Terence R Flotte

Terence R Flotte

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Terence R Flotte

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Women's Representation Among Members and Leaders of National Medical Specialty Societies.

Acad Med 2019 Oct 15. Epub 2019 Oct 15.

R. Jagsi is professor, Department of Radiation Oncology, University of Michigan, Ann Arbor, Michigan; ORCID: https://orcid.org/0000-0001-6562-1228. O. Means is a resident in plastic surgery, Spectrum Health, Grand Rapids, Michigan. D. Lautenberger is director, Women in Medicine and Science, Association of American Medical Colleges, Washington, DC. R.D. Jones is research area specialist intermediate, Center for Bioethics and Social Science in Medicine, University of Michigan, Ann Arbor, Michigan. K.A. Griffith is statistician expert, Center for Cancer Biostatistics, University of Michigan, Ann Arbor, Michigan. T.R. Flotte is the Celia and Isaac Haidak Professor, and dean, School of Medicine, University of Massachusetts Medical School, Worcester, Massachusetts. L.K. Gordon is professor of ophthalmology and senior associate dean, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, California; ORCID: https://orcid.org/0000-0002-4495-8830. K.M. Rexrode is chief, Division of Women's Health, Department of Medicine, associate professor of medicine, Harvard Medical School, Brigham and Women's Hospital, Boston, Massachusetts; ORCID: https://orcid.org/0000-0003-3387-8429. L.W. Wagner is professor of medicine, Department of Medicine, University of Louisville School of Medicine, Louisville, Kentucky; ORCID: https://orcid.org/0000-0002-6958-9103. A. Chatterjee is professor and chair, Department of Pediatrics, University of South Dakota Sanford School of Medicine/Sanford Children's Specialty Clinic, Sioux Falls, South Dakota.

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http://dx.doi.org/10.1097/ACM.0000000000003038DOI Listing
October 2019

AAV9 gene replacement therapy for respiratory insufficiency in very-long chain acyl-CoA dehydrogenase deficiency.

J Inherit Metab Dis 2019 Sep 3;42(5):870-877. Epub 2019 May 3.

Department of Pediatrics, Duke University, Durham, North Carolina.

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http://dx.doi.org/10.1002/jimd.12101DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6739149PMC
September 2019

Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here?

Annu Rev Virol 2019 Sep 5;6(1):601-621. Epub 2019 Jul 5.

Horae Gene Therapy Center and Department of Pediatrics, University of Massachusetts Medical School, Worcester, Massachusetts 01655, USA; email:

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http://dx.doi.org/10.1146/annurev-virology-092818-015530DOI Listing
September 2019

Epigenome Editing Strategies for Low Back Pain.

Authors:
Terence R Flotte

Hum Gene Ther 2019 Sep;30(9):1037-1038

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http://dx.doi.org/10.1089/hum.2019.29094.trfDOI Listing
September 2019

Senior Gene Therapy Scientists Take a Stand Against Human Embryo Gene Editing.

Authors:
Terence R Flotte

Hum Gene Ther 2019 Jul;30(7):793

Editor-In-Chief.

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http://dx.doi.org/10.1089/hum.2019.29088.trfDOI Listing
July 2019

New Horizons for Immune Gene Therapy.

Hum Gene Ther 2019 Apr;30(4):379-380

2 Editor-in-Chief.

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http://dx.doi.org/10.1089/hum.2019.29085.abaDOI Listing
April 2019

Assuring Integrity in the Residency Match Process.

Acad Med 2019 Mar;94(3):321-323

S.N. Chimienti is associate professor of medicine, vice provost for student life and enrollment, and associate dean for student affairs, School of Medicine, University of Massachusetts Medical School, Worcester, Massachusetts. D.M. DeMarco is professor of medicine, senior associate dean for clinical affairs, and associate dean for graduate medical education, School of Medicine, University of Massachusetts Medical School, Worcester, Massachusetts. T.R. Flotte is professor of pediatrics, Celia and Isaac Haidak Professor of Medical Education, executive deputy chancellor, provost, and dean, School of Medicine, University of Massachusetts Medical School, Worcester, Massachusetts. M.F. Collins is professor of quantitative health sciences, professor of medicine, and chancellor, University of Massachusetts Medical School, and senior vice president for the health sciences, University of Massachusetts, Worcester, Massachusetts.

