The invention provides improved gene therapy vectors, compositions, and methods.

The present invention relates to combination treatments for cystic fibrosis, particularly combinations of modulators of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) and gene therapy.

Compositions and methods for delivering immune modulatory molecules to result in a therapeutic effect are disclosed. The compositions and methods use stably integrating lentiviral delivery systems. The methods are useful for therapeutically and prophylactically treating cancer such as leukemia.

A ribonucleic acid (RNA) that includes from 5 to 3: an RNA Booster sequence that includes or is the following ribonucleic acid sequence: mmsknkkkm, wherein: m indicates an adenine (a) or cytosine (c); s indicates a guanine (g) or a cytosine (c); k indicates a guanine (g) or a uracyl (u); n indicates any nucleotide; and a sequence of interest. Also, methods for robust transient RNA expression.

Disclosed herein are compositions and methods that can be used to express a nucleotide sequence in a specific cell type. The compositions can comprise nucleic acid constructs comprising a start codon; and an intron cassette. The intron cassette can comprise a cell specific exon sequence, a splice donor site, a branch site, and an acceptor site. The cell specific exon sequence is out of frame with the start codon and comprises one or more frameshift mutations. The compositions can be used to treat human diseases.

The present invention provides compositions and methods for light-activated cation channel proteins and their uses within cell membranes and subcellular regions. The invention provides for proteins, nucleic acids, vectors and methods for genetically targeted expression of light-activated cation channels to specific cells or defined cell populations. In particular the invention provides millisecond-timescale temporal control of cation channels using moderate light intensities in cells, cell lines, transgenic animals, and humans. The invention provides for optically generating electrical spikes in nerve cells and other excitable cells useful for driving neuronal networks, drug screening, and therapy.

The invention provides compositions and methods of treating subjects afflicted with a photoreceptor disorder. Methods for treating a subject suffering from a disorder characterized by photoreceptor cell degeneration are provided, wherein a gene encoding a photosensitive protein is introduced into a retinal cell of a subject. In one aspect of the invention, the retinal cells which receive the photosensitive protein include non-photoreceptor cells such as horizontal cells, amacrine cells, bipolar cells, and ganglion cells.

The present relation relates to a transgenic Vero cell line expressing CD4 and CCR5. The present invention encompasses the preparation and purification of immunogenic compositions which are formulated into the vaccines of the present invention.

Disclosed herein are recombinant CMV vectors which may comprise a heterologous antigen that can repeatedly infect an organism while inducing a CD8+ T cell response to immunodominant epitopes of the heterologous antigen. The CMV vector may comprise a deleterious mutation in the US11 glycoprotein or a homolog thereof.

The present invention provides compositions and methods for rescuing FANCA expression in cells with diminished or no FANCA gene product. In particular, methods and compositions for gene therapy of Fanconi anemia are disclosed.