Human Gene Transfer Studies

Definitions

Human gene transfer is the process of transferring genetic material (DNA or RNA) into a person. At present, human gene transfer is experimental and is being studied to see whether it could treat certain health problems by compensating for defective genes, producing a potentially therapeutic substance, or triggering the immune system to fight disease.

Human gene transfer may help improve genetic disorders, particularly those conditions that result from inborn errors in a single gene (i.e., sickle cell anemia, hemophilia, and cystic fibrosis). It may also hold promise for diseases with more complex origins, like cancer and heart disease. Gene transfer is also being studied as a possible treatment for certain infectious diseases, such as AIDS. This type of experimentation is sometimes called 'gene therapy' research. 

Human Gene Transfer Research is defined by federal regulations as, "Any deliberate transfer of recombinant DNA, or DNA or RNA derived from recombinant DNA (technology), into human research participants."

Required Reviews

The National Institutes of Health Recombinant DNA Guidelines require review (or exemption from review) of human gene transfer studies by:

  • The NIH’s Office of Biotechnology Activities (OBA) Recombinant DNA Advisory Committee (RAC)
  • The Institutional Biosafety Committee (at UT Southwestern this is called the Biological and Chemical Safety Advisory Committee, a.k.a. the BCSAC) according to the NIH Guidelines For Research Involving Recombinant DNA Molecules, 2002 Section IV-B-2
  • The Institutional Review Board (IRB). Please note, Institutional Biosafety Committee approval or exemption is required before submission to the IRB for human subjects protection review and approval

IRB Review and Guidance

UT Southwestern IRB requires submission of a protocol that involves human gene transfer using eIRB application. Please see study submission instructions on Electronic Application (eIRB) web page. 

It is imperative that appropriate selection is made in eIRB application in section 25.0 (Drugs, Devices , Biologics) as "Recombinant DNA/ Human Gene Transfer (Any deliberate transfer of recombinant DNA, or DNA or RNA derived from recombinant DNA (technology), into human research participants)" in order for study to be routed to IBC for review and approval. Note: IBC is a "blocking review," which means that the study is first reviewed by IBC and only upon receipt of approval is routed to IRB for review.

For specific questions about IBC review and/ or adverse event reporting  requirements, please contact Biological Safety Compliance Officer, Patrick Conley at 214-648-2250 or at patrick.conley@utsouthwestern.edu

References and Additional Guidance