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http://dx.doi.org/10.1097/ACM.0000000000002531DOI Listing
March 2019

What the Gene Therapy Community Should Do About Sexual Harassment.

Hum Gene Ther 2019 Mar;30(3):249-251

2 Editor-in-Chief.

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http://dx.doi.org/10.1089/hum.2019.028DOI Listing
March 2019

Top Five Gene Therapy Stories of 2019.

Authors:
Terence R Flotte

Hum Gene Ther 2019 Jan;30(1):1-2

Editor-in-Chief.

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http://dx.doi.org/10.1089/hum.2018.29080.trfDOI Listing
January 2019

The Year in Review: The Top Five Papers of 2018.

Authors:
Terence R Flotte

Hum Gene Ther 2018 12;29(12):1339-1340

Editor-in-Chief.

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http://dx.doi.org/10.1089/hum.2018.29079.trfDOI Listing
December 2018

DNA Vaccination in 2018: An Update.

Hum Gene Ther 2018 09;29(9):963-965

1 Editors of HGT Special Issue on DNA Vaccines, University of Massachusetts Medical School , Worcester, Massachusetts.

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https://www.liebertpub.com/doi/10.1089/hum.2018.29073.trf
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http://dx.doi.org/10.1089/hum.2018.29073.trfDOI Listing
September 2018

In Vivo Genome Editing Partially Restores Alpha1-Antitrypsin in a Murine Model of AAT Deficiency.

Hum Gene Ther 2018 08 14;29(8):853-860. Epub 2018 May 14.

1 RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, Massachusetts.

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http://dx.doi.org/10.1089/hum.2017.225DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6110121PMC
August 2018

Alpha-1 Antitrypsin Deficiency as a Candidate for Gene Editing.

Authors:
Terence R Flotte

Hum Gene Ther 2018 08;29(8):843-844

Editor-in-Chief.

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http://dx.doi.org/10.1089/hum.2018.29070.trfDOI Listing
August 2018

Airway Basal Cells Are the Key for Cystic Fibrosis Gene Therapy.

Authors:
Terence R Flotte

Hum Gene Ther 2018 06;29(6):641-642

Editor-in-Chief.

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http://dx.doi.org/10.1089/hum.2018.29066.trfDOI Listing
June 2018

Quantification of Total Human Alpha-1 Antitrypsin by Sandwich ELISA.

Methods Mol Biol 2017 ;1639:211-216

Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, 01605, USA.

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http://dx.doi.org/10.1007/978-1-4939-7163-3_20DOI Listing
April 2018

Therapeutics: Gene Therapy for Alpha-1 Antitrypsin Deficiency.

Methods Mol Biol 2017 ;1639:267-275

Horae Gene Therapy Center, University of Massachusetts Medical School, Suite 340, 55 Lake Avenue North, Worcester, MA, 01655, USA.

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http://link.springer.com/10.1007/978-1-4939-7163-3_27
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http://dx.doi.org/10.1007/978-1-4939-7163-3_27DOI Listing
April 2018

The rapidly evolving state of gene therapy.

FASEB J 2018 04;32(4):1733-1740

Horae Gene Therapy Center, Department of Pediatrics, University of Massachusetts Medical School, Worcester, Massachusetts, USA.

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http://dx.doi.org/10.1096/fj.201700982RDOI Listing
April 2018

It Is Time for Zero Tolerance for Sexual Harassment in Academic Medicine.

