Mandana Arbab

Mandana Arbab is the Lodish Family Assistant Professor of Neurology at Harvard Medical School and faculty member of the Rosamund Stone Zander Translational Neuroscience Center at Boston Children's Hospital.

Her research program focuses on developing precision gene editing therapies for neurodegenerative disorders. Current work employs CRISPR-based technologies to correct genetic mutations underlying conditions like spinal muscular atrophy and telomere biology disorders.

Recent publications demonstrate innovations in base editing systems, including engineering novel editors with reduced off-target effects. Her 2023 Science paper established preclinical proof-of-concept for base editing in spinal muscular atrophy.

Dr. Arbab's lab combines high-throughput screening, machine learning, and molecular biology to optimize therapeutic genome editing. She was awarded the NIH Pathway to Independence Award for her work advancing neurological gene therapies.