Alpha-1 antitrypsin deficiency

Brantly Lab


Mark Brantly, MD
Professor of Medicine
Alpha-1 Foundation Research Professor

Personal Statement

I am a senior investigator specializing in rare lung disease translational research with a focus on alpha-1-antitrypsin deficiency, gene therapy, pulmonary fibrosis and alveolar macrophage function.

I have mentored a number of faculty in bench to bedside research at the NIH Clinical Center and the University of Florida. I am a former K24 grantee, a mentor on 2 T32 programs and the UF MD/PhD program. My role on applicant’s grant is to provide molecular biology support, access to lung tissue and guidance on the transition to patient-oriented research.

Lab Faculty and Staff

Professor of Medicine

Mark Brantly

Alpha-1 Foundation Research Professor



Tammy Flagg


Biological Scientist

Alek Aranyos


Research Assistant Professor

Nazli Khodayari


Research Scientist

Jungnam Lee


Graduate Assistant

Naweed Mohammad


Laboratory Manager

Regina Oshins


research administrator

Emily Mak



Daarian Rouhani


Laboratory Technician I

Chancellor McGriff


Laboratory Technician I

William Bowers


REsearch Support


  • Inhibrx 101 LP, ” An Open-Label, Multicenter, Phase 1 Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Ascending Intravenous Doses of Inhibrx rhAAT-Fc (INBRX-101) in Adults with Alpha-1 Antitrypsin Deficiency (AATD),” Brantly (PI) 1/1/2018-12/31/2019
  • Adverum Biotechnologies, Inc., “Phase 1/2 Study of Intravenous or Intrapleural Administration of a Serotype rh.10 Replication Deficient Adeno-associated Virus Gene Transfer Vector Expressing the Human Alpha-1 Antitrypsin cDNA to Individuals with Alpha-1 Antitrypsin Deficiency,”  Brantly (PI) 1/1/2018-12/31/2019
  • Kamada, LTD. “Follow up, surveillance study on patients who were exposed to inhaled AAT during the Kamada-AAT (inhaled)-006 study,” 1/1/2018-12/31/2018
  • Alpha-1 Foundation Research Professorship/Fernandez Liver Research Grant Research Endowment/4PBA Mediated Alpha-1 Antitrypsin Secretion Rescue Brantly (PI) 12/8/1998-
  • Alpha-1 Foundation / HHS-State of Florida/ Talecris Biotherapeutics/ HICL Principal Investigator Alpha-1 Foundation Tissue Bank/ ACT program/National AAT Screening Program/HICL AAT Screening Program, Brantly (PI) 7/1/2000
  • Grifols National AAT Screening Program,  Brantly (PI) 7/1/2000-12/31/18
  • NIH NCRR/Cincinnati Children’s Hospital Medical Center/LAM Foundation, “Quantitative Chest Computed Tomography Unmasking Emphysema Progression in Alpha-1 Antitrypsin Deficiency” Brantly (PI) 11/1/07-6/30/17


  • NIH NCRR/Cincinnati Children’s Hospital Medical Center/LAM Foundation “The Multicenter International Lymphangioleiomyomatosis Efficacy of Sirolimus (MILES),” Brantly (Site PI) 11/01/07- 06/30/2010
  • Kamada, LTD.,”Phase II/III Randomized Double-Blind Comparison of Alpha-1 Proteinase Inhibitor(Kamada-API) with Prolastin,”Brantly (PI) 04/23/07- 01/17/10
  • NIH/NIHLBI, “The Anti-Inflammatory Role of Alpha-1 Antitrypsin in the Lung (K24-HL004456)” Brantly (PI) 3/30/01-07/21/07
  • Florida Department of Health, “Tobacco Smoke-Induced Cell Injury in Lung Compartments,” Brantly (CO-I) 08/01/04-03/31/08
  • Washington University, “Washington University Testing Services Agreement,”Brantly (PI), 02/12/08 -06/30/10

Publication Highlights

  • Flotte, T.R., Trapnell, B.C., Humphries, M., Carey, B., Calcedo, R., Rouhani, F., Campbell-Thompson, M., Yachnis, A.T., Brantly, M.L., Sandhaus, R.A., McElvaney, N.G., et al. 2011. Phase 2 Clinical Trial of a Recombinant Adeno-Associated Viral Vector Expressing alpha(1)-Antitrypsin: Interim Results. Human gene therapy.
  • McCormack, F.X., Inoue, Y., Moss, J., Singer, L.G., Strange, C., Nakata, K., Barker, A.F., Chapman, J.T., Brantly, M.L., Stocks, J.M., et al. 2011. Efficacy and safety of sirolimus in lymphangioleiomyomatosis. The New England journal of medicine 364:1595-1606.
  • Grimstein, C., Choi, Y.K., Wasserfall, C.H., Satoh, M., Atkinson, M.A., Brantly, M.L., Campbell-Thompson, M., and Song, S. 2011. Alpha-1 antitrypsin protein and gene therapies decrease autoimmunity and delay arthritis development in mouse model. Journal of translational medicine 9:21.
  • Stocks, J.M., Brantly, M.L., Wang-Smith, L., Campos, M.A., Chapman, K.R., Kueppers, F., Sandhaus, R.A., Strange, C., and Turino, G. 2010. Pharmacokinetic comparability of Prolastin(R)-C to Prolastin(R) in alpha-antitrypsin deficiency: a randomized study. BMC clinical pharmacology 10:13.
  • Brantly, M.L., Mahr, A.D., Edberg, J.C., Stone, J.H., Hoffman, G.S., St Clair, E.W., Specks, U., Dellaripa, P.F., Seo, P., Spiera, R.F., Rouhani, F.N., et al. 2010. Alpha-antitrypsin deficiency-related alleles Z and S and the risk of Wegener’s granulomatosis. Arthritis and rheumatism 62:3760-3767.
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