Targeting Lentiviral Vectors to Specific Cells and Tissues
The current technology proposes the use of a lentiviral vector to target specific human immune cells as well as certain types of cancer cells. The approach has been validated using a mouse model for metastatic melanoma [Morizono K, et. al, 2005. Lentiviral vector retargeting to P-Glycoprotein on metastatic melanoma via intravenous injection. Nat Med. 11(3):346-52.] and for prostate cancer in a bone metastatic model. Current work is being done on central nervous system targeting in a mouse model which is directed to the blood brain barrier. [In principle, the approach can be generally applied to direct gene therapy to any desired cell or tissue in the body.]
Patent Number: US8449875B2
Application Number: US2009623265A
Inventor: Chen, Irvin S.Y. | Morizono, Kouki
Priority Date: 3 Jun 2004
Priority Number: US8449875B2
Application Date: 20 Nov 2009
Publication Date: 28 May 2013
IPC Current: A01N006300 | A61K003912 | A61K0039193 | A61K003921 | A61K0039395 | C12N001500 | C12N0015867 | A61K004800
US Class: 4240932 | 4241301 | 4241991 | 4242071 | 4242181
Assignee Applicant: The Regents of the University of California
Title: Targeting pseudotyped retroviral vectors
Usefulness: Targeting pseudotyped retroviral vectors
Summary: The pseudotyped, targeted retroviral vector is useful for specific targeting of therapeutic genes to affected cells while maintaining stable transduction and long term expression. It is also useful for treating, preventing, or diagnosing a disease state.
Novelty: New pseudotyped, targeted retroviral vector comprises a mutated Sindbis envelope and a targeting moiety linked to the Sindbis envelope, useful for specific targeting of therapeutic genes to affected cells
生物医学
DNA /基因工程
8449875
State of Development Dr. Irvin Chens lab is currently engaged in the further development of this technology through a five year, $1 million dollar federal grant. Background The main obstacle facing the use of gene therapy as a successful therapeutic modality for humans is the inability to accurately target vectors to organs and cells. Adenoviral and adeno-associated vectors have been associated with mutagenesis and liver toxicity. Other vectors have suffered from ineffective entry and failure to provide stable long term expression. Tech ID/UC Case 20067/2000-128-0 Related Cases 2000-128-0
美国
