Articles

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Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression (with Jeffrey D. Chulay, Bruce C. Trapnell, Margaret Humphries, Brenna Carey, Robert A. Sandhaus, Noel G. McElvaney, Louis M. Messina, Qiushi Tang, Farshid N. Rouhani, Martha Campbell-Thompson, Ann Dongtao Fu, Anthony Yachnis, David R. Knop, Guo-Jie Ye, Mark Brantly, Roberto Calcedo, Suryanarayan Somanathan, Lee P. Richman, Robert H. Vonderheide, Maigan A. Hulme, Todd M. Brusko, James M. Wilson, and Terence R. Flotte), The Journal of clinical investigation (2013)

Recombinant adeno-associated virus (rAAV) vectors have shown promise for the treatment of several diseases; however,...

 

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Gene-based therapy for alpha-1 antitrypsin deficiency (with Terence R. Flotte), COPD (2013)

Alpha-1 antitrypsin Deficiency (AATD) has been an attractive target for the development of gene therapy...

 

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Gene transfer in skeletal and cardiac muscle using recombinant adeno-associated virus (with Alisha M. Gruntman, Lawrence T. Bish, H. Lee Sweeney, Terence R. Flotte, and Guangping Gao), Current protocols in microbiology (2013)

Adeno-associated virus (AAV) is a DNA virus with a small ( approximately 4.7 kb) single-stranded...

 

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Immune responses in cystic fibrosis: are they intrinsically defective? (with Dmitry Ratner), American journal of respiratory cell and molecular biology (2012)

Cystic fibrosis (CF), the most common lethal single-gene disorder affecting Northern Europeans and North Americans,...

 

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Long-term correction of very long-chain acyl-coA dehydrogenase deficiency in mice using AAV9 gene therapy (with Allison M. Keeler, Thomas J. Conlon, Glenn Walter, Huadong Zeng, Scott A. Shaffer, Fu Dungtao, Kirsten E. Erger, Travis L. Cossette, Qiushi Tang, and Terence R. Flotte), Molecular therapy : the journal of the American Society of Gene Therapy (2012)

Very long-chain acyl-coA dehydrogenase (VLCAD) is the rate-limiting step in mitochondrial fatty acid oxidation. VLCAD-deficient...

 

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Sustained miRNA-mediated Knockdown of Mutant AAT With Simultaneous Augmentation of Wild-type AAT Has Minimal Effect on Global Liver miRNA Profiles (with Qiushi Tang, Alisha Gruntman, Keith S. Blomenkamp, Jeffrey H. Teckman, Lina Song, Phillip D. Zamore, and Terence R. Flotte), Molecular Therapy (2012)

Alpha-1 antitrypsin (AAT) deficiency can exhibit two pathologic states: a lung disease that is primarily...

 

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Induction of Group IVC Phospholipase A2 in Allergic Asthma: Transcriptional Regulation by TNF-α in Bronchoepithelial Cells (with Justin S. Bickford, Kimberly J. Newsom, John-David Herlihy, Benjamin Keeler, Xiaolei Qiu, Jewell N. Walters, Nan Su, Shannon M. Wallet, Terence R. Flotte, and Harry S. Nick), The Biochemical journal (2011)

Airway inflammation in allergen-induced asthma is associated with eicosanoid release. These bioactive lipids exhibit anti-...

 

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Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing α1-antitrypsin: interim results (with Terence R. Flotte, Bruce C. Trapnell, Margaret Humphries, Brenna Carey, Roberto Calcedo, Farshid Rouhani, Martha Campbell-Thompson, Anthony T. Yachnis, Robert A. Sandhaus, Noel G. McElvaney, Louis M. Messina, James M. Wilson, Mark L. Brantly, David R. Knop, Guo-jie Ye, and Jeffrey D. Chulay), Human gene therapy (2011)

Recombinant adeno-associated virus (rAAV) vectors offer promise for the gene therapy of α(1)-antitrypsin (AAT) deficiency....

