Eyebrow:Page Title
- PPMD 2024 A Next Generation Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy June 2024
- ASGCT 2024 Novel Mechanism to Increase AAV Yield through Blocking AAV Transduction of Manufacturing HEK293 Cells During AAV Production May 2024
- ASGCT 2024 Engineered Cardioskeletal-Directed AAV Capsids That Detarget the Liver May 2024
- ASGCT 2024 Designing Therapeutic Recombinant AAV Vectors Using <em>In Silico</em> Vector Modeling May 2024
- ASGCT 2024 Identification of an AAV Affinity Chromatography Elution Buffer that Maximizes Product Recovery and Minimizes Product Degradation May 2024
- ASGCT 2024 Systemic Delivery of SGT-003 Microdystrophin Gene Therapy Using the Novel Capsid AAV-SLB101 Ameliorates Muscle Pathology and Rescues Muscle Function in the mdx Mouse Model of Duchenne Muscular Dystrophy May 2024
- ASGCT 2023 Increasing Quality and Productivity with Dual Transfection (DT) for AAV Production May 2024
- ASGCT 2024 High-Throughput Workflows to Accelerate Development of Chromatographic Purification of rAAV Viral Vectors May 2024
- Frontiers in Physiology Prolonged voluntary wheel running reveals unique adaptations in mdx mice treated with microdystrophin constructs ± the nNOS-binding site June 2023
- ASGCT 2023 Novel AAV Capsid Identification and Characterization for Neuromuscular and Cardiac Indications May 2023
- ASGCT 2023 Genomic Characterization of AAV Products Using Multiplex ddPCR and Nanopore Sequencing May 2023
- ASGCT 2023 Efficacy and Safety of a Novel AAV FXN Gene Therapy (AVB-202) for the Treatment of Friedreich's Ataxia May 2023
- ASGCT 2023 Development and Qualification of Multiplexed ddPCR Assay to Evaluate DNA Integrity May 2023
- ASGCT 2023 Characterization of Genomic Heterogeneity in rAAV Preparations Using Short- and Long-Read Next Generation Sequencing May 2023
- ASGCT 2023 Characterization of Factors that Influence the Yield and Quality of rAAV Produced Using HSV Co-Infection May 2023
- MDA 2023 IGNITE DMD One-Year Post Treatment Study Analysis March 2023
- Science Translational Medicine Assessment of Systemic AAV-Microdystrophin Gene Therapy in the GRMD Model of DMD January 2023
- Neuromuscular Disorders Clinical Potential of Microdystrophin as a Surrogate Endpoint December 2022
- WMS 2022 IGNITE DMD Long-Term Outcomes and Expression Update October 2022
- WMS 2022 Actimyo and SV95C Data Evaluation in IGNITE DMD October 2022
- ESGCT 2022 Further Characterization of a Novel AAV Vector and Expanded Selection Criteria Platform for Muscle Gene Delivery October 2022
- ESGCT 2022 Enhancement of AAV Transduction in Muscle Cells In Vitro October 2022
- ASGCT 2022 IGNITE DMD Long-Term Outcomes and Biomarker Update May 2022
- MDA 2022 IGNITE DMD 2-Year Outcomes Update March 2022
- Gene Therapy Analytical Development Summit 2021 Approaches to Capsid Characterization for AAV November 2021
- WMS 2021 IGNITE DMD Update September 2021
- Child Neurology Society Annual Meeting 2021 Pulmonary Function Results in IGNITE DMD September 2021
- ASGCT 2020 IGNITE DMD Update May 2020
- ASGCT 2020 Characterization of Novel AAV Vectors Engineered for Muscle Gene Delivery May 2020
- Neuromuscular Disorders Membrane Recruitment of nNOS in Microdystrophin Gene Transer to Enhance Durability September 2019
- PPMD Annual Conference 2019 IGNITE DMD Update June 2019
- ASGCT 2019 Identification of Novel Muscle-Specific Promoters for AAV Gene Expression in Skeletal and Cardiac Muscles May 2019
- Molecular Therapy Development of Novel Microdystrophins with Enhanced Functionality February 2019
- Parent Project Onlus Annual Conference 2019 IGNITE DMD Update February 2019
- WMS 2018 Kinetic Fingerprints in DMD October 2018
- ASGCT 2018 Assessing anti-dystrophin T-cell responses by ELISpot following AAV9-microdystrophin gene therapy in dogs May 2018
- WMS 2017 SGT-001 Posters October 2017
- Molecular Therapy A Five-Repeat Microdystrophin Gene Ameliorated Dystrophic Phenotype in the Severe DBA2J-mdx Model of DMD July 2017