In vivo gene editing works in humans: Results of a phase 1 clinical trial for TTR amyloidosis

Affiliations

• ¹ Department of Neurology, University of Massachusetts Medical School, Worcester, MA 01605, USA; Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA 01605, USA.
• ² Department of Pediatrics, University of Massachusetts Medical School, Worcester, MA 01605, USA; Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA 01605, USA. Electronic address: terry.flotte@umassmed.edu.