Mandana Arbab

Mandana Arbab is an Assistant Professor of Neurology at Harvard Medical School and leads the Arbab Lab at the Translational Neuroscience Center of Boston Children’s Hospital. Her research focuses on advancing genome editing platforms to study and treat neurodegenerative diseases, including spinal muscular atrophy (SMA), Huntington’s disease, and Friedreich’s ataxia.

Research Interests

Dr. Arbab’s work centers on developing precise CRISPR-based technologies, such as base editing and prime editing, to correct disease-causing mutations or modulate genomic activity. Her lab employs machine learning to optimize editing outcomes and explores therapeutic strategies in cellular and murine models.

Publications Summary

Recent studies highlight her innovations in C•G-to-G•C base editing, predictive algorithms for edit outcomes, and cloning-free CRISPR methodologies. Her work bridges genomic engineering and neurology, aiming to translate these tools into clinical applications.

Labs & Collaborations

The Arbab Lab, based at Boston Children’s Hospital, fosters a diverse and collaborative environment. With a team of 5-10 members, they partner with institutions to develop gene-based therapeutics and investigate cellular mechanisms of neurodegeneration.

Contact: mandana.arbab@childrens.harvard.edu | arbablab.com