Base Editing Rescue of Spinal Muscular Atrophy in Cells and in Mice

Time: 1:30 pm
day: Day 1 Track B PM

Details:

• Presenting findings from a safe, efficient, and specific AAV-based base editing approach to SMA treatment
• Exploring the clinical relevancy of dosing schemes used to increase therapeutic windows
• Follow-up studies and research areas to drive this proof-of-concept towards the clinic

Speakers:

Mandana Arbab Manda Arbab is the Lodish Family Assistant Professor of Neurology at Harvard Medical School, and faculty member of the Rosamund Stone Zander Translational Neuroscience Center Read more