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The Horae (红瑞) Gene Therapy Center
About
About
Horae GTC Center
Faculty
Faculty Publications
Faculty Publications
Terence Flotte, MD
Guangping Gao, PhD
Claudio Punzo, PhD
Miguel Sena-Esteves, PhD
Neil Aronin, MD
Robert Brown, D.Phil, MD
Affiliated Faculty
Affiliates
Affiliates
Bioinformatics
RNA Therapeutics Institute (RTI)
Center for Clinical Translational Science
Sherman Center
China Program Office
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Resources Links
Labs
Labs
Flotte Lab
Gao Lab
Gray-Edwards Lab
Keeler Lab
Punzo Lab
Sena-Esteves Lab
Zhong Lab
Gene Therapy
Research
Research
Alpha-One Antitrypsin Deficiency
Amyotrophic Lateral Sclerosis (ALS)
Canavan Disease
Ciliopathies
Cystic Fibrosis
Fatty Acid Oxidation Deficiencies, VLCAD
Huntington Disease
Hypercholesterimia
Pompe Disease
Retinitis Pigmentosa
Tay-Sachs Disease
Clinical Trials
Clinical Trials
What is a Clinical Trial
Clinical Trials at UMass
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Viral Vectors
Adeno-Associated Viral (AAV) Vectors
Viral Vector Core
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Terence Flotte, MD
Guangping Gao, PhD
Claudio Punzo, PhD
Miguel Sena-Esteves, PhD
Neil Aronin, MD
Robert Brown, D.Phil, MD
Terence Flotte, MD
Guangping Gao, PhD
Claudio Punzo, PhD
Miguel Sena-Esteves, PhD
Neil Aronin, MD
Robert Brown, D.Phil, MD
Terence Flotte, MD
Terence R. Flotte, MD
Visit Lab Website
Contact Dr. Flotte
Total:
50
results
Analyzing clinical observations to better understand and manage immune responses to AAV gene therapies
Immunogenicity of Recombinant Adeno-Associated Virus (AAV) Vectors for Gene Transfer
Gene therapy for alpha-1 antitrypsin deficiency: an update
Nicholas Muzyczka, PhD [1947-2023]
Future Directions and Resource Needs for National Heart, Lung, and Blood Institute (NHLBI) Gene Therapy Research: A Report of an NHLBI Workshop
Nicholas Muzyczka, PhD
Defining Scholarship for Today and Tomorrow
ESGCT 2022: The New Normal for the Gene Therapy Community
Gene Therapy for Endocrine Disorders: A Promising Intervention
Secretion of functional α1-antitrypsin is cell type dependent: Implications for intramuscular delivery for gene therapy
Liver-directed SERPINA1 gene therapy attenuates progression of spontaneous and tobacco smoke-induced emphysema in α1-antitrypsin null mice
Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders
Gene Therapy and the Use of Animal Models: Why Mice Alone Are Not Sufficient
AAV gene therapy for Tay-Sachs disease
Gene Therapy for Rare Neurological Disorders
Modulating immune responses to AAV by expanded polyclonal T-regs and capsid specific chimeric antigen receptor T-regulatory cells
Precision of Exon Skipping with U7 Constructs
ESGCT 2021: Virtually Pan-European
In vivo gene editing works in humans: Results of a phase 1 clinical trial for TTR amyloidosis
Immune Responses to Recombinant Adenoviruses As Gene Therapy Vectors and COVID-19 Vaccines: A Two-Edged Sword
Base Editing to the Rescue
It Is Time to Call Anti-Asian Bias What It Is: Racism
Gene Therapy in Global Health: Meeting the Needs of Chinese Patients with Beta-Thalassemia
Women Lead Gene Therapy Science in 2021
Supporting Families Considering Participation in a Clinical Trial: Parent-Provider Perspectives
Improved prime editors enable pathogenic allele correction and cancer modelling in adult mice
Large-scale molecular epidemiological analysis of AAV in a cancer patient population
Common pathways to Dean of Medicine at U.S. medical schools
CRISPR/Cas-Dependent and Nuclease-Free In Vivo Therapeutic Gene Editing
CRISPR Keeps Things Fresh: Next-Generation Tools for Gene Editing
In Reply to Ramotshwana et al
Liver targeting with rAAV7: balancing tropism with immune profiles
Gene and Cell Therapy for Inherited and Acquired Immune Deficiency
Writing the Story of Gene Editing and CRISPR: An Interview with Kevin Davies, PhD
Science Policy and Funding Implications of the 2020 U.S. Election
Two-Plasmid Packaging System for Recombinant Adeno-Associated Virus
Black Lives Matter to Gene Therapy
SOD1 Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS
Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy
Accelerated Graduation and the Deployment of New Physicians During the COVID-19 Pandemic
Barrie J. Carter: The Science and the Scientist
Volume and Infusion Rate Dynamics of Intraparenchymal Central Nervous System Infusion in a Large Animal Model
Revisiting the "New" Inflammatory Toxicities of Adeno-Associated Virus Vectors
A Message to Our Community in the Midst of the COVID-19 Pandemic
Impact on Women's Health: Gene Therapy in Gynecologic Oncology
Free Gene and Cell Therapy Services Offered by the National Heart, Lung, and Blood Institute of the National Institutes of Health
Muscle-Directed Delivery of an AAV1 Vector Leads to Capsid-Specific T Cell Exhaustion in Nonhuman Primates and Humans
2020: Gene Therapy Enters Its Fourth Decade
Gene Therapy Untangles the Problem of Chronic Traumatic Encephalopathy
Ringing in the Changes
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About
About
Horae GTC Center
Faculty
Faculty Publications
Faculty Publications
Terence Flotte, MD
Guangping Gao, PhD
Claudio Punzo, PhD
Miguel Sena-Esteves, PhD
Neil Aronin, MD
Robert Brown, D.Phil, MD
Affiliated Faculty
Affiliates
Affiliates
Bioinformatics
RNA Therapeutics Institute (RTI)
Center for Clinical Translational Science
Sherman Center
China Program Office
Administrative Staff
Careers
Resources Links
Labs
Labs
Flotte Lab
Gao Lab
Gray-Edwards Lab
Keeler Lab
Punzo Lab
Sena-Esteves Lab
Zhong Lab
Gene Therapy
Research
Research
Alpha-One Antitrypsin Deficiency
Amyotrophic Lateral Sclerosis (ALS)
Canavan Disease
Ciliopathies
Cystic Fibrosis
Fatty Acid Oxidation Deficiencies, VLCAD
Huntington Disease
Hypercholesterimia
Pompe Disease
Retinitis Pigmentosa
Tay-Sachs Disease
Clinical Trials
Clinical Trials
What is a Clinical Trial
Clinical Trials at UMass
Clinical Trial Resources
Viral Vectors
Viral Vectors
Adeno-Associated Viral (AAV) Vectors
Viral Vector Core
IBC-Related Facts
News & Events
News & Events
Gene Therapy News
Past Events & Videos
GTC Labs Blog Posts
Partner with Us
Contact
Contact
Contact Information
For Media
Donate
Additional Resources