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1
AAV Gene Therapy Applications for Prader-Willi Syndrome and Metabolic Disease
AAV Gene Therapy Applications for Prader-Willi Syndrome and Metabolic Disease
Queen, Nicholas Jonathan. | The Ohio State University | 2023
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2
Advancements in Viral Vector-Mediated Gene Therapy: Development and Assessment of Lentiviral and γ-Retroviral Approaches for Genetic Hematologic Disorders
Advancements in Viral Vector-Mediated Gene Therapy: Development and Assessment of Lentiviral and γ-Retroviral Approaches for Genetic Hematologic Disorders
Hart, Kevyn Lopez. | University of California, Los Angeles | 2024
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3
Pre-Clinical Modeling of Cell and Gene Therapy of Fanconi Anemia
Pre-Clinical Modeling of Cell and Gene Therapy of Fanconi Anemia
Liu, Suying. | University of Pennsylvania | 2024
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4
Engineering Adeno-Associated Virus for Protease Targeted Gene Therapy and Immune Avoidance
Engineering Adeno-Associated Virus for Protease Targeted Gene Therapy and Immune Avoidance
Robinson, Tawana Michelle. | Rice University | 2019
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5
Gene Therapy for Autosomal Dominant Polycystic Kidney Disease
Gene Therapy for Autosomal Dominant Polycystic Kidney Disease
Rubin, Jeffrey Donald. | College of Medicine - Mayo Clinic | 2021
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6
Modeling Gene Therapy for Intractable Developmental and Epileptic Encephalopathy
Modeling Gene Therapy for Intractable Developmental and Epileptic Encephalopathy
Aimiuwu, Osasumwen Virginia. | Columbia University | 2021
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7
Suppression-Replacement KCNQ1 Gene Therapy for Type 1 Long QT Syndrome
Suppression-Replacement KCNQ1 Gene Therapy for Type 1 Long QT Syndrome
Dotzler, Steven Michael. | College of Medicine - Mayo Clinic | 2021
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8
CRISPR-Based Approaches for Proteinopathies of the Central Nervous System
CRISPR-Based Approaches for Proteinopathies of the Central Nervous System
Zeballos Castro, Maria Alejandra. | University of Illinois at Urbana-Champaign | 2024
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9
Translational Design of Lipid Nanoparticles to Deliver Mrna Therapies to Solid Tumors and to the Lungs
Translational Design of Lipid Nanoparticles to Deliver Mrna Therapies to Solid Tumors and to the Lungs
Moreno, Sebastian Gonzalo Huayamares. | Georgia Institute of Technology | 2023
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10
A Fluorescence Spectroscopic Approach to Studying Nanoparticle Chemical Environments, Architecture, and Assembly
A Fluorescence Spectroscopic Approach to Studying Nanoparticle Chemical Environments, Architecture, and Assembly
Berardi, Anthony J. | University of Michigan | 2024
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