Acad Med 2018 02;93(2):163-165

C.K. Bates is associate dean for faculty affairs and associate professor of medicine, Harvard Medical School, Boston, Massachusetts. R. Jagsi is professor and deputy chair, Department of Radiation Oncology, University of Michigan Medical School, Ann Arbor, Michigan; ORCID: https://orcid.org/0000-0001-6562-1228. L.K. Gordon is senior associate dean of diversity affairs and professor of ophthalmology, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, California. E. Travis is associate vice president of women and minority faculty inclusion and professor, Department of Experimental Radiation Oncology, University of Texas MD Anderson Cancer Center, Houston, Texas. A. Chatterjee is professor and chair, Department of Pediatrics, and senior associate dean of faculty development, University of South Dakota Sanford School of Medicine, Sioux Falls, South Dakota. M. Gillis is professor, chief of division, and director of faculty advancement, Department of Humanities, Health, and Society, Herbert Wertheim College of Medicine, Florida International University, Miami, Florida. O. Means is a medical student, University of South Alabama College of Medicine, Mobile, Alabama. L. Chaudron is professor of psychiatry, pediatrics, and obstetrics and gynecology and associate vice president and senior associate dean for inclusion and culture development, University of Rochester School of Medicine and Dentistry, Rochester, New York. R. Ganetzky is an attending physician, Mitochondrial Medicine Center, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania. M. Gulati is professor of medicine and chief of cardiology, University of Arizona, Phoenix, Arizona. B. Fivush is associate dean for women in science and medicine and professor of pediatrics, Johns Hopkins University School of Medicine, Baltimore, Maryland. P. Sharma is professor and chair, Department of Pathology, Creighton University School of Medicine and CHI Health, Omaha, Nebraska. A. Grover is associate professor of surgery, Surgical Oncology, Virginia Commonwealth University Health, Richmond, Virginia. D. Lautenberger is director, Constituent Engagement, Women in Medicine and Science and Diversity and Inclusion, Association of American Medical Colleges, Washington, DC. T.R. Flotte is dean, School of Medicine, provost and executive deputy chancellor, and Celia and Isaac Haidak Professor of Medical Education, University of Massachusetts, Worcester, Massachusetts.

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http://dx.doi.org/10.1097/ACM.0000000000002050DOI Listing
February 2018

Gene and Cell Therapy in 2018: A Look Ahead.

Authors:
Terence R Flotte

Hum Gene Ther 2018 01;29(1)

Editor-in-Chief.

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http://dx.doi.org/10.1089/hum.2017.29060.trfDOI Listing
January 2018

Therapeutic Advances in Germany and Beyond.

Authors:
Terence R Flotte

Hum Gene Ther 2017 12;28(12):1117

Editor-in-Chief.

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http://www.liebertpub.com/doi/10.1089/hum.2017.29059.trf
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http://dx.doi.org/10.1089/hum.2017.29059.trfDOI Listing
December 2017

Survival Advantage of Both Human Hepatocyte Xenografts and Genome-Edited Hepatocytes for Treatment of α-1 Antitrypsin Deficiency.

Mol Ther 2017 11 25;25(11):2477-2489. Epub 2017 Sep 25.

Department of Pediatrics and Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA 01605, USA. Electronic address:

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http://dx.doi.org/10.1016/j.ymthe.2017.09.020DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5675605PMC
November 2017

Gene Therapy for Alcoholism and Other Substance Use Disorders.

Authors:
Terence R Flotte

Hum Gene Ther 2017 09;28(9):701-702

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http://dx.doi.org/10.1089/hum.2017.29048.trfDOI Listing
September 2017

Sleeping Beauty Awakens New Interest.

Authors:
Terence R Flotte

Hum Gene Ther 2017 07;28(7):533

Editor-in-Chief.

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http://dx.doi.org/10.1089/hum.2017.29046.trfDOI Listing
July 2017

One More Controversy: Adeno-Associated Virus in Stem Cells.

Authors:
Terence R Flotte

Hum Gene Ther 2017 06;28(6):449

Editor-in-Chief.

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http://dx.doi.org/10.1089/hum.2017.29043.trfDOI Listing
June 2017

CAR T-Cell Therapy: Progress and Prospects.

Hum Gene Ther Methods 2017 04;28(2):61-66

2 Department of Pediatrics and Horae Gene Therapy Center, University of Massachusetts Medical School , Worcester, Massachusetts.

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http://dx.doi.org/10.1089/hgtb.2016.153DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5429042PMC
April 2017

AAV Is Now a Medicine: We Had Better Get This Right.

