Mass General Brigham’s Gene and Cell Therapy Institute (GCTI) is committed to expediting the pace of discovery and development of new gene and cell therapies by bridging the gap between research and practice. The GCTI offers expertise in core laboratory services, including the Gene Editing Core and the RNA Therapeutics Core, which serve as resources for the entire Mass General Brigham research community and beyond. Mass General Brigham plans to introduce additional lab cores in the near future, further enhancing our support for groundbreaking gene and cell therapy development.
About the RNA Therapeutics Core
The GCTI has launched the RNA Therapeutics Core (RTC), a state-of-the-art facility and resource to advance the use of RNA technologies within and beyond the Mass General Brigham research ecosystem. This new core is dedicated to accelerating the application of cutting-edge circular RNA technology to translate RNA-based medicines into clinical practice.
The RTC provides valuable tools for researchers to advance their work in RNA research, development, and therapeutics. The RTC manufactures optimized circRNAs and long noncoding RNAs (lncRNAs) and formulates them into lipid nanoparticles (LNPs). In addition to purchasing stock RNAs encoding various reporter and gene-editing proteins, customers can request custom syntheses in a range of different backbone vectors. Each order includes a basic analytics package, with additional specialized analytics available on request. The RTC has also developed unique codon optimization algorithms which may be used for custom syntheses.
As part of the GCTI's effort to support clinical translation, the RTC is currently building capacity for GMP-grade circRNA manufacturing and LNP formulation for first-in-human clinical trials. GMP manufacturing will be offered as a core service as it becomes available; please inquire with interest.
Contact the RNA Therapeutics Core
About the Gene Editing Core
The Gene Editing Core (GEC) at the GCTI is a state-of-the-art facility established to advance genome editing capabilities across the Mass General Brigham ecosystem and beyond to support gene and cell therapy development.
The GEC’s mission is to provide specialized expertise and high-quality services, including:
- Precision genome editing
- Highly sensitive off-target detection
- Patient-derived induced pluripotent stem cells (iPSC) reprogramming
- Genome perturbation screening
- Lentivirus packaging
- Tailored consulting services
The GEC leverages a comprehensive genome editing platform incorporating CRISPR/Cas9, base editing, and prime editing technologies. The core offers single-nucleotide variant (SNV), deletion and insertion editing and perturbation screening services with stringent and customizable QC assays. The workflow is optimized for high efficiency, accuracy, reproducibility, and scalability across a variety of cell types, including more complex cell types, such as iPSC, primary immune cells, and therapeutically relevant cell models.
The GEC is committed to fostering collaborative research, driving scientific innovation, and accelerating the development of transformative therapies that improve patient outcomes. Please inquire with interest.
