UI Healthcare LogoInternal Medicine Home Pagespacer imageEducation InformationPatient Care InformationResearch Informationspacer imageDivisions of the DepartmentFaculty
Diversity and Culture ConferencesNews and EventsGiving Opportunitiesspacer imageServicesIntranet (login required)Useful LinksContact Usspacer imageThe University of Iowa

 

UI Healthcare Navigation Bar UI Healthcare Home Page About Us Appointments Contact UI Healthcare Staff Departments Health Topics Search

Department of Internal Medicine

Infectious Diseases Faculty


Anton P. McCaffrey, Ph.D.
Assistant Professor

Laboratory interests

RNA interference therapeutics targeting hepatitis B virus

Our previous studies have shown that hepatitis B virus (HBV) RNA interference (RNAi) can be effective at inhibiting HBV replication in mouse models. However, expression of too much RNAi can be toxic because it competes for the endogenous miRNA machinery. We use rational approaches to design more potent RNAi that functions at much lower doses. We are currently expressing this HBV RNAi from self-complementary adeno-associated virus serotype 8 (AAV8) vectors for testing in HBV transgenic mice.

Designing and constructing zinc-finger nucleases that recognize and cleave the genome of HBV

Current treatments for HBV are effective in only ~50 % of patients. This is in part because of the persistence of the HBV DNA genome. We have set out to design the first class of drugs that directly target the genome of a DNA virus for destruction. We build designer restriction enzymes, called zinc-finger nucleases (ZFNs), that specifically bind to HBV sequences and catalyze their cleavage in cells. We are currently testing this new type of therapeutic in mice.

The role of microRNAs in normal and pathogenic liver function

MiRNAs (miRNAs) are small regulatory RNAs that modulate gene expression by binding to the 3’ untranslated regions of mRNAs and causing translational inhibition or mRNA degradation. The McCaffrey lab studies miRNAs involved in normal liver functions as well as miRNAs involved in pathological processes. We utilize mouse models and also obtain human clinical samples through collaborations with hepatologists and transplant surgeons here at the University of Iowa.

Anton McCaffrey photo

Education:
B.A., University of California,
San Diego
Ph.D., University of Colorado, Boulder

Fellowship:
Stanford University

Recent Publications

  1. McCaffrey, A. P., Fawcett, P., Nakai, H., Ehrhardt, A., Pham, T.T., Pandey, K., Xu, H., Feuss, S., Storm, T., and Kay, M.A., Host Response to Helper Dependent Adenovirus and Adeno-associated Virus in Mouse Liver. Molecular Therapy (in press). 2008.
  2. Marquez RT, McCaffrey AP., Advances in microRNAs: implications for gene therapists. Human Gene Therapy, 19: 27-38, 2008.
  3. McCaffrey, A.P., Nakai, H., Pandey, K., Huang, H., Xu, H., Salazar, F.H., Marion, P.L. and Kay, M.A. Inhibition of Hepatitis B Virus in Mice by RNA Interference. Nat Biotechnol 21:639-644, 2003.
  4. McCaffrey, A.P., Meuse, L., Karimi, M. and Kay, M.A. Potent Morpholino Antisense Inhibitors of Hepatitis C Internal Ribosome Entry Site Translation in Mice. Hepatology 38:503-508, 2003.
  5. McCaffrey, A.P., Meuse, L., Pham, T.T. and Kay, M.A. RNA interference in adult mice.  Nature 418:38-39, 2002.

Links of Interest

Back to Infectious Diseases Faculty List


UI Healthcare Home | About Us | Appointments | Contact UI Healthcare | Departments | Health Library | Search

Home | Education | Patient Care | Research
Divisions | Faculty | Conferences | News & Events | Giving Opportunities
Services | Intranet | Useful Links | Contact Us