Abstract: The present disclosure provides methods of treating a human having a disease or disorder that would benefit from increasing platelet counts. The method involves inhibiting the enzyme activity of biliverdin IX? reductase (BLVRB) activity or inhibiting the expression of BLVRB gene.

Abstract: The invention provides adeno-associated virus (AAV) replication (Rep) sequences. In one embodiment, the invention provides nucleotide sequences encoding a chimeric protein, wherein the encoded chimeric protein contains a wild type AAV Rep inhibitory amino acid sequence, and wherein the nucleotide sequences contain a scrambled and/or deoptimized polynucleotide sequence encoding the wild type AAV Rep inhibitory amino acid sequence. The invention provides vectors, cells, and viruses containing the invention's sequences. Also provided are methods for detecting portions of the AAV Rep inhibitory amino acid sequence, which reduce replication and/or infection and/or productive infection by viruses. The invention's compositions and methods are useful for site-specific integration and/or expression of heterologous sequences by recombinant adeno-associated virus (rAAV) vectors and by rAAV virus particles, such as hybrid viruses (e.g., Ad-AAV) comprising such vectors.

Abstract: The present disclosure provides methods of treating a human having a disease or disorder that would benefit from increasing platelet counts. The method involves inhibiting the enzyme activity of biliverdin IX? reductase (BLVRB) activity or inhibiting the expression of BLVRB gene.

Abstract: The invention provides adeno-associated virus (AAV) replication (Rep) sequences. In one embodiment, the invention provides nucleotide sequences encoding a chimeric protein, wherein the encoded chimeric protein contains a wild type AAV Rep inhibitory amino acid sequence, and wherein the nucleotide sequences contain a scrambled and/or deoptimized polynucleotide sequence encoding the wild type AAV Rep inhibitory amino acid sequence. The invention provides vectors, cells, and viruses containing the invention's sequences. Also provided are methods for detecting portions of the AAV Rep inhibitory amino acid sequence, which reduce replication and/or infection and/or productive infection by viruses. The invention's compositions and methods are useful for site-specific integration and/or expression of heterologous sequences by recombinant adeno-associated virus (rAAV) vectors and by rAAV virus particles, such as hybrid viruses (e.g., Ad-AAV) comprising such vectors.

Abstract: The present disclosure provides methods of treating a human having a disease or disorder that would benefit from increasing platelet counts. The method involves inhibiting the enzyme activity of biliverdin IX? reductase (BLVRB) activity or inhibiting the expression of BLVRB gene.

Abstract: The present disclosure provides methods of treating a human having a disease or disorder that would benefit from increasing platelet counts. The method involves inhibiting the enzyme activity of biliverdin IX? reductase (BLVRB) activity or inhibiting the expression of BLVRB gene.

Abstract: The invention provides adeno-associated virus (AAV) replication (Rep) sequences. In one embodiment, the invention provides nucleotide sequences encoding a chimeric protein, wherein the encoded chimeric protein contains a wild type AAV Rep inhibitory amino acid sequence, and wherein the nucleotide sequences contain a scrambled and/or deoptimized polynucleotide sequence encoding the wild type AAV Rep inhibitory amino acid sequence. The invention provides vectors, cells, and viruses containing the invention's sequences. Also provided are methods for detecting portions of the AAV Rep inhibitory amino acid sequence, which reduce replication and/or infection and/or productive infection by viruses. The invention's compositions and methods are useful for site-specific integration and/or expression of heterologous sequences by recombinant adeno-associated virus (rAAV) vectors and by rAAV virus particles, such as hybrid viruses (e.g., Ad-AAV) comprising such vectors.

Abstract: The invention provides adeno-associated virus (AAV) replication (Rep) sequences. In one embodiment, the invention provides nucleotide sequences encoding a chimeric protein, wherein the encoded chimeric protein contains a wild type AAV Rep inhibitory amino acid sequence, and wherein the nucleotide sequences contain a scrambled and/or deoptimized polynucleotide sequence encoding the wild type AAV Rep inhibitory amino acid sequence. The invention provides vectors, cells, and viruses containing the invention's sequences. Also provided are methods for detecting portions of the AAV Rep inhibitory amino acid sequence, which reduce replication and/or infection and/or productive infection by viruses. The invention's compositions and methods are useful for site-specific integration and/or expression of heterologous sequences by recombinant adeno-associated virus (rAAV) vectors and by rAAV virus particles, such as hybrid viruses (e.g., Ad-AAV) comprising such vectors.