 

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N-glycosylation augmentation of the cystic fibrosis epithelium improves Pseudomonas aeruginosa clearance (with Ashley T. Martino, Sofia Braag, Pedro E. Cruz, Martha Campbell-Thompson, Shouguang Jin, and Terence R. Flotte), American journal of respiratory cell and molecular biology (2011)

Chronic lung colonization with Pseudomonas aeruginosa is anticipated in cystic fibrosis (CF). Abnormal terminal glycosylation...

 

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Lack of cystic fibrosis transmembrane conductance regulator in CD3+ lymphocytes leads to aberrant cytokine secretion and hyperinflammatory adaptive immune responses (with Sofia A. Braag, Allison M. Keeler, Craig Hodges, Mitchell Drumm, and Terence R. Flotte), American journal of respiratory cell and molecular biology (2011)

Cystic fibrosis (CF), the most common fatal monogenic disease in the United States, results from...

 

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Hepatocyte-specific hypoxia-inducible factor-1alpha is a determinant of lipid accumulation and liver injury in alcohol-induced steatosis in mice (with Bharath D. Nath, Ivan Levin, Timea Csak, Jan Petrasek, Karen Kodys, Donna Catalano, Pranoti Mandrekar, and Gyongyi Szabo), Hepatology (Baltimore, Md.) (2011)

Chronic alcohol causes hepatic steatosis and liver hypoxia. Hypoxia-regulated hypoxia-inducible factor 1-alpha, (HIF-1alpha) may regulate...

 

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MicroRNA-regulated, systemically delivered rAAV9: a step closer to CNS-restricted transgene expression (with Jun Xie, Qing Xie, Hongwei Zhang, Stefan L. Ameres, Jui-Hung Hung, Qin Su, Ran He, Xin Mu, Seemin Seher Ahmed, Soyeon Park, Hiroki Kato, Chengjian Li, Craig C. Mello, Zhiping Weng, Terence R. Flotte, Phillip D. Zamore, and Guangping Gao), Molecular Therapy (2011)

Recombinant adeno-associated viruses (rAAVs) that can cross the blood-brain-barrier and achieve efficient and stable transvascular...

 

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Modulation of Exaggerated-IgE Allergic Responses by Gene Transfer-mediated Antagonism of IL-13 and IL-17e. (with Allison M. Keeler, Sofia Braag, Timothy Menz, Qiushi Tang, and Terence R. Flotte), Molecular therapy : the journal of the American Society of Gene Therapy (2010)

Asthma and allergic rhinitis are almost invariable accompanied by elevated levels of immunoglobin E (IgE),...

 

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Dual reporter comparative indexing of rAAV pseudotyped vectors in chimpanzee airway (with Terence R. Flotte, Anne C. Fischer, Jason Goetzmann, Liudmila Cebotaru, Ziying Yan, Lilli Wang, James M. Wilson, William B. Guggino, and John F. Engelhardt), Molecular therapy : the journal of the American Society of Gene Therapy (2010)

Selecting the most efficient recombinant adeno-associated virus (rAAV) serotype for airway gene therapy has been...

 

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In vitro and in vivo functional characterization of gutless recombinant SV40-derived CFTR vectors (with M. S. Strayer, Jeffrey Sirninger, Sofia A. Braag, Francisco Branco, Jean-Pierre Louboutin, Terence R. Flotte, and David S. Strayer), Gene therapy (2010)

In cystic fibrosis (CF), respiratory failure caused by progressive airway obstruction and tissue damage is...

 

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Cystic Fibrosis (with Michael S. Stalvey and Terence R. Flotte), Endocrinology/Diabetes (2010)

Citation: Stalvey, M., Mueller, C., and Flotte, T. “Cystic Fibrosis”, in Domino FJ, ed., The...

