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OwnerPaul L. Hallenbeck, Yung-Nien Chang +1inventors
InventorsPaul L. Hallenbeck, Yung-Nien Chang, Yawen L. Chiang
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The invention generally relates to targeted gene therapy using recombinant vectors and particularly adenovirus vectors. The invention specifically relates to replication-conditional vectors and methods for using them. Such vectors are able to selectively replicate in a target tissue to provide a therapeutic benefit from the presence of the vector per se or from heterologous gene products expressed from the vector and distributed throughout the tissue. In such vectors, a gene essential for replication is placed under the control of a heterologous tissue-specific transcriptional regulatory sequence. Thus, replication is conditioned on the presence of a factor(s) that induces transcription or the absence of a factor(s) that inhibits transcription of the gene by means of the transcriptional regulatory sequence with this vector; therefore, a target tissue can be selectively treated.
A vector capable of tissue-specific replication comprising:
A method for distributing a polynucleotide in a tissue in vivo, comprising introducing a replication-conditional vector containing said polynucleotide into said tissue, wherein said vector contains a gene essential for vector replication, the coding region of which gene is operably linked to a transcriptional regulatory sequence that functions specifically in said tissue so that replication of the vector occurs in said tissue and not in a tissue in which said transcriptional regulatory sequence does not function.
A cell containing a vector capable of tissue-specific replication, said vector comprising
A cell containing a virion capable of tissue-specific replication, said virion comprising
Dependencies are inferred from the claim text.