Benjamin P. Kleinstiver
The Kleinstiver group takes engineering-focused approaches to develop new genome engineering technologies for research applications and for the treatment of human diseases. They have synergistic efforts in the fields of genome editing technology development, protein engineering, directed evolution, and pre-clinical efforts to develop new genetic therapies. Some of the goals of the Kleinstiver lab include:
To develop safe, effective, and versatile technologies capable of editing genomes.
To optimize novel genome editing tools that overcome the inherent limitations of naturally occurring CRISPR enzymes, while also expanding the capabilities within the genome editing ‘toolbox’.
To engineer enzymes with enhanced properties to enable the precise installation of small, medium, or kilobase-sized genome edits (e.g. base editors, prime editors, click editors, or larger sequence editors).
To deploy novel editing enzymes in pre-clinical studies to treat various human diseases including inborn errors of immunity, spinal muscular atrophy, deafness disorders, Huntington’s disease, sickle cell disease, vascular diseases, and others of major unmet need.
To overcome the challenge of heterogeneity of patient mutations, which imposes challenges to scale, develop, and access to new genetic therapies.
The engineered enzymes developed by the Kleinstiver lab have enhanced properties including dramatically broadened genomic access (making the entire genome targetable and thus ‘druggable’ from an editing perspective), improved safety by minimizing off-target edits, increased efficiency to maximize editing outcomes, a higher propensity to result in more defined edit-outcomes, and those with entirely new editing capabilities. These innovations are in principle applicable to a range of different diseases. Their tech dev research naturally lends itself to fruitful collaborative efforts, partnering with clinicians and researchers with diverse expertise to solve high-impact problems and accelerate their synergistic goals. More broadly, they seek to push the boundaries of scientific discovery by creatively developing solutions to important challenges, while cultivating a welcoming and productive environment for trainees of all career stages to learn, grow, and succeed in their research group.
For recent manuscripts, please visit: https://www.kleinstiverlab.org/publications
For recent news from the Kleinstiver lab, please visit: https://www.kleinstiverlab.org/