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Targeted Transposons for Gene Therapy

Award Information

Agency:
Department of Health and Human Services
Branch:
N/A
Award ID:
85608
Program Year/Program:
2007 / STTR
Agency Tracking Number:
EB007451
Solicitation Year:
N/A
Solicitation Topic Code:
N/A
Solicitation Number:
N/A
Small Business Information
DISCOVERY GENOMICS, INC.
614 MC KINLEY PL NE MINNEAPOLIS, MN -
View profile »
Woman-Owned: No
Minority-Owned: No
HUBZone-Owned: No
 
Phase 1
Fiscal Year: 2007
Title: Targeted Transposons for Gene Therapy
Agency: HHS
Contract: 1R41EB007451-01
Award Amount: $174,011.00
 

Abstract:

DESCRIPTION (provided by applicant): Discovery Genomics, Inc. is focused on development of the Sleeping Beauty (SB) transposon system as a non-viral means of integrating new gene sequences in cells and tissues for therapeutic purposes. Here we propose to d evelop a method for improved targeting of the SB system to specific cell types following systemic injection by coupling a targeting tether to the plasmid carrier of the transposon. This approach will allow 'universal' vectors that could carry any gene and be directed to any cell type without modification of the transposase or transposon for each cellular target. This project will dovetail with DGI's ongoing SBIR project to deliver Factor VIII and IX genes in appropriate animal model systems. Thus, this proj ect has high-impact potential because the techniques we will develop can be used for treatment of multiple diseases using a 'universal' non-viral vector system whose properties will be applicable to many therapeutic transgenes. The Specific Aims of the pro ject are to: 1. Construct a Liver- directed Targeting Tether (LTT) vector system to increase uptake of SB transposons into liver cells. This aim will be accomplished by construction of a LTT targeting tether comprised of a LexA DNA-binding domain fused to a ligand specific for hepatocytes and construction of a plasmid, pKLAT2, that contains an SB transposon plus multimeric LexA operator sites to which the targeting tether can bind. 2: Evaluate the efficiency in cultured liver cells (HuH7 and HepG2) of a LTT targeting tether to enhance uptake and transposition of a pKLAT2 transposon vector. HeLa cells will serve as a control for cells that lack appropriate liver-specific receptors. 3: Evaluate the efficiency of a LTT targeting tether to enhance uptake and tra nsposition of a pKLAT2 transposon vector into liver cells in mice.

Principal Investigator:

Perry B. Hackett
6126564485
PERRYH@DISCOVERYGENOMICS.NET

Business Contact:

Rscott Mcivor
perryH@discoverygenomics.net
Small Business Information at Submission:

DISCOVERY GENOMICS, INC.
DISCOVERY GENOMICS, INC. 614 MC KINLEY PL NE MINNEAPOLIS, MN 55413

EIN/Tax ID: 411990513
DUNS: N/A
Number of Employees: N/A
Woman-Owned: No
Minority-Owned: No
HUBZone-Owned: No
Research Institution Information:
UNIVERSITY OF MINNESOTA
UNIVERSITY OF MINNESOTA
100 CHURCH ST SE
MINNEAPOLIS, MN 55455 3000
RI Type: Nonprofit college or university