INDUCIBLE VIRAL TRANSDUCTION SYSTEM FOR IN VIVO GENOME MODIFICATION
Researchers at the Chan Zuckerberg Biohub have developed a cellular transduction method for target cell genome modification.
Gene therapy, or targeted genome modification, has great potential for biomedicine. There are several genome modification systems available for genome editing, most notably CRISPR-Cas9. Engineered recombinant viruses are the predominant method for delivering genome modification systems, for both in vitro and in vivo genome editing. However, in vivo use of viral-based gene therapeutics has resulted in low gene correction frequency with high toxicity. Therefore, improved methods for the targeted delivery of genome modification systems are needed.
Stage of Research
The inventors have developed a novel cellular transduction platform to perform in vivo genome modification. Their regulated viral vector delivery system (RVVDS) is composed of an engineered and inducible transducer cell population that produces and releases viral transduction particles (VTPs) encoding a genome modification system (GMS). Upon induction, VTPs are delivered to the target cell for desired genomic modification. This cellular transduction platform is modular and customizable for target cell type and desired genome modification.
Applications
- Targeted gene therapy applications in any nucleated cell, such as Duchenne Muscular Dystrophy (DMD) correction
Advantages
- Inducible regulated viral vector delivery system (RVVDS) for controlled RVVDS activation and viral transduction particle (VTP) production
- Induction can be mediated by target cell proximity or contact, or through exogenous small molecule treatment
- The RVVDS system is modular and customizable, with a wide range of applicable transducer cell types, including patient-derived cells
- Viral vector systems may be selected based on the nature of the target cell
Stage of Development
Research – in vitro
Publications
WO/2020/146899
Keywords
Gene therapy, genome engineering, adenoviral vectors
Technology Reference
Chan Zuckerberg CZB-117
