Jaagsiekte sheep retrovirus (JSRV env). Pseudotyped viruses or viral particles may have a modified host range that is influenced by the properties of the heterologous envelope glycoprotein.

Abstract: The present invention provides novel pseudotyped retroviral vectors that can transduce human and other cells. Vectors are provided that are packaged efficiently in packaging cells and cell lines to generate high titer recombinant virus stocks expressing novel envelope glycoproteins. The present invention further relates to compositions for gene therapy.

Abstract: Embodiments of the invention include compositions comprising and methods utilizing a retroviral integrase complex comprising a recombinant integrase having a domain comprising a non-native protein binding site, and a DNA binding protein comprising a DNA binding domain and a peptide binding domain that binds the non-native protein binding site of the recombinant integrase.

Abstract: The present invention is directed to small interfering RNA molecules targeted against a gene of interest in respiratory epithelial cells, and methods of using these RNA molecules.

Abstract: The present invention employs an iterative application of BLAST and Hidden Markov Model (HMM) based searches which identified 34 ?-defensin genes in the human genome and 48 in the mouse genome. The present invention relates to novel antimicrobial peptides and derivatives thereof as well as the ?-defensin genes encoding the peptides. The invention further relates to methods of use of the peptides including a method of inhibiting microbial growth by administering an effective amount of the peptide alone or in combination with other antimicrobial agents or antibiotics.

Abstract: The present invention concerns compositions and methods related to the use of peptides and polypeptides corresponding to all or part of the SPLUNC1 and LPLUNC1 proteins. Particular aspects of the invnetion include the use of SPLUNC1 and LPLUNC1 peptides and polypeptides as antimicrobials, anti-inflammatory, and immune modulatory agents.

Abstract: The present invention provides novel pseudotyped retroviral vectors that can transduce human and other cells. Vectors are provided that are packaged efficiently in packaging cells and cell lines to generate high titer recombinant virus stocks expressing novel envelope glycoproteins. The present invention further relates to compositions for gene therapy.

Abstract: The present invention provides novel pseudotyped retroviral vectors that can transduce human and other cells. Vectors are provided that are packaged efficiently in packaging cells and cell lines to generate high titer recombinant virus stocks expressing novel envelope glycoproteins. The present invention further relates to compositions for gene therapy.

Abstract: The invention provides a method to introduce a selected nucleic acid sequence into an airway epithelial cell that involves contacting the airway epithelial cell with a pseudotyped retrovirus that includes a glycoprotein in which a portion of an O-glycosylation region within the glycoprotein has been deleted and a retroviral capsid that includes the selected nucleic acid sequence. The invention also provides a method to reduce or eliminate the symptoms of cystic fibrosis in a mammal that involves contacting an airway epithelial cell of the mammal with a pseudotyped retrovirus that includes a glycoprotein in which a portion of an O-glycosylation region within the glycoprotein has been deleted and a retroviral capsid that includes a nucleic acid sequence that encodes a cystic fibrosis transmembrane regulator protein.

Abstract: The present invention provides novel pseudotyped retroviral vectors that can transduce human and other cells. Vectors are provided that are packaged efficiently in packaging cells and cell lines to generate high titer recombinant virus stocks expressing novel envelope glycoproteins. The present invention further relates to compositions for gene therapy.

Abstract: The present invention concerns the methods and compositions related to pseudotyped viral vectors. Embodiments of the invention include pseudotyping expression cassettes that include nucleic acid elements for enhancing the titer of pseudotyped viral particles. Embodiments of the invention include novel methods and compositions related to making high titer pseudotyped retroviral vector compositions. A heterologous envelope glycoprotein is typically incorporated into the virus during the budding or virus production process. Certain embodiments of the invention include pseudotyped retroviral vectors comprising a heterologous envelope glycoprotein derived from a Jaagsiekte sheep retrovirus (JSRV env). Pseudotyped viruses or viral particles may have a modified host range that is influenced by the properties of the heterologous envelope glycoprotein.