Mandana Arbab

Mandana Arbab

Assistant Professor of Neurology

Arbab Lab
3 Blackfan Circle
Boston, MA 02115
Email: mandana.arbab@childrens.harvard.edu

Website: arbablab.com
Lab Size: 5-10

Summary

Genome editing may provide a lasting treatment for many genetic disorders by correcting disease mutations or making compensatory changes to the genome. We develop platforms using genome editing tools to investigate cellular mechanisms of neurodegeneration, and develop genome editing therapeutic strategies to treat these disorders in cells and in mice.

Publications

Arbab M, Matuszek Z, Kray KM, Du A, Newby GA, Blatnik AJ, Raguram A, Richter MF, Zhao KT, Levy JM, Shen MW, Arnold WD, Wang D, Xie J, Gao G, Burghes AHM, Liu DR. Base editing rescue of spinal muscular atrophy in cells and in mice. Science. 2023 Apr 21;380(6642):eadg6518. doi: 10.1126/science.adg6518. Epub 2023 Apr 14. PMID: 36996170; PMCID: PMC10270003.

Arbab M, Shen MW, Mok B, Wilson C, Matuszek Ż, Cassa CA, Liu DR. Determinants of Base Editing Outcomes from Target Library Analysis and Machine Learning. Cell. 2020 Jul 23;182(2):463-480.e30. doi: 10.1016/j.cell.2020.05.037. Epub 2020 Jun 12. PMID: 32533916; PMCID: PMC7384975.

Shen MW, Arbab M, Hsu JY, Worstell D, Culbertson SJ, Krabbe O, Cassa CA, Liu DR, Gifford DK, Sherwood RI. Predictable and precise template-free CRISPR editing of pathogenic variants. Nature. 2018 Nov;563(7733):646-651. doi: 10.1038/s41586-018-0686-x. Epub 2018 Nov 7. Erratum in: Nature. 2019 Mar;567(7746):E1-E2. PMID: 30405244; PMCID: PMC6517069.

Koblan LW, Arbab M, Shen MW, Hussmann JA, Anzalone AV, Doman JL, Newby GA, Yang D, Mok B, Replogle JM, Xu A, Sisley TA, Weissman JS, Adamson B, Liu DR. Efficient C•G-to-G•C base editors developed using CRISPRi screens, target-library analysis, and machine learning. Nat Biotechnol. 2021 Nov;39(11):1414-1425. doi: 10.1038/s41587-021-00938-z. Epub 2021 Jun 28. PMID: 34183861; PMCID: PMC8985520.

Arbab M, Srinivasan S, Hashimoto T, Geijsen N, Sherwood RI. Cloning-free CRISPR. Stem Cell Reports. 2015 Nov 10;5(5):908-917. doi: 10.1016/j.stemcr.2015.09.022. Epub 2015 Oct 29. PMID: 26527385; PMCID: PMC4649464.