Paul Hallenbeck - Gaithersburg MD, US Yung-Nien Chang - Cockysville MD, US Yawen Chiang - Fremont CA, US
International Classification:
A61K048/00 C12N015/86
US Classification:
424/093200, 435/456000, 435/320100
Abstract:
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.
Paul Hallenbeck - Gaithersburg MD, US Yung-Nien Chang - Cockysville MD, US Yawen Chiang - Fremont CA, US
International Classification:
A61K048/00 C12N015/86 C12N005/08
US Classification:
424/093210, 435/456000, 435/320100, 435/366000
Abstract:
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.
Paul Hallenbeck - Gaithersburg MD, US Yung-Nien Chang - Cockysville MD, US Yawen Chiang - Fremont CA, US
International Classification:
A61K048/00 C12N015/86 C12N005/08
US Classification:
424/093210, 435/320100, 435/366000, 435/456000
Abstract:
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.
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