Gene Editing of Stem Cells for Universal Cell Therapy - Joanna Brewer

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Development and Regulation of Immunotherapy - Klaus Cichutek

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Economic and Commercial Bases for Pricing of Genetic Therapies - Eric Auger

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Electroporation-mediated Gene Delivery to the Heart for Coronary Artery Disease - Richard Heller

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Engineered T cells for Cancer Therapy in 2018 - Saar Gill

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Engraftment and Phenotypic Correction of Hematopoietic Stem Cells in Fanconi Anemia Patients

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Ethics: from Clinical Trials to Products - Jonathan Kimmelman

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Fundamentals of T cell-based Immunotherapy - Nicholas Restifo

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Gene Correction at Targeted DNA Nicks - Nancy Maizels

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Developing Non-viral/Nanoparticle Gene Delivery for Retinal Degeneration - Muna Naash

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Gene Therapy for X-Linked Chronic Granulomatous Disease - Donald Kohn

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Genome Editing in Human Stem Cells for Blood Disease Modeling and Treatment - Linzhao Cheng

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History of CAR Approval and Next Generation CARs - Carl June

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How Pharma Establishes Prices of Genetic Therapies - Jason Meyenburg

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Immunogenicity of AAV Vectors and Transgenes - Roland Herzog

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In Utero Gene Correction Mediated by PNA-Nanoparticles - Adele Ricciardi

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Inducing Immune Tolerance with Liver-directed Expression by AAV - Dwight Koeberl

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Cardiac Regeneration - Amit Patel

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A Phase I Clinical Trial of Malignant Pleural Disease Treated...Prasad Adusumilli

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AAV Gene Therapy to CNS: Past, Present and Future - Miguel Sena-Esteves

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AAV Vectors in the Eye - Shannon Boye

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AAV Vectors in the Liver - Ian Alexander

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Adipose Stromal Cell Therapy for Osteoarthritis of the Knee - Frank Barry

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Ancestral AAV Capsid Reconstruction: Interrogating Viral Evolution to Inform Rational AAV Design

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Autologous Tgf?-Resistant HPV-16/18 E6/E7-Specific T Lymphocytes...for the Treatment of Cancers

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Bioethics of Genome Editing - Silvia Camporesi

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Induction of HbF to Treat ?-hemoglobinopathies- Bringing CRISPR to the Clinic - Tony Ho

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Cell Therapeutics for Pulmonary Disease - Daniel Weiss

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Clinical Electrochemotherapy and Gene Electrotransfer for Tumor Treatment - Maja Cemazar

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Clonal Tracking of Genetically Engineered Hematopoietic Cells in Humans - Luca Biasco

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CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients in hiPSC-derived Muscle

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Decrease in B-Lymphocyte Levels...Reduces the Efficacy of Autologous Bone Marrow Cell Therapy

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Design and Translational Application of HSV Gene Vectors in Nervous System Diseases

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Design, Formulation and Testing of Systemically Dosed Peptide and Polymer DNA and mRNA Nanoparticles

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Thalassemia Clinical Trials - Alexandria Petrusich

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Reprogramming Human T Cell Circuitry - Alexander Marson

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Retinal Progenitor Cells for Treatment of Retinitis Pigmentosa - Henry Klassen

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SCID Immunodeficiency Trial - Brian Sorrentino

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Seeing the Light with Retinal Gene Therapy: From Fantasy to Reality - Jean Bennett

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Small RNAs for Cardiac Gene Editing and Regeneration - Mauro Giacca

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Sticker Shock: How to Purchase a CAR - Carl June

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Structure-Function Relationship in Chimeric Antigen Receptors - Maksim Mamonkin

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Targeting Onco-microRNA Dependence of Malignant Glioma by Gene Editing - Anna Krichevsky

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Regulatory T-cells for Inducing Tolerance to FVIII - Carol Miao

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The Challenge to Develop and Commercialize Gene-Based Therapies for Rare Diseases

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Translating RNAi for Huntington’s Disease - Megan Keiser

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Transplantation and Persistence of CRISPR/Cas9-Edited Hematopoietic Stem and Progenitors Cells

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Treatment of Recurrent HGG Patients with the Retroviral Replicating Vector Toca 511 and Toca FC

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Turning Genes into Medicines: Therapeutics for the New Millenium - Katherine High

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Update on Phase I/II Clinical Trial of Intracerebral Administration of rAAV2/5hNAGLU Vector

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Viral Infections in Transplantation - Ann Leen

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Update on Phase I/II Clinical Trial for Ornithine Transcarbamylase (OTC) Deficiency Using AAV

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Reducing the Immunogenicity of AAV through Engineering the Vector - George Church

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Reconciling the Stem/Progenitor and Paracrine Functions of MSCs - Donald Phinney

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Readministration of AAV vector in Pompe Disease - Manuela Corti

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Pluripotent Cell-Derived NK Cells as a Cornerstone Approach for Off-the-Shelf Cancer Immunotherapy

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Phase III Trial for Treatment of Cervical Dysplasia Caused by Human Papillomavirus - Mark Bagarazzi

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Phase I/II Clinical Trial for Recessive Dystrophic Epidermolysis Bullosa Using EB-101 - J.Y. Tang

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Patient Issues and Opportunities for Fair Access to Gene-based Therapies in Rare Disease

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Panel Discussion

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Next-generation Approaches for Rapid Production of Nanoparticles for Use in Gene and Cell Therapies

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Nucleic Acid Delivery Systems for RNA Therapy and Gene Editing - Daniel Anderson

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Noninvasive Imaging Technologies to Monitor Biodistribution, Activity, and Toxicity of Viruses

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New AAVs for Sensory Organ Targeting - Casey Maguire

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New AAVs for Central Nervous System Targeting - David Schaffer

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MR Image-Guided Transfection of the CNS with Focused Ultrasound and Brain-Penetrating Nanoparticles

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Melanoma Skin Trial - Robert Andtbacka

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13ème Assises de Génétiques Humaine et Médicale 2025

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• 693    
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ASHG Conférence Annuelle 2025

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• 1356    
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28ème Congrès annuel de l'American Society of Gene & Cell Therapy - ASGCT 2025

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• 961    
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51ème Congrès Annule de l'EBMT

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• 1042    
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Empowering patients 2025 : un sommet de l'American Society of Gene and Cell Therapies – ASGCT 2025

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• 691    
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