I am a pulmonary and critical care clinician-scientist with a long-standing focus on regenerative medicine and stem cell biology. My goal is to advance understanding of and treatment for genetic causes of chronic obstructive pulmonary disease (COPD) and the most common genetic cause of COPD, alpha-1 antitrypsin deficiency (AATD). To accomplish this goal, I have established an integrated clinical and research program here at BU and BMC that includes the following components: 1) The Alpha-1 Center which I direct and co-founded with Dr. Darrell Kotton has become a nationally recognized center of excellence for the care of AATD patients and their families; 2) Patient stem cell repositories: I have overseen the creation of and direct two large stem cell repositories, housed at the CReM. First, we house the world’s largest AATD patient-specific induced pluripotent stem cell (iPSC) repository, comprised of iPSCs and reprogrammable blood samples from over 100 AATD patients linked to phenotypic data including imaging, pulmonary function, and liver biopsy results. Second, in collaboration with the Framingham Heart Study (FHS) and Vasan Ramachandran, the CReM now houses the FHS iPSC Repository that includes iPSCs and reprogrammable blood samples from >6500 highly phenotyped participants in the FHS; 3) Clinical-epidemological AATD Research: Under my direction as site PI, BU is one three sites in the country funded by the Alpha-1 Foundation to recruit 100 AATD subjects to undergo liver biopsy, detailed phenotyping, and 5 years of follow-up to define the prevalence of, risk factors for, and non-invasive biomarkers associated with AATD-associated liver disease; 4) Translational bench research: my lab in the CReM is focused on the application of patient-derived iPSCs to study AATD and COPD.
The 4 core areas of my research are: I) to confirm the clinical significance of the iPSC platform to model in vivo patient biology and demonstrate its potential for testing potential therapeutic agents; II) to better understand the genetic factors and mechanistic drivers that predispose subsets of AATD patients to develop clinical disease; III) to elucidate the mechanistic contribution of putative COPD susceptibility genes to lung disease pathogenesis; and IV) to develop gene or cell-based therapies for AATD.
Research interests include:
-Alpha-1 antitrypsin deficiency
-Pluripotent stem cells
Clinical interests include:
-Alpha-1 antitrypsin deficiency
- Investigator, Framingham Heart Study
- Member, Pulmonary Center, Boston University
- Member, Center for Regenerative Medicine, Boston University
- Member, Evans Center for Interdisciplinary Biomedical Research, Boston University
- Graduate Faculty (Primary Mentor of Grad Students), Boston University Chobanian & Avedisian School of Medicine, Graduate Medical Sciences
- University of Texas Southwestern Medical School, MD
- Williams College, BA
- Published on 9/20/2023
Werder RB, Berthiaume KA, Merritt C, Gallagher M, Villacorta-Martin C, Wang F, Bawa P, Malik V, Lyons SM, Basil MC, Morrisey EE, Kotton DN, Zhou X, Cho MH, Wilson AA. The COPD GWAS gene ADGRG6 instructs function and injury response in human iPSC-derived type II alveolar epithelial cells. Am J Hum Genet. 2023 Oct 05; 110(10):1735-1749. PMID: 37734371.
- Published on 12/6/2022
Kaserman JE, Werder RB, Wang F, Matte T, Higgins MI, Dodge M, Lindstrom-Vautrin J, Bawa P, Hinds A, Bullitt E, Caballero IS, Shi X, Gerszten RE, Brunetti-Pierri N, Liesa M, Villacorta-Martin C, Hollenberg AN, Kotton DN, Wilson AA. Human iPSC-hepatocyte modeling of alpha-1 antitrypsin heterozygosity reveals metabolic dysregulation and cellular heterogeneity. Cell Rep. 2022 Dec 06; 41(10):111775. PMID: 36476855.
- Published on 9/8/2022
Scoon WA, Mancio-Silva L, Suder EL, Villacorta-Martin C, Lindstrom-Vautrin J, Bernbaum JG, Mazur S, Johnson RF, Olejnik J, Flores EY, Mithal A, Wang F, Hume AJ, Kaserman JE, March-Riera S, Wilson AA, Bhatia SN, Mühlberger E, Mostoslavsky G. Ebola virus infection induces a delayed type I IFN response in bystander cells and the shutdown of key liver genes in human iPSC-derived hepatocytes. Stem Cell Reports. 2022 Oct 11; 17(10):2286-2302. PMID: 36084636.
