IN VIVO SKIN TARGETED GENE THERAPY BY ELECTROPORATION

Information

  • Research Project
  • 2794711
  • ApplicationId
    2794711
  • Core Project Number
    R43AR044453
  • Full Project Number
    1R43AR044453-01A1
  • Serial Number
    44453
  • FOA Number
  • Sub Project Id
  • Project Start Date
    3/1/1999 - 25 years ago
  • Project End Date
    8/31/2000 - 23 years ago
  • Program Officer Name
    MOSHELL, ALAN N.
  • Budget Start Date
    3/1/1999 - 25 years ago
  • Budget End Date
    8/31/2000 - 23 years ago
  • Fiscal Year
    1999
  • Support Year
    1
  • Suffix
    A1
  • Award Notice Date
    2/26/1999 - 25 years ago
Organizations

IN VIVO SKIN TARGETED GENE THERAPY BY ELECTROPORATION

In vivo transfer of genetic information to cells of the skin may offer a useful therapy for skin-specific indications as well as systemic diseases such as hemophilia. The skin presents a large, easily accessible target that contains cells such as keratinocytes that can provide secretory capability to the circulatory system. Currently, the greatest obstacle to skin-targeted gene therapy is the lack of a simple and effective delivery system. The transient nature of transgene expression in the epidermis demands that the delivery method be readily repeatable, and thus preferably based on the use of naked DNA. We present here a novel method, based on the electroporation of naked DNA, for topical delivery of DNA to human skin. The purpose of the Phase I study plan is to advance our preliminary data on gene transfer and develop an optimized, effective protocol for targeting keratinocytes in human skin xenografts. In Phase II and beyond, our goal is the development of a therapeutic method and device for the treatment of hemophilia A and B. PROPOSED COMMERCIAL APPLICATION: The goal of this research is the development of a novel gene delivery method and device that will enable us to enter clinical trials for gene therapy. Ultimately we envision the creation of a small, hand-held, battery-operated applicator. The device would enable the user to safely self-administer a pharmaceutically prepared packet of therapeutic DNA directly to the skin through the use of disposable electrodes.

IC Name
NATIONAL INSTITUTE OF ARTHRITIS AND MUSCULOSKELETAL AND SKIN DISEASES
  • Activity
    R43
  • Administering IC
    AR
  • Application Type
    1
  • Direct Cost Amount
  • Indirect Cost Amount
  • Total Cost
  • Sub Project Total Cost
  • ARRA Funded
  • CFDA Code
    846
  • Ed Inst. Type
  • Funding ICs
  • Funding Mechanism
  • Study Section
    ZMH1
  • Study Section Name
  • Organization Name
    GENETRONICS, INC.
  • Organization Department
  • Organization DUNS
    122735749
  • Organization City
    SAN DIEGO
  • Organization State
    CA
  • Organization Country
    UNITED STATES
  • Organization Zip Code
    921211318
  • Organization District
    UNITED STATES