Alexander LeNail

I develop gene therapies for neurodegenerative diseases as a postdoctoral fellow in George Church’s lab at the Wyss Institute and Harvard Medical School. I am also co-founder of Refactor Therapeutics, where I am taking gene therapy toward clinical development for X-linked adrenoleukodystrophy (ALD), a disease that runs in my family.

During my PhD at MIT, I designed and conducted a pooled in vivo Perturb-seq screen of 146 transcription factors directly in the aging mouse motor cortex, measuring the empirical regulons of each rejuvenation factor in each neuronal cell type. We identified transcription factors that induce gene-expression programs for mitochondrial biogenesis, synaptogenesis, and repair in vivo. This work brought together computational predictions, gene delivery, and single-cell measurements to test potential rejuvenation strategies in a living brain.

My background spans software engineering, computational biology, and experimental gene therapy. I’m drawn to problems that require building across these disciplines, from developing the tools to understand disease to designing interventions that might change its course.