Helen Willsey, PhD

Assistant Professor
M_Psych-Core-Rsch

The Willsey Lab at UCSF focuses on uncovering the molecular mechanisms that drive autism spectrum disorder and its frequently co-occurring conditions. Our lab investigates how diverse autism-associated genes, many of which display striking pleiotropy, converge on shared biological pathways during development. We use Xenopus and human cell culture models to identify points of convergence among seemingly unrelated genetic risk factors, revealing common molecular mechanisms that contribute not only to autism but also to its comorbidities, including congenital heart disease and gastrointestinal dysfunction. Our approach integrates high-throughput functional genomics, live imaging, and in vivo perturbation to map the developmental trajectories altered by neurodevelopmental risk genes. By resolving the complexity introduced by genetic pleiotropy, we aim to define actionable entry points for therapeutic discovery. We're passionate about advancing both fundamental neuroscience and translational insight, while also mentoring the next generation of scientists. If you’re interested in collaboration, mentorship, or learning more about our work, feel free to connect.

Research Summary
We use Xenopus tropicalis, diploid frogs, as a high-throughput platform for genetic analysis to identify convergent biology underlying psychiatric disorder risk genes

Publications

Convergence of autism proteins at the cilium.

bioRxiv : the preprint server for biology

Kostyanovskaya E, Lasser MC, Wang B, Schmidt J, Bader E, Buteo C, Arbelaez J, Sindledecker AR, McCluskey KE, Castillo O, Wang S, Dea J, Helde KA, Michael Graglia J, Brimble E, Kastner DB, Ehrlich AT, State MW, Jeremy Willsey A, Willsey HR

A foundational atlas of autism protein interactions reveals molecular convergence.

bioRxiv : the preprint server for biology

Wang B, Vartak R, Zaltsman Y, Naing ZZC, Hennick KM, Polacco BJ, Bashir A, Eckhardt M, Bouhaddou M, Xu J, Sun N, Lasser MC, Zhou Y, McKetney J, Guiley KZ, Chan U, Kaye JA, Chadha N, Cakir M, Gordon M, Khare P, Drake S, Drury V, Burke DF, Gonzalez S, Alkhairy S, Thomas R, Lam S, Morris M, Bader E, Seyler M, Baum T, Krasnoff R, Wang S, Pham P, Arbalaez J, Pratt D, Chag S, Mahmood N, Rolland T, Bourgeron T, Finkbeiner S, Swaney DL, Bandyopadhay S, Ideker T, Beltrao P, Willsey HR, Obernier K, Nowakowski TJ, Hüttenhain R, State MW, Willsey AJ, Krogan NJ

DYRK1A-related intellectual disability: a syndrome associated with congenital anomalies of the kidney and urinary tract.

Genetics in medicine : official journal of the American College of Medical Genetics

Blackburn ATM, Bekheirnia N, Uma VC, Corkins ME, Xu Y, Rosenfeld JA, Bainbridge MN, Yang Y, Liu P, Madan-Khetarpal S, Delgado MR, Hudgins L, Krantz I, Rodriguez-Buritica D, Wheeler PG, Al-Gazali L, Mohamed Saeed Mohamed Al Shamsi A, Gomez-Ospina N, Chao HT, Mirzaa GM, Scheuerle AE, Kukolich MK, Scaglia F, Eng C, Willsey HR, Braun MC, Lamb DJ, Miller RK, Bekheirnia MR

The Psychiatric Cell Map Initiative: A Convergent Systems Biological Approach to Illuminating Key Molecular Pathways in Neuropsychiatric Disorders.

Cell

Willsey AJ, Morris MT, Wang S, Willsey HR, Sun N, Teerikorpi N, Baum TB, Cagney G, Bender KJ, Desai TA, Srivastava D, Davis GW, Doudna J, Chang E, Sohal V, Lowenstein DH, Li H, Agard D, Keiser MJ, Shoichet B, von Zastrow M, Mucke L, Finkbeiner S, Gan L, Sestan N, Ward ME, Huttenhain R, Nowakowski TJ, Bellen HJ, Frank LM, Khokha MK, Lifton RP, Kampmann M, Ideker T, State MW, Krogan NJ