Nature Communications (May 2022)
Engineered Cas12i2 is a versatile high-efficiency platform for therapeutic genome editing
- Colin McGaw,
- Anthony J. Garrity,
- Gabrielle Z. Munoz,
- Jeffrey R. Haswell,
- Sejuti Sengupta,
- Elise Keston-Smith,
- Pratyusha Hunnewell,
- Alexa Ornstein,
- Mishti Bose,
- Quinton Wessells,
- Noah Jakimo,
- Paul Yan,
- Huaibin Zhang,
- Lauren E. Alfonse,
- Roy Ziblat,
- Jason M. Carte,
- Wei-Cheng Lu,
- Derek Cerchione,
- Brendan Hilbert,
- Shanmugapriya Sothiselvam,
- Winston X. Yan,
- David R. Cheng,
- David A. Scott,
- Tia DiTommaso,
- Shaorong Chong
Affiliations
- Colin McGaw
- Arbor Biotechnologies, 20 Acorn Park Drive, Tower 500
- Anthony J. Garrity
- Arbor Biotechnologies, 20 Acorn Park Drive, Tower 500
- Gabrielle Z. Munoz
- Arbor Biotechnologies, 20 Acorn Park Drive, Tower 500
- Jeffrey R. Haswell
- Arbor Biotechnologies, 20 Acorn Park Drive, Tower 500
- Sejuti Sengupta
- Arbor Biotechnologies, 20 Acorn Park Drive, Tower 500
- Elise Keston-Smith
- Arbor Biotechnologies, 20 Acorn Park Drive, Tower 500
- Pratyusha Hunnewell
- Arbor Biotechnologies, 20 Acorn Park Drive, Tower 500
- Alexa Ornstein
- Arbor Biotechnologies, 20 Acorn Park Drive, Tower 500
- Mishti Bose
- Arbor Biotechnologies, 20 Acorn Park Drive, Tower 500
- Quinton Wessells
- Arbor Biotechnologies, 20 Acorn Park Drive, Tower 500
- Noah Jakimo
- Arbor Biotechnologies, 20 Acorn Park Drive, Tower 500
- Paul Yan
- Arbor Biotechnologies, 20 Acorn Park Drive, Tower 500
- Huaibin Zhang
- Arbor Biotechnologies, 20 Acorn Park Drive, Tower 500
- Lauren E. Alfonse
- Arbor Biotechnologies, 20 Acorn Park Drive, Tower 500
- Roy Ziblat
- Arbor Biotechnologies, 20 Acorn Park Drive, Tower 500
- Jason M. Carte
- Arbor Biotechnologies, 20 Acorn Park Drive, Tower 500
- Wei-Cheng Lu
- Arbor Biotechnologies, 20 Acorn Park Drive, Tower 500
- Derek Cerchione
- Arbor Biotechnologies, 20 Acorn Park Drive, Tower 500
- Brendan Hilbert
- Arbor Biotechnologies, 20 Acorn Park Drive, Tower 500
- Shanmugapriya Sothiselvam
- Arbor Biotechnologies, 20 Acorn Park Drive, Tower 500
- Winston X. Yan
- Arbor Biotechnologies, 20 Acorn Park Drive, Tower 500
- David R. Cheng
- Arbor Biotechnologies, 20 Acorn Park Drive, Tower 500
- David A. Scott
- Arbor Biotechnologies, 20 Acorn Park Drive, Tower 500
- Tia DiTommaso
- Arbor Biotechnologies, 20 Acorn Park Drive, Tower 500
- Shaorong Chong
- Arbor Biotechnologies, 20 Acorn Park Drive, Tower 500
- DOI
- https://doi.org/10.1038/s41467-022-30465-7
- Journal volume & issue
-
Vol. 13,
no. 1
pp. 1 – 11
Abstract
Cas12i is a genome editing platform with compact size that fits in AAV vector with short 43-mer gRNA, absence of tracrRNA, ability to process pre-crRNA, and high specificity. Here the authors present an unbiased mutational scanning approach to engineer Cas12i, which shows low activity in mammalian cells, and identify single substitutions that significantly improve indel activity.