Abstract: The present disclosure provides methods of treating a human having a disease or disorder that would benefit from increasing platelet counts. The method involves inhibiting the enzyme activity of biliverdin IX? reductase (BLVRB) activity or inhibiting the expression of BLVRB gene.

Abstract: The invention provides adeno-associated virus (AAV) replication (Rep) sequences. In one embodiment, the invention provides nucleotide sequences encoding a chimeric protein, wherein the encoded chimeric protein contains a wild type AAV Rep inhibitory amino acid sequence, and wherein the nucleotide sequences contain a scrambled and/or deoptimized polynucleotide sequence encoding the wild type AAV Rep inhibitory amino acid sequence. The invention provides vectors, cells, and viruses containing the invention's sequences. Also provided are methods for detecting portions of the AAV Rep inhibitory amino acid sequence, which reduce replication and/or infection and/or productive infection by viruses. The invention's compositions and methods are useful for site-specific integration and/or expression of heterologous sequences by recombinant adeno-associated virus (rAAV) vectors and by rAAV virus particles, such as hybrid viruses (e.g., Ad-AAV) comprising such vectors.

Abstract: The invention provides adeno-associated virus (AAV) replication (Rep) sequences. In one embodiment, the invention provides nucleotide sequences encoding a chimeric protein, wherein the encoded chimeric protein contains a wild type AAV Rep inhibitory amino acid sequence, and wherein the nucleotide sequences contain a scrambled and/or deoptimized polynucleotide sequence encoding the wild type AAV Rep inhibitory amino acid sequence. The invention provides vectors, cells, and viruses containing the invention's sequences. Also provided are methods for detecting portions of the AAV Rep inhibitory amino acid sequence, which reduce replication and/or infection and/or productive infection by viruses. The invention's compositions and methods are useful for site-specific integration and/or expression of heterologous sequences by recombinant adeno-associated virus (rAAV) vectors and by rAAV virus particles, such as hybrid viruses (e.g., Ad-AAV) comprising such vectors.

Abstract: The present invention relates to a method to determine the gene expression profile of thrombocytotic sensitive genes. The method comprises the following steps; obtaining hematologic samples from subjects in a training set, analyzing the obtained hematologic samples with a microarray, measuring the expression values of each gene on the microarray, performing analysis to identify differentially expressed genes in the training set among three cohorts of thrombocytosis, obtaining hematologic samples from subjects in an independent testing set, and validating the identity of the differentially expressed genes in the independent testing set among the three cohorts of thrombocytosis. The invention also relates to a method to distinguish thrombocytosis cohorts. The method includes the following steps; obtaining a hematologic sample from a subject, determining gene expression of a biomarker subset, analyzing gene expression of the biomarker subset, and classifying the subject into one of three cohorts.

Abstract: The invention provides a non-human mammal or a cell line that has a targeted gene disruption in an endogenous Iqgap2 gene. The invention also provides methods of identifying a compound as a therapeutic agent for the treatment of hepatocellular carcinoma and methods of treating or preventing hepatocellular carcinoma.

Abstract: The invention provides recombinant vectors including adenovirus/adeno-associated virus (Ad/AAV) vectors and mini-adenovirus (mAd) vectors. Further, the invention provides cells containing these vectors, and methods for making and using the vectors and cells. The compositions and methods of the invention are useful in transferring nucleotide sequences of interest into a cell, including, but not limited to, in gene therapy applications.

Abstract: The invention provides recombinant vectors including adenovirus/adeno-associated virus (Ad/AAV) vectors and mini-adenovirus (mAd) vectors. Further, the invention provides cells containing these vectors, and methods for making and using the vectors and cells. The compositions and methods of the invention are useful in transferring nucleotide sequences of interest into a cell, including, but not limited to, in gene therapy applications.

Abstract: The invention provides recombinant vectors including adenovirus/adeno-associated virus (Ad/AAV) vectors and mini-adenovirus (mAd) vectors. Further, the invention provides cells containing these vectors, and methods for making and using the vectors and cells. The compositions and methods of the invention are useful in transferring nucleotide sequences of interest into a cell, including, but not limited to, in gene therapy applications.