Cas9-derived fusion proteins can be effective regulators of transcription, but are too large for packaging into adeno-associated virus vectors. This technology overcomes this size limitation by engineering highly compact Cas9-based transcriptional factors.
Transcriptional reprogramming technologies are at the forefront of research and medicine. CRISPER-Cas systems have advanced the field of programmable DNA tools. In particular, Cas9-derived fusion proteins are effective at modulating transcription, but are too large for packaging into conventionally used adeno-associated virus (AAV) vectors for gene therapy. In order to use this new technology for development of precision medical treatments for a variety of diseases, the proteins must be modified to be smaller without losing efficiency.
Researchers at The Ohio State University have engineered highly compact modified Cas9-derived fusion proteins for the packaging of these effective transcriptional modulators into AAV vectors. This novel system for modulation of gene expression utilizes the modified Cas9 protein, a guide RNA, and an effector domain. In vivo delivery using AVV vectors is possible with this method, allowing for targeted genomic editing and modulation of specific sequences.
- Disease treatment by direct tuning of infectious or host transcription profiles
- Highly compact Cas9-derived transcription modulators could be packaged into other vectors for use in other species
- Highly compact expression modulators can be packaged into a wider range of viral vectors for genomic engineering
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