Abstract: Genetically engineered cells and microorganisms are provided that produce fatty alcohols from the fatty acid biosynthetic pathway, as well as methods of their use.

Abstract: A method of treating polycystic kidney disease by administering an oligonucleotide antisense to c-myc is described. The antisense oligonucleotide is preferably a morpholino oligonucleotide.

Abstract: A composition antisense oligomer directed to an mRNA preferentially expressed together with methods for treating stem cells with such a composition to increase the number of lineage committed progenitor cells and their progeny, and/or slow the growth of cancer cells. Also described is the use of such compositions and antisense oligonucleotide-treated stem cells in methods for treatment of cancer.

Abstract: Recombinant lentiviruses and transfer vectors for transgene delivery as well as methods for gene therapy using such vectors are disclosed. The invention provides a third generation lentiviral packaging system and a set of vectors for producing recombinant lentiviruses, as well as novel tissue specific enhancer and promoter elements useful for optimizing liver specific transgene delivery. The transgene is preferably a blood clotting factor such as human factor IX (hFIX) or human factor VIII (hFVIII) and can be used for treatment of hemophilia.

Abstract: A method for selecting packaging cells that express high levels of gag/pol is provided.

Abstract: The present invention provides a retroviral gene delivery system that resists complement inactivation through the incorporation of a complement regulatory protein into retroviral particles. In particular, the present invention provides a lentiviral packaging system comprising at least two vectors: a first vector which comprises a nucleotide sequence comprising a gag, a pol, or gag and pol genes; and a second vector which comprises a nucleotide sequence comprising a gene that encodes a complement regulatory protein (CRP) and, optionally, a gene that encodes a heterologous or functionally modified envelope protein. The genes encoding a heterologous or functionally modified envelope protein and a CRP are provided either together in a second nucleotide sequence, or separately in second and third nucleotide sequences. Producer cells comprising the packaging constructs of the present invention and a transgene can be used to produce recombinant retroviral particles for transgene delivery.

Abstract: Polypeptides of adeno-associated virus (AAV) that bind to AAV antibodies or block binding of AAV to mammalian cells are described. Derivatives of peptides can be less immunogenic, enhance binding to cells, render a virus tissue specific and so on. The nucleic acid sequence encoding those derivatives can be incorporated into a capsid encoding sequence to enable a virus to express such a derivative and be less immunogenic, have enhanced transduction efficiency or be tissue specific.

Abstract: Disclosed herein are replication-competent adenovirus vectors comprising co-transcribed first and second genes under transcriptional control of a heterologous, target cell-specific transcriptional regulatory element (TRE), wherein the second gene is under translational control of an internal ribosome entry site. Methods for the preparation and use of such vectors are also provided. The vectors provide target cell-specific virus replication in applications such as cancer therapy and gene therapy.

Abstract: A method of separating components having a given negative or positive charge and contained in a sample is disclosed. The method involves, in one embodiment, loading a microchannel with a sample, placed between a trailing-edge electrolyte having a selected concentration of a titratable species, and a leading-edge electrolyte.

Abstract: The invention relates to compositions comprising oligomers antisense to bacterial 16S or 23S rRNA and capable of selectively modulating the biological activity thereof, and methods for their use. More particularly, the invention relates to antisense oligomers directed to 16S or 23S rRNA found in one or more particular bacteria, or generally conserved among bacteria in general, and to pharmaceutical compositions and methods of treatment comprising the same.

Abstract: The subject invention concerns a recombinant adeno-associated virus vector characterized as being capable of delivering and expressing at least one mammalian gene into a genome of a mammalian host cell such that the expression of the gene is regulated in a tissue specific manner by cis-acting regulatory and promoter elements of the gene. A method of using this recombinant adeno-associated virus vector for therapeutic purposes is also provided.

Abstract: Suppressor tRNA's are used to regulate expression of transgenes that are toxic, or the expression thereof requires a factor that is toxic, to the host cell.

