Assessment of Allele-Specific Expression in Cells and Tissue

Tech ID: 19021 / UC Case 1996-B33-0

Brief Descripton

The success of gene therapy methods such as small fragment homologous recombination and cDNA-based gene therapies is often difficult to quantify. These methods often lack an endogenous selection mechanism that can be used to differentiate and quantify targeted cells. Therefore, it is difficult to monitor and map the success of gene therapy in patients. UCSF investigators have developed methods and compounds enabling the measurement of expression of mutated and non-mutated alleles in the tissue or cells of a human subject. The method, an in situ RT-PCR assay, can be used for diagnosis of allelic variation and the monitoring of gene therapy for a variety of gene-based diseases, especially cystic fibrosis.

Full Description

BACKGROUND: The success of gene therapy methods such as small fragment homologous recombination and cDNA-based gene therapies is often difficult to quantify. These methods often lack an endogenous selection mechanism that can be used to differentiate and quantify targeted cells. Therefore, it is difficult to monitor and map the success of gene therapy in patients.

DESCRIPTION: UCSF investigators have developed methods and compounds enabling the measurement of expression of mutated and non-mutated alleles in the tissue or cells of a human subject. The method, an in situ RT-PCR assay, can be used for diagnosis of allelic variation and the monitoring of gene therapy for a variety of gene-based diseases, especially cystic fibrosis.

Applications

Assessment of the efficacy of gene therapy, including small fragment homologous recombination (SFHR) and cDNA-based gene therapies Diagnosis of allelic variation in cystic fibrosis

Patent Status

Country	Type	Number	Dated	Case
United States Of America	Issued Patent	5,804,383	09/08/1998	1996-B33