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Science
Overview
Zinc Fingers
Cell Therapy
Genome Regulation
Bioethics
Publications
Presentations
Manufacturing Capabilities
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Programs
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Patients
Our Commitment
Patient Stories
Bioethics
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ASGCT 2023
Epigenetic Regulation for Neurology
Viral Delivery for Neurology
Genomic Engineering Platform Evolution
Assays to Support Clinical Programs
Epigenetic Regulation for Neurology
ASGCT 2023
Zinc Finger Transcriptional Regulator Mediated Repression of SCN9A Gene as a Therapeutic Approach for Painful Peripheral Neuropathies
ASGCT 2023
Engineered Zinc Finger Transcriptional Regulators Specifically Reduce Prion Expression and Extend Survival in an Aggressive Prion Disease Model
ASGCT 2023
Gene Activation Mediated by Zinc Finger Transcriptional Regulators (ZF-TRs) as a Therapeutic Approach for CNS Disorders
ASGCT 2023
Cell-Type Specific Reduction of Prion Expression in Neurons and Astrocytes Using Engineered Zinc Finger Transcriptional Regulators
ASGCT 2023
Intercellular Zinc Finger Protein Delivery for Cross-Corrective Epigenetic Regulation in the CNS
Viral Delivery for Neurology
ASGCT 2023
Evolution of Blood-Brain Barrier Penetrant AAV Capsids in Non-Human Primates Using a Multiplexed Transcription Dependent Capsid Engineering Platform
ASGCT 2023
Strategic Formulation Development for AAV Delivered Gene Therapies – a Case Study
ASGCT 2023
Fitness Maturation of Engineered AAV Capsids STAC-102 and STAC-103 Enhances Central Nervous System Transduction after CSF Administration in Cynomolgus Macaques
ASGCT 2023
Process and Platform Development for Production and Purification of CNS-Tropic Engineered AAV Capsids
Genomic Engineering Platform Evolution
ASGCT 2023
Engineered Zinc Finger Transcriptional Regulators Enable Robust and Reliable Epigenetic Regulation in the Mouse Brain
ASGCT 2023
A Robust and Flexible Baculovirus-Insect Cell System for AAV Vector Production with Improved Yield, Capsid Ratios and Potency
ASGCT 2023
Zinc Finger-Transcriptional Repressors Against Immune Checkpoint Molecules to Improve Anti-Tumor Activity of Gene-Modified T Cells
Assays to Support Clinical Programs
ASGCT 2023
Overcoming the Effect of Previous Enzyme Replacement Therapy on the Detection of Anti-Transgene Protein Antibodies After Treatment with Gene Therapy
ASGCT 2023
Development of a Competitive Ligand-Binding Assay to Detect Neutralizing Antibodies Against Chimeric Antigen Receptor of Regulatory T Cells