Publications by authors named "Ronald Crystal"

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Where did my vector go and how well did it function? PET biodistribution studies of viral gene therapy vectors.
Douglas J Ballon, Ronald G Crystal

Mol Ther· September 2025

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Genes required for to survive the transition from aerosol to pulmonary alveolar lining fluid and early infection in a model of transmission.
Prabhat Ranjan Singh, Saurabh Mishra, Adrian Jinich, Xiuju Jiang, Felipe Tsang, Ronald G Crystal

bioRxiv· September 2025

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Four decades of adenovirus gene transfer vectors: History and current use.
Neil R Hackett, Ronald G Crystal

Mol Ther· May 2025

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AAVrh.10 delivery of novel APOE2-Christchurch variant suppresses amyloid and Tau pathology in Alzheimer's disease mice.
Caner Günaydin, Dolan Sondhi, Stephen M Kaminsky, Hailey C Lephart, Philip L Leopold, Ronald G Crystal

Mol Ther· December 2024

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Suppression of CNS APOE4 Expression by miRNAs Delivered by the S2 AAVrh.10 Capsid-Modified AAV Vector.
Kalpita R Karan, Slawomir Andrzejewski, Katie M Stiles, Neil R Hackett, Ronald G Crystal

Hum Gene Ther· November 2024

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Management of Select Adverse Events Following Delandistrogene Moxeparvovec Gene Therapy for Patients With Duchenne Muscular Dystrophy.
Craig M Zaidman, Natalie L Goedeker, Amal A Aqul, Russell J Butterfield, Anne M Connolly, Ronald G Crystal, Crystal M Proud

J Neuromuscul Dis· May 2024

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Angiogenic Gene Therapy for Refractory Angina: Results of the EXACT Phase 2 Trial.
Kenta Nakamura, Timothy D Henry, Jay H Traverse, David A Latter, Nahush A Mokadam, Ronald G Crystal

Circ Cardiovasc Interv· May 2024

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Expression and processing of mature human frataxin after gene therapy in mice.
Teerapat Rojsajjakul, Nithya Selvan, Bishnu De, Jonathan B Rosenberg, Stephen M Kaminsky, Ronald G Crystal

Sci Rep· April 2024

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Are Airway Epithelial p73 Levels a Therapeutic Target to Treat Chronic Obstructive Pulmonary Disease?
Ronald G Crystal

Am J Respir Crit Care Med· January 2024

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Vectorized Human Antibody-Mediated Anti-Eosinophil Gene Therapy.
Maria Gioulvanidou, Selenay Sarklioglu, Xinlei Chen, Irina V Lebedeva, Yeliz Inalman, Ronald G Crystal

Hum Gene Ther· January 2024

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Twenty-Year Survival Analysis of Adeno-Associated Virus Vector Serotype 2-Mediated Gene Therapy to the Central Nervous System for CLN2 Disease.
Dolan Sondhi, Stephen M Kaminsky, Jonathan B Rosenberg, Mahboubeh R Rostami, Neil R Hackett, Ronald G Crystal

Hum Gene Ther· January 2024

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Predicted deleterious variants in the human genome relevant to gene therapy with adeno-associated virus vectors.
Mahboubeh R Rostami, Philip L Leopold, Jenifer M Vasquez, Miguel de Mulder Rougvie, Alya Al Shakaki, Ronald G Crystal

Mol Ther Methods Clin Dev· December 2023

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Expression and processing of mature human frataxin after gene therapy in mice.
Teerapat Rojsajjakul, Nithya Selvan, Bishnu De, Jonathan B Rosenberg, Stephen M Kaminsky, Ronald G Crystal

Res Sq· December 2023

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Positron Emission Tomography Quantitative Assessment of Off-Target Whole-Body Biodistribution of I-124-Labeled Adeno-Associated Virus Capsids Administered to Cerebral Spinal Fluid.
Jonathan B Rosenberg, Edward K Fung, Jonathan P Dyke, Bishnu P De, Howard Lou, Ronald G Crystal

Hum Gene Ther· November 2023

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Serial Sampling of the Small Airway Epithelium to Identify Persistent Smoking-dysregulated Genes.
Yael Strulovici-Barel, Mahboubeh R Rostami, Robert J Kaner, Jason G Mezey, Ronald G Crystal

Am J Respir Crit Care Med· October 2023

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Gene Therapy for Immunoglobulin E, Complement-Mediated, and Eosinophilic Disorders.
Odelya E Pagovich, Ronald G Crystal

Hum Gene Ther· October 2023

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Assessment of Safety and Biodistribution of AAVrh.10hCLN2 Following Intracisternal Administration in Nonhuman Primates for the Treatment of CLN2 Batten Disease.
Bishnu P De, Jonathan B Rosenberg, Nithya Selvan, Isabelle Wilson, Nadir Yusufzai, Ronald G Crystal

Hum Gene Ther· September 2023

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Assessment of Residual Full-Length SV40 Large T Antigen in Clinical-Grade Adeno-Associated Virus Vectors Produced in 293T Cells.
Bishnu P De, Sara Cram, Hyunmi Lee, Jonathan B Rosenberg, Dolan Sondhi, Ronald G Crystal

Hum Gene Ther· August 2023

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EXACT Trial: Results of the Phase 1 Dose-Escalation Study.
Thomas J Povsic, Timothy D Henry, Jay H Traverse, R David Anderson, Geoffrey A Answini, Ronald G Crystal

Circ Cardiovasc Interv· August 2023

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Identification of Safe and Effective Intravenous Dose of AAVrh.10hFXN to Treat the Cardiac Manifestations of Friedreich's Ataxia.
Carlos Munoz-Zuluaga, Monica Gertz, Melissa Yost-Bido, Alessandria Greco, Nicholas Gorman, Ronald G Crystal

Hum Gene Ther· July 2023

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High apoptotic endothelial microparticle levels measured in asthma with elevated IgE and eosinophils.
Yael Strulovici-Barel, Robert J Kaner, Ronald G Crystal

Respir Res· July 2023

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Oral epithelium response of electronic cigarette users to electronic cigarette.
Gwendolyn S Reeve, Mahboubeh R Rostami, Renee F Reich, David A Behrman, Philip L Leopold, Ronald G Crystal

J Oral Pathol Med· May 2023

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Use of the Iron Chelator Deferiprone to Restore Function in BAL Fluid Macrophages in Smoking and Chronic Obstructive Pulmonary Disease.
Kihwan Kim, William Z Zhang, Sarah Ann Kikkers, Sarah L O'Beirne, Yael Strulovici-Barel, Ronald G Crystal

Am J Respir Cell Mol Biol· April 2023

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Gene Therapy to Treat Osteopenia Associated With Chronic Ethanol Consumption and Aldehyde Dehydrogenase 2 Deficiency.
Anna E Camilleri, Michelle Cung, Fiona M Hart, Odelya E Pagovich, Ronald G Crystal

JBMR Plus· April 2023

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Safety of Intravenous Administration of an AAV8 Vector Coding for an Oxidation-Resistant Human α1-Antitrypsin for the Treatment of α1-Antitrypsin Deficiency.
Jonathan B Rosenberg, Bishnu P De, Alessandria Greco, Nicholas Gorman, Vikrum Kooner, Ronald G Crystal

Hum Gene Ther· February 2023

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