Abstract: The present invention relates to methods and compositions for increasing the production of high titre stocks of recombinant AAV (rAAV) through regulation of expression of the AAV REP and CAP proteins. The methods and compositions of the invention are based on the observation that the low level expression of the AAV REP protein increases the production of AAV viral capsid protein and efficiency of packaging resulting in production of higher titre recombinant viral stocks. The invention encompasses recombinant AAV vectors that direct the expression of AAV REP and CAP proteins and the use of such vectors for the production of novel stable cell lines capable of generating high titre rAAV vectors. The invention provides methods for regulating the expression of the AAV REP gene at the transcriptional and post-translational level.

Abstract: Replication-competent adenovirus vectors specific for target cells and methods of use of such viruses are provided. These adenoviruses comprise a first adenoviral gene under control of a cell specific heterologous (i.e., non-adenoviral) transcriptional regulatory element (TRE) and at least a second gene under control of a second heterologous TRE, where the heterologous TREs are different from each other in polynucleotide sequence but functional in the same cell. The adenoviral gene can be, for example, a gene required for adenoviral replication. The second gene can be, for example, a second adenoviral gene or a transgene, such as a gene which can contribute to cytotoxicity in the target cell. Adenoviral replication can be restricted to target cells in which the heterologous TREs are functional and thus, the adenovirus vectors can provide selective cytotoxicity to the target cells, particularly neoplastic cells.

Abstract: The present invention is directed to apple fruit-associated and Thi 1.3:actin fusion promoters capable of promoting the expression of heterologous genes. The invention provides apple fruit-associated and Thi 1.3:actin fusion promoters together with heterologous nucleic acid constructs, vectors, kits, transformation methods, transgenic plant cells and transgenic plants comprising such promoters.

Abstract: The present invention provides a method for targeting a particular mRNA sequence in vivo by oral administration of a morpholino antisense compound having uncharged phosphorus-containing backbone linkages. Also disclosed is a non-invasive method of detecting and quantitating the in vivo presence of RNA containing one or more selected target sequences. The method includes administering to a subject a nuclease-resistant antisense oligomer which hybridizes by Watson-Crick base pairing to a region of the target RNA with a Tm substantially greater than 37° C. The oligomer is able to complex intracellularly with target RNA, and is released from intracellular sites as a nuclease-resistant heteroduplex, which can then be measured in a body fluid sample, e.g., urine.

Abstract: A plant transformation expression cassette for transforming host plant cells with a selectable marker gene is disclosed. The cassette includes, operatively linked in sequence in a 5′ to 3′ direction, (i) a DNA promoter sequence from the rice beta-glucanase 9 (gns9) gene; (ii) a selectable marker gene, and (iii) a 3′ untranslated terminator region. Also disclosed are a expression cassette pair containing the transformation vector, a method of obtaining transformed monocots whose seeds produce a selected heterologous protein during sed germination, and a plant whose cells are transformed with the chimeric selectable marker gene.

Abstract: Disclosed are therapeutic compositions and methods for inducing cytotoxic T cell responses in vitro and in vivo. The therapeutic compositions consist of antigen presenting cells activated by contact with a polypeptide complex constructed by joining together a dendritic cell-binding protein and a polypeptide antigen. Also disclosed are expression vectors and systems for producing the polypeptide complexes.

Abstract: The present invention is directed to novel polynucleotides and the polypeptides encoded by them, each of which are specific to human prostate tumor cells. The present invention further provides chimeric polypeptide molecules comprising the polypeptides of the present invention fused to heterologous polypeptide sequences, and antibodies which bind to the polypeptides of the present invention. Also provided herein are methods for producing the polypeptides of the present invention, as are detection assays that detect the presence of tumor cells in tissue or bodily fluid samples and methods for identifying novel compositions which modulate the activity of prostate tumor antigens and the use of such compositions in diagnosis and treatment of disease.

Abstract: The present invention provides methods and compositions relating to cytomegalovirus (CMV) latent transcripts, latency-associated polypeptides and antibodies directed against such polypeptide. The polypeptides are encoded by CMV DNA sequences and are produced specifically during latent infection. Also provided are methods of detecting CMV in a sample, particularly CMV in a latent state. The methods include RT-PCR-based methods and immunodiagnostic methods.