Engineered nuclease for CCR5 gene editing

Information

  • Research Project
  • 7747074
  • ApplicationId
    7747074
  • Core Project Number
    R41GM085876
  • Full Project Number
    1R41GM085876-01A1
  • Serial Number
    85876
  • FOA Number
    PA-08-051
  • Sub Project Id
  • Project Start Date
    9/1/2009 - 15 years ago
  • Project End Date
    2/28/2011 - 14 years ago
  • Program Officer Name
    PORTNOY, MATTHEW
  • Budget Start Date
    9/1/2009 - 15 years ago
  • Budget End Date
    2/28/2011 - 14 years ago
  • Fiscal Year
    2009
  • Support Year
    1
  • Suffix
    A1
  • Award Notice Date
    8/17/2009 - 15 years ago

Engineered nuclease for CCR5 gene editing

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 funded, 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 applications. 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 pathways, 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 isolation 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. Such 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 HIV 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.

IC Name
NATIONAL INSTITUTE OF GENERAL MEDICAL SCIENCES
  • Activity
    R41
  • Administering IC
    GM
  • Application Type
    1
  • Direct Cost Amount
  • Indirect Cost Amount
  • Total Cost
    101156
  • Sub Project Total Cost
  • ARRA Funded
    False
  • CFDA Code
    859
  • Ed Inst. Type
  • Funding ICs
    NIGMS:101156\
  • Funding Mechanism
    SBIR-STTR
  • Study Section
    ZRG1
  • Study Section Name
    Special Emphasis Panel
  • Organization Name
    PRECISION GENOME ENGINEERING, INC.
  • Organization Department
  • Organization DUNS
    781229211
  • Organization City
    SEATTLE
  • Organization State
    WA
  • Organization Country
    UNITED STATES
  • Organization Zip Code
    981038602
  • Organization District
    UNITED STATES