Hum Gene Ther 2017 04;28(4):307

2 Editor.

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http://dx.doi.org/10.1089/hum.2017.29041.trfDOI Listing
April 2017

The Negative Effects of Immigration Restrictions on the Gene Therapy Community.

Authors:
Terence R Flotte

Hum Gene Ther 2017 03;28(3):227

Editor-in-Chief.

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http://dx.doi.org/10.1089/hum.2017.29040.trfDOI Listing
March 2017

Retro-Orbital Venous Sinus Delivery of rAAV9 Mediates High-Level Transduction of Brain and Retina Compared with Temporal Vein Delivery in Neonatal Mouse Pups.

Hum Gene Ther 2017 03;28(3):228-230

1 Horae Gene Therapy Center, University of Massachusetts Medical School , Worcester, Massachusetts.

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http://dx.doi.org/10.1089/hum.2017.037DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5824662PMC
March 2017

Class I-restricted T-cell responses to a polymorphic peptide in a gene therapy clinical trial for α-1-antitrypsin deficiency.

Proc Natl Acad Sci U S A 2017 02 30;114(7):1655-1659. Epub 2017 Jan 30.

Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104;

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http://dx.doi.org/10.1073/pnas.1617726114DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5320988PMC
February 2017

Empty Adeno-Associated Virus Capsids: Contaminant or Natural Decoy?

Authors:
Terence R Flotte

Hum Gene Ther 2017 02;28(2):147-148

Editor-in-Chief.

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http://dx.doi.org/10.1089/hum.2017.29039.trfDOI Listing
February 2017

The Science Policy Implications of a Trump Presidency.

Authors:
Terence R Flotte

Hum Gene Ther 2017 01 6;28(1):1-2. Epub 2016 Dec 6.

Editor-in-Chief.

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http://www.liebertpub.com/doi/10.1089/hum.2016.29037.trf
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http://dx.doi.org/10.1089/hum.2016.29037.trfDOI Listing
January 2017

Developing Core Competencies for the Prevention and Management of Prescription Drug Misuse: A Medical Education Collaboration in Massachusetts.

Acad Med 2016 Oct;91(10):1348-1351

K.H. Antman is dean, Boston University School of Medicine, and provost, Boston University Medical Campus, Boston, Massachusetts.H.A. Berman is dean and professor of public health and community medicine, Tufts University School of Medicine, Boston, Massachusetts.T.R. Flotte is dean, provost and executive deputy chancellor, and Celia and Isaac Haidak Professor of Medical Education, University of Massachusetts Medical School, Worcester, Massachusetts.J. Flier is dean, Harvard Medical School, Boston, Massachusetts.D.M. Dimitri is clinical associate professor, Department of Family Medicine and Community Health, University of Massachusetts Medical School, Worcester, Massachusetts, and past president, Massachusetts Medical Society, Waltham, Massachusetts.M. Bharel is commissioner, Massachusetts Department of Public Health, Boston, Massachusetts.

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http://dx.doi.org/10.1097/ACM.0000000000001347DOI Listing
October 2016

The Renaissance of Gene and Cell Therapy: Florence 2016.

Hum Gene Ther 2016 10;27(10):727-728

3 Editor-in-Chief, Human Gene Therapy.

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http://dx.doi.org/10.1089/hum.2016.29034.lnaDOI Listing
October 2016

Gene Drives: Biological Shield or Ecological Menace?

Authors:
Terence R Flotte

Hum Gene Ther 2016 08;27(8):561-2

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http://dx.doi.org/10.1089/hum.2016.29032.trfDOI Listing
August 2016

The Target's the Thing.

Authors:
Terence R Flotte

Hum Gene Ther 2016 07;27(7):477

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http://dx.doi.org/10.1089/hum.2016.29030.trfDOI Listing
July 2016

Why Human Gene Therapy Scientists Should Care About Model Organisms.

Authors:
Terence R Flotte

Hum Gene Ther 2016 May;27(5):339-40

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http://dx.doi.org/10.1089/hum.2016.29026.trfDOI Listing
May 2016

Development of rAAV2-CFTR: History of the First rAAV Vector Product to be Used in Humans.

