Engineered nuclease for CCR5 gene editing

Award Information
Agency:
Department of Health and Human Services
Branch
n/a
Amount:
$101,156.00
Award Year:
2009
Program:
STTR
Phase:
Phase I
Contract:
1R41GM085876-01A1
Award Id:
93801
Agency Tracking Number:
GM085876
Solicitation Year:
n/a
Solicitation Topic Code:
n/a
Solicitation Number:
n/a
Small Business Information
PRECISIONGENOME ENGINEERING, 454 N 34TH ST, SEATTLE, WA, 98103
Hubzone Owned:
N
Minority Owned:
N
Woman Owned:
N
Duns:
781229211
Principal Investigator:
ANDREWSCHARENBERG
(206) 987-7314
ANDREWMS@U.WASHINGTON.EDU
Business Contact:
LAURIEBEITX
() -
Research Institute:
SEATTLE CHILDREN'S HOSPITAL

Office of Sponsored Research
4800 SAND POINT WAY NE
SEATTLE, WA, 98105 1274

Domestic nonprofit research organization
Abstract
DESCRIPTION (provided by applicant): State the application's broad, long-term objectives and specific aims, making reference to the health relatedness of the project (i.e., relevance to the mission of the agency). Describe concisely the research design and methods for achieving these goals. Describe the rationale and techniques you will use to pursue these goals. In addition, in two or three sentences, describe in plain, lay language the relevance of this research to public health. If the application is fun ded, this description, as is, will become public information. Therefore, do not include proprietary/confidential information. Genome engineering is an emerging field in which targeted genome modifications are made for biotechnological and therapeutic appli cations. Site specific rare cutting endonucleases are a crucial tool for genome engineering, as they are required to create DNA double strand breaks at desired genomic sites. Endonuclease-induced double strand breaks are resolved by endogenous DNA repair p athways, resulting in high efficiency gene editing if resolved via non-homologous end joining, or targeted gene modification if resolved via homologous recombination. Precision Genome Engineering has developed proprietary methods for generation and isolati on of rare cutting endonucleases based on the use of the I-AniI LAGLIDADG homing endonuclease as a scaffold. This phase I STTR will support the application of this approach to generate a novel LAGLIDADG nuclease capable of cleaving the human CCR5 gene. Suc h a nuclease can be applied for generation of CCR5- deficient T-cells. CCR5-deficient T-cells are resistant to infection by CCR5-tropic strains of HIV, the most common form of HIV in the United States. Generation of such T-cells and re-engraftment in an HI V infected patient represents a new approach to treatment of established HIV infections with significant promise. PUBLIC HEALTH RELEVANCE: This project will support development of an engineered site specific nuclease capable of cleaving the human CCR5 gene . This protein or refined derivatives will be applicable to generation of CCR5-deficient T-cells, a novel approach to therapy of HIV infections.

* information listed above is at the time of submission.

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