 

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Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy (with Mark L. Brantly, Jeffrey D. Chulay, Lili Wang, Margaret Humphries, L. Terry Spencer, Farshid Rouhani, Thomas J. Conlon, Roberto Calcedo, Michael R. Betts, Carolyn Spencer, Barry J. Bryne, James M. Wilson, and Terence R. Flotte), Proceedings of the National Academy of Sciences of the United States of America (2009)

Alpha-1 antitrypsin (AAT) deficiency is well-suited as a target for human gene transfer. We performed...

 

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The pros and cons of immunomodulatory IL-10 gene therapy with recombinant AAV in a Cftr-/- -dependent allergy mouse model (with Sofia A. Braag, A. T. Martino, Qiushi Tang, M. Campbell-Thompson, and Terence R. Flotte), Gene therapy (2009)

Cystic fibrosis (CF) patients have decreased levels of lung epithelial interleukin (IL)-10 and increased levels...

 

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Recombinant AAV serotype and capsid mutant comparison for pulmonary gene transfer of alpha-1-antitrypsin using invasive and noninvasive delivery (with Rejean Liqun Wang, Thomas J. McLaughlin, Travis Cossette, Qiushi Tang, Kevin Foust, Martha Campbell-Thompson, Ashley Martino, Pedro Cruz, Scott A. Loiler, and Terence R. Flotte), Molecular therapy : the journal of the American Society of Gene Therapy (2009)

Recombinant adeno-associated viral (rAAV) vectors have been widely used in pulmonary gene therapy research. In...

 

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Gene therapy for cystic fibrosis (with Terence R. Flotte), Clinical reviews in allergy and immunology (2008)

Cystic Fibrosis (CF) is an autosomal recessive disorder due to mutations in the CF transmembrane...

 

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CFTR mutations impart elevated immune reactivity in a murine model of cystic fibrosis related diabetes (with Michael S. Stalvey, Todd M. Brusko, Clive H. Wasserfall, Desmond A. Schatz, Mark A. Atkinson, and Terence R. Flotte), Cytokine (2008)

Increased life expectancy in cystic fibrosis (CF) is accompanied by an increasing incidence of CF...

 

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Apparently nonspecific enzyme elevations after portal vein delivery of recombinant adeno-associated virus serotype 2 vector in hepatitis C virus-infected chimpanzees (with Terence R. Flotte, Jason Goetzmann, James Caridi, Joseph Paolillo, Thomas J. Conlon, Mark Potter, and Barry J. Bryne), Human gene therapy (2008)

Hepatic gene transfer is envisioned as a substitute for protein replacement therapies, many of which...

 

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Clinical gene therapy using recombinant adeno-associated virus vectors (with Terence R. Flotte), Gene therapy (2008)

Recombinant adeno-associated virus (rAAV) vectors possess a number of properties that may make them suitable...

 

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Partial correction of the CFTR-dependent ABPA mouse model with recombinant adeno-associated virus gene transfer of truncated CFTR gene (with Daniel M. Torrez, Sofia Braag, Ashley Martino, Tracy Clarke, Martha Campbell-Thompson, and Terence R. Flotte), The journal of gene medicine (2008)

Recently, we have developed a model of airway inflammation in a CFTR knockout mouse utilizing...

 

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In vivo post-transcriptional gene silencing of alpha-1 antitrypsin by adeno-associated virus vectors expressing siRNA (with Pedro E. Cruz, Travis L. Cossette, Alexandra Golant, Qiushi Tang, Stuart G. Beattie, Mark Brantly, Martha Campbell-Thompson, Keith S. Blomenkamp, Jeffrey H. Teckman, and Terence R. Flotte), Laboratory investigation; a journal of technical methods and pathology (2007)

alpha-1 Antitrypsin (AAT) deficiency is one of the most common genetic diseases in North America,...

