Modified SYNGAP1 Protein Expressed in a Lentiviral Vector for the Treatment of Patients with SYNGAP1-related Intellectual Disability

Abstract

Researchers at the University of California, Davis have developed a novel stem cell gene therapy approach utilizing a modified SYNGAP1 protein to treat Synaptic Ras GTPase Activating Protein 1-related intellectual disability (SRID).

Full Description

This technology involves the modification of the wild type SYNGAP1 protein to include a secretion signal and additional N-glycan sites, allowing for its secretion and uptake by neurons. The modified SYNGAP1 gene is cloned into a lentiviral vector for expression in targeted cells, specifically designed for transduction of human CD34+ hematopoietic stem and progenitor cells. This approach aims to deliver therapeutic levels of functional SYNGAP1 to affected neurons, offering a potential treatment strategy for SRID and other neurodevelopmental disorders.

Applications

• Gene therapy for Synaptic Ras GTPase Activating Protein 1-related intellectual disability (SRID) and other neurodevelopmental disorders. 
• Stem cell therapies for genetic and neurodegenerative diseases. 
• Biotechnological research and development in gene modification and delivery systems.

Features/Benefits

• Introduction of a secretion signal and N-glycan sites enhances the delivery and uptake of SynGAP1 by neurons. 
• Utilizes lentiviral vectors for stable transduction and expression in target cells. 
• Enables cross-correction by allowing gene-modified hematopoietic stem cells to differentiate into microglia and deliver functional SynGAP1. 
• Has shown significant improvement in SRID-related phenotypes in a mouse model.

Patent Status

Patent Pending

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