Hum Gene Ther Methods 2016 Apr 19;27(2):49-58. Epub 2016 Feb 19.

1 Horae Gene Therapy Center, University of Massachusetts Medical School , Worcester, Massachusetts.

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http://dx.doi.org/10.1089/hgtb.2015.150DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4834522PMC
April 2016

A Gene Therapy Scientist's Life Well-Lived.

Authors:
Terence R Flotte

Hum Gene Ther 2016 Apr;27(4):279

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http://dx.doi.org/10.1089/hum.2016.29023.trfDOI Listing
April 2016

Special Note from the Editor-in-Chief.

Authors:
Terence R Flotte

Hum Gene Ther 2016 Feb;27(2):95

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http://dx.doi.org/10.1089/hum.2016.29019.trfDOI Listing
February 2016

Recombinant Adeno-Associated Virus Vector Genomes Take the Form of Long-Lived, Transcriptionally Competent Episomes in Human Muscle.

Hum Gene Ther 2016 Jan;27(1):32-42

1 The Children's Hospital of Philadelphia and the University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania.

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http://dx.doi.org/10.1089/hum.2015.136DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5374867PMC
January 2016

Ethical Implications of the Cost of Molecularly Targeted Therapies.

Authors:
Terence R Flotte

Hum Gene Ther 2015 Sep;26(9):573-4

Editor in Chief.

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http://dx.doi.org/10.1089/hum.2015.29010.trfDOI Listing
September 2015

Delivery of Adeno-Associated Virus Gene Therapy by Intravascular Limb Infusion Methods.

Hum Gene Ther Clin Dev 2015 Sep 10;26(3):159-64. Epub 2015 Sep 10.

1 Gene Therapy Center, University of Massachusetts Medical School , Worcester, Massachusetts.

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http://dx.doi.org/10.1089/humc.2015.116DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4606036PMC
September 2015

The End of the Beginning of Gene Therapy.

Authors:
Terence R Flotte

Hum Gene Ther 2015 Jul;26(7):407-8

Editor-in-Chief.

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http://dx.doi.org/10.1089/hum.2015.29003.trfDOI Listing
July 2015

Progress with Recombinant Adeno-Associated Virus Vectors for Gene Therapy of Alpha-1 Antitrypsin Deficiency.

Hum Gene Ther Methods 2015 Jun;26(3):77-81

1 Gene Therapy Center, University of Massachusetts Medical School , Worcester, MA 01655.

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http://dx.doi.org/10.1089/hgtb.2015.086DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4559188PMC
June 2015

Therapeutic Germ Line Alteration: Has CRISPR/Cas9 Technology Forced the Question?

Authors:
Terence R Flotte

Hum Gene Ther 2015 May 30;26(5):245-6. Epub 2015 Apr 30.

Incoming Editor-in-Chief.

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http://dx.doi.org/10.1089/hum.2015.28999.tflDOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4442580PMC
May 2015

Current status of gene therapy for α-1 antitrypsin deficiency.

Expert Opin Biol Ther 2015 Mar 3;15(3):329-36. Epub 2014 Nov 3.

University of Massachusetts Medical School, Gene Therapy Center, Department of Pediatrics , Suite 340 55 Lake Avenue North Worcester, MA 01655 , USA +1 508 856 2107 ; +1 508 856 8181 ;

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http://dx.doi.org/10.1517/14712598.2015.978854DOI Listing
March 2015

What is suppression of anti-adeno-associated virus capsid T-cells achieving?

Hum Gene Ther 2014 Mar;25(3):178-9

Gene Therapy Center and Department of Pediatrics, University of Massachusetts Medical School , Worcester, MA 01655.

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http://dx.doi.org/10.1089/hum.2014.2506DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4442564PMC
March 2014

A single intravenous rAAV injection as late as P20 achieves efficacious and sustained CNS Gene therapy in Canavan mice.

Mol Ther 2013 Dec 2;21(12):2136-47. Epub 2013 Jul 2.

