Human Gene Transfer

Overview

“Human Gene Transfer” or “HGT” is used to describe research involving the transfer of recombinant DNA, or DNA or RNA derived from recombinant DNA, into human subjects. It is also sometimes referred to as “Gene Therapy.”  HGT is currently being investigated by researchers at Ohio State in a variety of human diseases to see whether it can treat certain health problems by supplying a missing protein, compensating for a defective gene, providing a therapeutic substance, or activating the subject’s own immune response.

HGT touches on a number of scientific, medical, ethical, and safety issues.  The National Institutes of Health (NIH) Office of Science Policy (OSP), the office charged with oversight of recombinant DNA research, is responsible for publishing and updating the NIH Guidelines for Research Involving Recombinant or Synthetic Nucleic Acid Molecules (NIH Guidelines).  The NIH Guidelines set forth the guiding principles regarding oversight and provide specific guidance on the conduct of human gene transfer to ensure the safe handling and containment of recombinant DNA and define human gene transfer as the deliberate transfer into human research participants of either:

  1. Recombinant nucleic acid molecules, or DNA or RNA derived from recombinant nucleic acid molecules, or
  2. Synthetic nucleic acid molecules, or DNA or RNA derived from synthetic nucleic acid molecules, that meet any one of the following criteria:
    1. Contain more than 100 nucleotides; or
    2. Possess biological properties that enable integration into the genome (e.g., cis elements involved in integration); or
    3. Have the potential to replicate in a cell; or
    4. Can be translated or transcribed.

The NIH Guidelines were recently updated and streamlined to eliminate the Recombinant DNA Advisory Committee (RAC) and remove the requirement to register and report human gene therapy protocols to the NIH OSP, thereby granting full authority to local oversight bodies (e.g. Institutional Biosafety Committee [IBC] and Institutional Review Board [IRB]) to determine whether the research presents significant biosafety, scientific, societal, or ethical concerns.

At OSU, HGT research must be reviewed and approved locally by the IBC and the IRB. Lastly, all HGT research at Ohio State must undergo a formal institutional review and approval process, which includes coordination of special contract provisions, risk assessments, and College and University administration approvals. For additional information on conducting HGTs at the university, see the institutional policy on Human Gene Transfer along with the Human Gene Transfer Flowchart for an overview of the HGT Institutional Review Process.

The IBC and Office of Research Compliance coordinate the HGT review process at the university, which is briefly described above. The first step is that investigators should register their HGT research with the IBC.  For more information, see the Investigator Guidance. For general information on HGT clinical trial research, contact Courtney Mankowski, Research Integrity and Compliance Manager at mankowski.4@osu.edu or (614)292-5714.

Regulations

Policies & Procedures