Description of Invention:
Gene therapy is a technique based on the idea that a genetic disorder can be treated by replacing a dysfunctional gene with a functional copy of that gene. Currently, retroviral vectors and adenoviral vectors are most frequently used for gene therapy clinical trials. Retroviral vectors provide long term gene expression and are capable of transferring genes into non-dividing cells, unlike their adenoviral counterparts. However, retroviral vectors often suffer from weak viral titers and inefficient encapsidation of the therapeutic gene, detracting from their therapeutic value. Thus, there is a need in the art for improved retroviral gene therapy vectors.
This technology family is directed to a retroviral vector system comprising a packaging vector and a transfer vector, and a method of using the vectors for gene therapy. The packaging vector is the result of an HIV-2 lentiviral vector containing mutations in sequences surrounding a splice donor site within the packaging signal. The transfer vector comprises mutations that render a splice donor site non-functional. These mutations increase the viral titer and expression/encapsidation of the transgene, but without a corresponding increase in the packaging of viral RNA. Thus, these vectors may address some of the pressing concerns with current gene therapy vectors systems.
Applications:
Improved lentivirus based vector system with practical application in gene therapy/gene transfer
Two vector system minimizes possibility of HIV infection
Packaging vector is a result of HIV-2 Lentivirus vector
Improved packaging and expression ability addresses current low viral titer problem
Market:
The only gene therapy product currently in the market was approved in China in 2004.
The R&D market of gene therapy is projected to grow to several billion dollars in the next 5 years.
Development Status:
The technology is currently in the pre-clinical stage of development.
Relevant Publication: SK Arya et al. Human immunodeficiency virus type 2 lentivirus vectors for gene transfer: expression and potential for helper virus-free packaging. Hum Gene Ther. 1998 Jun 10;9(9):1371-1380. [PubMed abs]
Licensing Status: Available for exclusive or non-exclusive licensing.
Portfolios: Gene Based Therapies
Gene Based Therapies -Therapeutics-Gene Therapy-Vectors-Viral Gene Based Therapies -Therapeutics
For Additional Information Please Contact: David A. Lambertson Ph.D.
NIH Office of Technology Transfer
6011 Executive Blvd, Suite 325
Rockville, MD 20852-3804
Phone: (301)435-4632
Email: lambertsond@mail.nih.gov
Fax: (301) 402-0220