1] Gene Therapy Center, University of Massachusetts Medical School, Worcester, Massachusetts, USA [2] Department of Microbiology & Physiological Systems, University of Massachusetts Medical School, Worcester, Massachusetts, USA.

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http://dx.doi.org/10.1038/mt.2013.138DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3863789PMC
December 2013

Birth of a new therapeutic platform: 47 years of adeno-associated virus biology from virus discovery to licensed gene therapy.

Authors:
Terence R Flotte

Mol Ther 2013 Nov;21(11):1976-81

Gene Therapy Center, Department of Pediatrics, University of Massachusetts Medical School, Worcester, Massachusetts, USA.

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http://dx.doi.org/10.1038/mt.2013.226DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3831048PMC
November 2013

Gene transfer in skeletal and cardiac muscle using recombinant adeno-associated virus.

Curr Protoc Microbiol 2013 ;Chapter 14:Unit 14D.3

Gene Therapy Center, University of Massachusetts Medical School, Worcester Massachusetts, USA.

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http://dx.doi.org/10.1002/9780471729259.mc14d03s28DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3641885PMC
July 2013

Effect of allergy and inflammation on eicosanoid gene expression in CFTR deficiency.

J Cyst Fibros 2013 May 15;12(3):258-65. Epub 2012 Sep 15.

Department of Neuroscience, College of Medicine, University of Florida, Gainesville, FL 32610, United States.

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http://dx.doi.org/10.1016/j.jcf.2012.08.014DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4086715PMC
May 2013

Gene-based therapy for alpha-1 antitrypsin deficiency.

COPD 2013 Mar;10 Suppl 1:44-9

Gene Therapy Center and Department of Pediatrics, University of Massachusetts Medical School, Worcester, Massachusetts 01655, USA.

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http://dx.doi.org/10.3109/15412555.2013.764978DOI Listing
March 2013

Histone deacetylase inhibitor (HDACi) suberoylanilide hydroxamic acid (SAHA)-mediated correction of α1-antitrypsin deficiency.

J Biol Chem 2012 Nov 20;287(45):38265-78. Epub 2012 Sep 20.

Department of Cell Biology, The Scripps Research Institute, La Jolla, California 92037, USA.

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http://dx.doi.org/10.1074/jbc.M112.404707DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3488095PMC
November 2012

Gene transfer in the lung using recombinant adeno-associated virus.

Curr Protoc Microbiol 2012 Aug;Chapter 14:Unit14D.2

Gene Therapy Center, Department of Pediatrics, University of Massachusetts Medical School, Worcester, Massachusetts, USA.

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http://dx.doi.org/10.1002/9780471729259.mc14d02s26DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3486738PMC
August 2012

Long-term correction of very long-chain acyl-coA dehydrogenase deficiency in mice using AAV9 gene therapy.

Mol Ther 2012 Jun 6;20(6):1131-8. Epub 2012 Mar 6.

Gene Therapy Center, Department of Pediatrics, University of Massachusetts Medical School, Worcester, Massachusetts, USA.

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http://dx.doi.org/10.1038/mt.2012.39DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3370259PMC
June 2012

Cell and gene therapy for genetic diseases: inherited disorders affecting the lung and those mimicking sudden infant death syndrome.

Hum Gene Ther 2012 Jun;23(6):548-56

Gene Therapy Center and Department of Pediatrics, University of Massachusetts Medical School, Worcester, MA 01655, USA.

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http://dx.doi.org/10.1089/hum.2012.087DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3392613PMC
June 2012

Preclinical study design for rAAV.

Methods Mol Biol 2011 ;807:317-37

Department of Pediatrics, University of Massachusetts Medical School Gene Therapy Center, Worcester, MA, USA.

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http://dx.doi.org/10.1007/978-1-61779-370-7_14DOI Listing
March 2012

No immune responses by the expression of the yeast Ndi1 protein in rats.

PLoS One 2011 3;6(10):e25910. Epub 2011 Oct 3.

Department of Molecular and Experimental Medicine, The Scripps Research Institute, La Jolla, California, United States of America.