 

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The promise of gene therapy for the treatment of alpha-1 antitrypsin deficiency (with Pedro E. Cruz and Terence R. Flotte), Pharmacogenomics (2007)

In the last 13 years, three gene therapy trials for the treatment of alpha-1 antitrypsin...

 

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Cystic fibrosis transmembrane conductance regulator deficiency exacerbates islet cell dysfunction after beta-cell injury (with Michael S. Stalvey, Desmond A. Schatz, Clive H. Wasserfall, Martha L. Campbell-Thompson, Douglas W. Theriaque, Terence R. Flotte, and Mark A. Atkinson), Diabetes (2006)

The cause of cystic fibrosis-related diabetes (CFRD) remains unknown, but cystic fibrosis transmembrane conductance regulator...

 

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Enhanced IgE allergic response to Aspergillus fumigatus in CFTR-/- mice (with Sofia A. Braag, John-David Herlihy, Clive H. Wasserfall, Sarah E. Chesrown, Harry S. Nick, Mark A. Atkinson, and Terence R. Flotte), Laboratory investigation; a journal of technical methods and pathology (2006)

To gain insight into aberrant cytokine regulation in cystic fibrosis (CF), we compared the phenotypic...

 

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Functional characterization of a recombinant adeno-associated virus 5-pseudotyped cystic fibrosis transmembrane conductance regulator vector (with Jeffrey Sirninger, Sofia A. Braag, Qiushi Tang, Hungwen Yue, Carol Detrisac, Thomas Ferkol, William B. Guggino, and Terence R. Flotte), Human gene therapy (2004)

Despite extensive experience with recombinant adeno-associated virus (rAAV) 2 vectors in the lung, gene expression...

 

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Effects of CFTR, interleukin-10, and Pseudomonas aeruginosa on gene expression profiles in a CF bronchial epithelial cell Line (with Isabel Virella-Lowell, John-David Herlihy, Barry Liu, Cecilia Lopez, Pedro Cruz, Henry V. Baker, and Terence R. Flotte), Molecular therapy : the journal of the American Society of Gene Therapy (2004)

Mutations in CFTR lead to a complex phenotype that includes increased susceptibility to Pseudomonas infections,...

 

Contributions to Books

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Gene transfer in the lung using recombinant adeno-associated virus (with Alisha Gruntman, Terence R. Flotte, and Guangping Gao), Current Protocols in Microbiology (2012)

Adeno-associated virus (AAV) is a small replication-deficient DNA virus belonging to the Parvovirinae family. It...

 

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Production and discovery of novel recombinant adeno-associated viral vectors (with Li Zhong, Dmitry Ratner, Miguel Sena-Esteves, and Guangping Gao), Current Protocols in Microbiology (2012)

In this unit, we describe the detailed procedure for a three-plasmid transfection method for rAAV...

 

Posters and Presentations

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rAAV9 airway delivery results in effective knockdown of mutant alpha 1-antitrypsin in the liver while upregulating wildtype alpha 1-antitrypsin in the lung (with Alisha Gruntman, Qiushi Tang, Lina Song, James M. Wilson, Terence R. Flotte, and Maria P. Limberis), Clinical and Translational Science Research Retreat (2012)

Alpha 1-Antitrypsin (AAT) deficiency is a human genetic disease resulting in the production of mutant...

 

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Codon Optimization for Alpha 1-Antitrypsin Disease (with Timothy Menz, Qiushi Tang, Lina Song, and Terence R. Flotte), Senior Scholars Program (2012)

Alpha 1-antitrypsin deficiency is a genetic disorder caused by defective production of alpha 1-antitrypsin (AAT)....

 

Other

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Lack of CFTR in CD3+ Lymphocytes Leads to Aberrant Cytokine Secretion and Hyper-Inflammatory Adaptive Immune Responses: A Master's Thesis, GSBS Dissertations and Theses (2012)

Background: Cystic fibrosis (CF) remains the most common fatal monogenic disease in the US,...