Our Science

Scientific Publications & Presentations

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