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.
Type:
Grant
Filed:
December 15, 1998
Date of Patent:
April 22, 2003
Assignee:
Genetic Therapy, Inc.
Inventors:
Paul L. Hallenbeck, Yung-Nien Chang, Yawen L. Chiang