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http://journals.plos.org/plosone/article?id=10.1371/journal.pone.0025910PLOS
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3185062PMC
February 2012

The role of gene and cell therapy in the era of health care reform.

Hum Gene Ther 2011 Nov 24;22(11):1307-9. Epub 2011 Oct 24.

University of Massachusetts Medical School and UMass Memorial Healthcare, Inc., Worcester, MA 01655, USA.

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http://dx.doi.org/10.1089/hum.2011.2526DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6463979PMC
November 2011

Recombinant adeno-associated virus-mediated gene transfer for the potential therapy of adenosine deaminase-deficient severe combined immune deficiency.

Hum Gene Ther 2011 Aug 13;22(8):935-49. Epub 2011 Mar 13.

Department of Pediatrics and Powell Gene Therapy Center, University of Florida College of Medicine, Gainesville, FL 32610, USA.

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http://dx.doi.org/10.1089/hum.2010.121DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6468955PMC
August 2011

N-glycosylation augmentation of the cystic fibrosis epithelium improves Pseudomonas aeruginosa clearance.

Am J Respir Cell Mol Biol 2011 Jun 6;44(6):824-30. Epub 2010 Aug 6.

Department of Molecular Genetics and Microbiology, College of Medicine, University of Florida, Gainesville, Florida, USA.

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http://dx.doi.org/10.1165/rcmb.2009-0285OCDOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3135844PMC
June 2011

Lack of cystic fibrosis transmembrane conductance regulator in CD3+ lymphocytes leads to aberrant cytokine secretion and hyperinflammatory adaptive immune responses.

Am J Respir Cell Mol Biol 2011 Jun 19;44(6):922-9. Epub 2010 Aug 19.

University of Massachusetts Medical School Department of Pediatrics and Gene Therapy Center, Worcester, Massachusetts 01605, USA.

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http://dx.doi.org/10.1165/rcmb.2010-0224OCDOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3135852PMC
June 2011

Cellular fusion for gene delivery to SCA1 affected Purkinje neurons.

Mol Cell Neurosci 2011 May 17;47(1):61-70. Epub 2011 Mar 17.

Department of Neuroscience, The Evelyn F. and William L. McKnight Brain Institute of the University of Florida, USA.

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http://dx.doi.org/10.1016/j.mcn.2011.03.003DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3100720PMC
May 2011

Moving forward toward a cure for Parkinson's: neuropathology of the nigrostriatal pathway determines the location of growth factor delivery.

Mol Ther 2011 May;19(5):827-9

Gene Therapy Center and Department of Pediatrics, University of Massachusetts Medical School, Worcester, Massachusetts 01520, USA.

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http://dx.doi.org/10.1038/mt.2011.76DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3098651PMC
May 2011

Gene therapy for alpha-1 antitrypsin deficiency.

Hum Mol Genet 2011 Apr 16;20(R1):R87-92. Epub 2011 Apr 16.

Gene Therapy Center and Department of Pediatrics, University of Massachusetts Medical School, S1-340, 55 Lake Avenue North, Worcester, MA 01655, USA.

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http://dx.doi.org/10.1093/hmg/ddr156DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3095063PMC
April 2011

Autoimmunity in a genetic disease—a cautionary tale.

N Engl J Med 2010 Oct;363(15):1473-5

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http://dx.doi.org/10.1056/NEJMe1009056DOI Listing
October 2010

Dual reporter comparative indexing of rAAV pseudotyped vectors in chimpanzee airway.

Mol Ther 2010 Mar 13;18(3):594-600. Epub 2009 Oct 13.

Department of Pediatrics, University of Massachusetts Medical School, Worcester, Massachusetts 01655, USA.

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http://dx.doi.org/10.1038/mt.2009.230DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2839428PMC
March 2010

Modulation of exaggerated-IgE allergic responses by gene transfer-mediated antagonism of IL-13 and IL-17e.

Mol Ther 2010 Mar 24;18(3):511-8. Epub 2009 Nov 24.

Gene Therapy Center, University of Massachusetts Medical School, Worcester, Massachusetts 01605, USA.

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http://dx.doi.org/10.1038/mt.2009.264DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2839443PMC
March 2010