- Published on 7/13/2022
Werder RB, Liu T, Abo KM, Lindstrom-Vautrin J, Villacorta-Martin C, Huang J, Hinds A, Boyer N, Bullitt E, Liesa M, Silverman EK, Kotton DN, Cho MH, Zhou X, Wilson AA. CRISPR interference interrogation of COPD GWAS genes reveals the functional significance of desmoplakin in iPSC-derived alveolar epithelial cells. Sci Adv. 2022 Jul 15; 8(28):eabo6566. PMID: 35857525.
- Published on 6/24/2022
Hume AJ, Heiden B, Olejnik J, Suder EL, Ross S, Scoon WA, Bullitt E, Ericsson M, White MR, Turcinovic J, Thao TTN, Hekman RM, Kaserman JE, Huang J, Alysandratos KD, Toth GE, Jakab F, Kotton DN, Wilson AA, Emili A, Thiel V, Connor JH, Kemenesi G, Cifuentes D, Mühlberger E. Correction: Recombinant Lloviu virus as a tool to study viral replication and host responses. PLoS Pathog. 2022 Jun; 18(6):e1010659. PMID: 35749346.
- Published on 4/15/2022
Werder RB, Huang J, Abo KM, Hix OT, Minakin K, Alysandratos KD, Merritt C, Berthiaume K, Alber AB, Burgess CL, Kotton DN, Wilson AA. Generating 3D Spheres and 2D Air-Liquid Interface Cultures of Human Induced Pluripotent Stem Cell-Derived Type 2 Alveolar Epithelial Cells. J Vis Exp. 2022 Apr 15; (182). PMID: 35499347.
- Published on 3/22/2022
Abo KM, Sainz de Aja J, Lindstrom-Vautrin J, Alysandratos KD, Richards A, Garcia-de-Alba C, Huang J, Hix OT, Werder RB, Bullitt E, Hinds A, Falconer I, Villacorta-Martin C, Jaenisch R, Kim CF, Kotton DN, Wilson AA. Air-liquid interface culture promotes maturation and allows environmental exposure of pluripotent stem cell-derived alveolar epithelium. JCI Insight. 2022 Mar 22; 7(6). PMID: 35315362.
- Published on 2/9/2022
Miorin L, Mire CE, Ranjbar S, Hume AJ, Huang J, Crossland NA, White KM, Laporte M, Kehrer T, Haridas V, Moreno E, Nambu A, Jangra S, Cupic A, Dejosez M, Abo KA, Tseng AE, Werder RB, Rathnasinghe R, Mutetwa T, Ramos I, de Aja JS, de Alba Rivas CG, Schotsaert M, Corley RB, Falvo JV, Fernandez-Sesma A, Kim C, Rossignol JF, Wilson AA, Zwaka T, Kotton DN, Mühlberger E, García-Sastre A, Goldfeld AE. The oral drug nitazoxanide restricts SARS-CoV-2 infection and attenuates disease pathogenesis in Syrian hamsters. bioRxiv. 2022 Feb 09. PMID: 35169796.
- Published on 2/4/2022
Hume AJ, Heiden B, Olejnik J, Suder EL, Ross S, Scoon WA, Bullitt E, Ericsson M, White MR, Turcinovic J, Thao TTN, Hekman RM, Kaserman JE, Huang J, Alysandratos KD, Toth GE, Jakab F, Kotton DN, Wilson AA, Emili A, Thiel V, Connor JH, Kemenesi G, Cifuentes D, Mühlberger E. Recombinant Lloviu virus as a tool to study viral replication and host responses. PLoS Pathog. 2022 02; 18(2):e1010268. PMID: 35120176.
- Published on 1/25/2022
Sauler M, McDonough JE, Adams TS, Kothapalli N, Barnthaler T, Werder RB, Schupp JC, Nouws J, Robertson MJ, Coarfa C, Yang T, Chioccioli M, Omote N, Cosme C, Poli S, Ayaub EA, Chu SG, Jensen KH, Gomez JL, Britto CJ, Raredon MSB, Niklason LE, Wilson AA, Timshel PN, Kaminski N, Rosas IO. Characterization of the COPD alveolar niche using single-cell RNA sequencing. Nat Commun. 2022 01 25; 13(1):494. PMID: 35078977.
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