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Artiodactyl Gene Modification with Tal Effector Nucleases

Award Information

Agency:
Department of Health and Human Services
Branch:
N/A
Award ID:
Program Year/Program:
2012 / SBIR
Agency Tracking Number:
R43OD010564
Solicitation Year:
2012
Solicitation Topic Code:
OD
Solicitation Number:
PA11-096
Small Business Information
RECOMBINETICS, INC.
1246 University Ave. W ST PAUL, MN 55104-
View profile »
Woman-Owned: No
Minority-Owned: No
HUBZone-Owned: No
 
Phase 1
Fiscal Year: 2012
Title: Artiodactyl Gene Modification with Tal Effector Nucleases
Agency: HHS
Contract: 1R43OD010564-01A1
Award Amount: $232,864.00
 

Abstract:

DESCRIPTION (provided by applicant): Large animal models of human disease are indispensible for the development of relevant preclinical therapeutic protocols. Gene inactivation and gene conversion are powerful approaches for creating genocopies of allelesthat cause disease in patients and for the modification of genes for biomedical materials development. A DNA double strand break (DSB) at a desired genome location is a flexible substrate for genome engineering, enabling; mutation of a target sequence, targeted integration of transgenes, or the stimulation of gene conversion by homologous recombination (HR) with a repair template. Consequently, the tools for genome engineering are rapidly converging on the concept of DSB induction using a variety of technologies, including the widely publicized Zinc-Finger Nucleases (ZFN) and Meganuclease (MGN) platforms. While both MGN and ZFN are efficient and reasonably precise, significant limitations in targetable sites, difficulty in developing new enzymes, and a constrained IP landscape limit their widespread commercial application. The recently described TAL effector nucleases (TALENs) are a novel DSB-inducing platform that appears to be easier to deploy and with a greater flexibility in target site selection, since their modular DNA binding motifs enable localization of nuclease activity to a broad range of targets. Recombinetics proposes to investigate enablement of TALENs for gene inactivation and, in combination with RCI technologies to stimulate gene conversion in livestock cells and embryos. The studies proposed here will establish a new paradigm for the genetic modification of artiodactyls (pigs, sheep, and cattle) for use as large animal models of human disease and the production of biomedical materials.PUBLIC HEALTH RELEVANCE: Large animal models of human disease are indispensible for the development of relevant preclinical therapeutic protocols. We propose to investigate enablement of TALENs for gene inactivation in livestock cells and, in combination with RCI technologies to stimulate gene conversion in livestock cells and embryos. The studies proposed here will establish a new paradigm for the genetic modification of artiodactyls (pigs, sheep, and cattle) for use as large animal models of human disease.

Principal Investigator:

Scott C. Fahrenkrug
612-670-2078
scott@recombinetics.com

Business Contact:

Scott C. Fahrenkrug
612-670-2078
scott@recombinetics.com
Small Business Information at Submission:

RECOMBINETICS, INC.
1246 University Ave. W ST PAUL, MN 55104-

EIN/Tax ID: 126312147
DUNS: N/A
Number of Employees: N/A
Woman-Owned: No
Minority-Owned: No
HUBZone-Owned: No