Safety & Security

Human Gene Transfer Protocols

"Human Gene Transfer" or "HGT" is used to describe research involving the transfer of recombinant or synthetic nucleic acid molecules, or DNA or RNA derived from recombinant or synthetic nucleic acid molecules into human subjects. It is also sometimes referred to as "Gene Therapy." Human gene transfer is the process of transferring genetic material (DNA or RNA) into a person. Nucleic acids (DNA or RNA) may be transferred as "naked" nucleic acid, encapsulated nucleic acid, or nucleic acid within another organism, such as a virus.

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.

Section III-C-1 - (Experiments Involving the Deliberate Transfer of Recombinant or Synthetic Nucleic Acid Molecules, or DNA or RNA Derived from Recombinant or Synthetic Nucleic Acid Molecules, into One or More Human Research Participants) of NIH guidelines defines-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:
    • Contain more than 100 nucleotides; or
    • Possess biological properties that enable integration into the genome (e.g. cis elements involved in integration); or
    • Have the potential to replicate in a cell; or
    • Can be translated or transcribed.

Research cannot be initiated until Institutional Biosafety Committee and all other applicable institutional and regulatory authorization(s) and approvals have been obtained.

The deliberate transfer of recombinant or synthetic nucleic acids into one human research participant, conducted under a Food and Drug Administration (FDA) regulated individual patient expanded access Investigational New Drug (IND) or protocol, including for emergency use, is not research subject to the NIH Guidelines and thus does not need to be submitted to an IBC for review and approval.

Section I-A-1-a - For experiments involving the deliberate transfer of recombinant or synthetic nucleic acid molecules, or DNA or RNA derived from recombinant or synthetic nucleic acid molecules, into human research participants (human gene transfer), no human gene transfer experiment shall be initiated (see definition of initiation in Section I-E-4) until Institutional Biosafety Committee (IBC) approval (from the clinical trial site) has been obtained and all applicable regulatory authorization(s) and approvals have been obtained.

The use of rDNA in human research subjects at the local level requires IBC review and approval in addition to IRB approval prior to initiation of the study. IBC policy Sections 8.3 and 8.4 (PDF) provide information regarding HGT here at USF.

REQUIREMENTS FOR IBC REVIEW OF THE PROTOCOL

An IBC application for the use of rDNA must be submitted for review and approval. All new IBC applications are now submitted through our electronic format BiosafetyNet. 

  1. Clinical Protocol
  2. Investigational Brochure
  3. Based on case by case reviews the IBC may require other communication letters from other agencies such as the FDA with modifications of the protocol based on FDA requirements.

REPORTING REQUIREMENTS

All amendments and continuing reviews that are submitted to the IRB related to the gene transfer study or in support of gene transfer protocol must be submitted to the IBC for review. This includes:

  1. All Continuing Review Reports to the IRB.
  2. All Change in Procedures/Investigator’s brochure reported to the IRB.
  3. Report(s) of violations of the NIH Guidelines, or any significant research-related accidents and illnesses.