The State University of New York

Wadie F Bahou

Patrick Hearing

Varsha Sitaraman

Ziv Sandalon

Dmitri V Gnatenko

Song Wu

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. The invention's compositions and methods find application in, for example, gene therapy and/or vaccines.

Adeno-associated-virus Rep sequences, vectors and viruses

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&#x3b2; reductase (BLVRB) activity or inhibiting the expression of BLVRB gene.

Methods for increasing platelet count by inhibiting biliverdin IX&#x3b2; reductase

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.

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Setauket, NY, United States of America

Wadie F Bahou