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US 6,551,587 B2Granted
Vectors for tissue-specific replication
AssigneeGenetic Therapy, Inc.
InventorsPaul L. Hallenbeck, Yung-Nien Chang, Yawen L. Chiang
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AssigneeGenetic Therapy, Inc.
InventorsPaul L. Hallenbeck, Yung-Nien Chang, Yawen L. Chiang
Estimating the term…
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 tissue-specific replication-conditional adenovirus vector comprising a heterologous tissue-specific transcriptional regulatory sequence operably linked to the coding region of a gene that is essential for replication of said vector.
A replication-conditional adenovirus vector comprising the coding region of an adenovirus early gene essential for propagation under the transcriptional control of a carcinoembryonic antigen (CEA) response element.
An isolated cell containing a tissue-specific replication-conditional adenovirus vector, said vector comprising a heterologous tissue-specific transcriptional regulatory sequence operably linked to the coding region of a gene that is essential for replication of said vector, wherein said transcriptional regulatory sequence functions in said cell so that replication of the vector occurs in said cell.
An isolated cell containing a tissue-specific replication-conditional adenovirus virion, said virion comprising a heterologous tissue-specific transcriptional regulatory sequence operably linked to the coding region of a gene that is essential for replication of said virion, wherein said transcriptional regulatory sequence functions in said cell so that replication of the virion occurs in said cell.
A tissue-specific replication-conditional adenoviral virion comprising a heterologous tissue-specific transcriptional regulatory sequence operably linked to the coding region of a gene that is essential for replication of said virion.
Dependencies are inferred from the claim text.