Title:
薬理学的に誘導される導入遺伝子アブレーション系
Document Type and Number:
Japanese Patent JP5922095
Kind Code:
B2
Abstract:
The present invention relates to gene therapy systems designed for the delivery of a therapeutic product to a subject using replication-defective virus composition(s) engineered with a built-in safety mechanism for ablating the therapeutic gene product, either permanently or temporarily, in response to a pharmacological agent—preferably an oral formulation, e.g., a pill. The invention is based, in part, on the applicants' development of an integrated approach, referred to herein as PITA (Pharmacologically Induced Transgene Ablation), for ablating a transgene or negatively regulating transgene expression. In this approach, replication-deficient viruses are used to deliver a transgene encoding a therapeutic product (an RNA or a protein) so that it is expressed in the subject, but can be reversibly or irreversibly turned off by administering the pharmacological agent; e.g., by administration of a small molecule that induces expression of an ablator specific for the transgene or its RNA transcript.
Inventors:
Wilson, The Ames M
Chiang, Xu-Yen
Anna Pee, Torreia Koba
Chiang, Xu-Yen
Anna Pee, Torreia Koba
Application Number:
JP2013502701A
Publication Date:
May 24, 2016
Filing Date:
March 28, 2011
Export Citation:
Assignee:
The Trustees of the University of Pennsylvania
International Classes:
C12N15/09; A61K35/76; A61K38/22; A61K38/44; A61K39/395; A61K48/00; A61P1/16; A61P3/10; A61P7/04; A61P25/00; A61P31/18; C12N5/10; C12N7/04; G01N33/68
Domestic Patent References:
| JP2003033179A | ||||
| JP2009171880A |
Foreign References:
| DE10023887A1 | ||||
| WO2008051854A1 | ||||
| WO2009146179A1 | ||||
| WO2002070740A1 |
Other References:
MOLECULAR THERAPY, 2006,vol. 13, no. 1,p.160-166
ウイルス, 2007, vol.57, no.1, p.45-56
NATURE BIOTECHNOLOGY, 2007,vol. 25, no. 7,p.778-785
ウイルス, 2007, vol.57, no.1, p.45-56
NATURE BIOTECHNOLOGY, 2007,vol. 25, no. 7,p.778-785
Attorney, Agent or Firm:
Patent business corporation Odashima patent office