The Gene Writing platform addresses key limitations of gene therapy and gene editing techniques. The new technology can efficiently insert genes and exons, introduce small insertions or deletions, or change single or multiple DNA base pairs. This could help fix errors associated with monogenic disorders (caused by variation in a single gene) or allow precise gene regulation in other complex diseases (neurodegenerative diseases, autoimmune disorders, and metabolic diseases).
The technology was developed by a collaboration of Massachusetts Institute of Technology researchers and scientists at Flagship Labs. Tessera was founded in 2018 to create a platform that could design, make, and launch Gene Writing medicines. Mobile genetic elements -- genetic material that can move around within a genome -- are engineered to create the technology. RNA or DNA templates are used to write therapeutic messages into the genome.
The Gene Writing platform helps overcome many of the limitations of previous genetic medicine approaches. Specifically, it is able to do the following:
- Make alterations to the genome of somatic cells with minimal reliance upon host DNA repair pathways
- Permanently add new DNA to dividing cells
- Write new DNA sequences into the genome by delivering only RNA
- Allow repeated administration of treatments to dose to effect
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