Deep learning AAV promoters

Deep learning-guided design of cell type-specific AAV promoters

Sean K Wang*#, Boxiong Deng*, Surag Nair, Xiaobai Ren, Jiaying Li, Jenna Tijerina, Praveen Prakhar, Ziming Luo, Chelsea Nnebe, Samuel H Kim, Yimin Zhou, Sahil H Shah, Alexander Davis, Rohan Mahajan, Yilin Qiao, Yuxi Zhou, Jenny Zhang, Yunlu Xue, Jeffrey L Goldberg, Wei Wei, Anshul Kundaje, Howard Y Chang#, Sui Wang#

bioRxiv · 2026

RPE single-cell multiome

Single-cell multiome and enhancer connectome of human retinal pigment epithelium and choroid nominate pathogenic variants in age-related macular degeneration

Sean K Wang*, Jiaying Li*, Surag Nair, Reshma Kosaraju, Yun Chen, Yuanyuan Zhang, Anshul Kundaje, Yuwen Liu, Ningli Wang, Howard Y Chang

Cell Reports · 2026

Circular RNA engineering

Engineering circular RNA for enhanced protein production

Robert Chen*, Sean K Wang*, Julia A Belk, Laura Amaya, Zhijian Li, Angel Cardenas, Brian T Abe, Chun-Kan Chen, Paul A Wender, Howard Y Chang

Nature Biotechnology · 2023

Human retina single-cell multiome

Single-cell multiome of the human retina and deep learning nominate causal variants in complex eye diseases

Sean K Wang, Surag Nair, Rui Li, Katerina Kraft, Anusri Pampari, Aman Patel, Joyce B Kang, Christy Luong, Anshul Kundaje, Howard Y Chang

Cell Genomics · 2022

CD47-SIRPα cone protection

Augmentation of CD47-SIRPα signaling protects cones in genetic models of retinal degeneration

Sean K Wang, Yunlu Xue, Constance L Cepko

JCI Insight · 2021

SABER-FISH AAV detection

In situ detection of adeno-associated viral vector genomes with SABER-FISH

Sean K Wang, Sylvain W Lapan, Christin M Hong, Tyler B Krause, Constance L Cepko

Molecular Therapy – Methods & Clinical Development · 2020

Microglia TGF-β1 cone protection

Microglia modulation by TGF-β1 protects cones in mouse models of retinal degeneration

Sean K Wang, Yunlu Xue, Constance L Cepko

Journal of Clinical Investigation · 2020

CX3CL1 gene therapy retinitis pigmentosa

Soluble CX3CL1 gene therapy improves cone survival and function in mouse models of retinitis pigmentosa

Sean K Wang, Yunlu Xue, Parimal Rana, Christin M Hong, Constance L Cepko

Proceedings of the National Academy of Sciences · 2019

Multiomics and deep learning dissect regulatory syntax in human development

Betty B Liu, Selin Jessa, Samuel H Kim, Yan Ting Ng, Soon Il Higashino, Georgi K Marinov, Derek C Chen, Benjamin E Parks, Li Li, Tri C Nguyen, Austin T Wang, Sean K Wang, Meng How Tan, Serena Y Tan, Michael Kosicki, Len A Pennacchio, Eyal Ben-David, Anca M Pasca, Anshul Kundaje, Kyle KH Farh, William J Greenleaf

Nature · 2026

Genetic elements promote retention of extrachromosomal DNA in cancer cells

Venkat Sankar, King L Hung, Aditi Gnanasekar, Ivy Tsz-Lo Wong, Quanming Shi, Katerina Kraft, Matthew G Jones, Britney Jiayu He, Xiaowei Yan, Julia A Belk, Kevin J Liu, Sangya Agarwal, Sean K Wang, Anton G Henssen, Paul S Mischel, Howard Y Chang

Nature · 2026

Discrete Immolative Guanidinium Transporters deliver mRNA to specific organs and red blood cells

Zhijian Li, Aloysius Ee, Laura Amaya, Jennifer L Hamad, Pavan K Yadav, Sean K Wang, Howard Y Chang, Paul A Wender

Nature Communications · 2025

ERG responses to high-frequency flickers require FAT3 signaling in mouse retinal bipolar cells

Evelyn C Avilés, Sean K Wang, Sarina Patel, Sebastian Cordero, Shuxiang Shi, Lucas Lin, Vladimir J Kefalov, Lisa V Goodrich, Constance L Cepko, Yunlu Xue

Journal of General Physiology · 2025

Organ- and cell-selective delivery of mRNA in vivo using guanidinylated serinol charge-altering releasable transporters

Zhijian Li, Laura Amaya, Aloysius Ee, Sean K Wang, Alok Ranjan, Robert M Waymouth, Howard Y Chang, Paul A Wender

Journal of the American Chemical Society · 2024

Charge-altering releasable transporters enhance mRNA delivery in vitro and exhibit in vivo tropism

Zhijian Li, Laura Amaya, Ruoxi Pi, Sean K Wang, Alok Ranjan, Robert M Waymouth, Catherine A Blish, Howard Y Chang, Paul A Wender

Nature Communications · 2023

Chromophore supply modulates cone function and survival in retinitis pigmentosa mouse models

Yunlu Xue, Xiaomei Sun, Sean K Wang, Gayle B Collin, Vladimir J Kefalov, Constance L Cepko

Proceedings of the National Academy of Sciences · 2023

AAV-Txnip prolongs cone survival and vision in mouse models of retinitis pigmentosa

Yunlu Xue, Sean K Wang, Parimal Rana, Emma R West, Christin M Hong, Helian Feng, David M Wu, Constance L Cepko

eLife · 2021

Engineering adeno-associated viral vectors to evade innate immune and inflammatory responses

Ying Kai Chan, Sean K Wang, Colin J Chu, David A Copland, Alexander J Letizia, Helena Costa Verdera, Jessica J Chiang, Meher Sethi, May K Wang, William J Neidermyer Jr, Yingleong Chan, Elaine T Lim, Amanda R Graveline, Melinda Sanchez, Ryan F Boyd, Thomas S Vihtelic, Rolando Gian Carlo O Inciong, Jared M Slain, Priscilla J Alphonse, Yunlu Xue, Lindsey R Robinson-McCarthy, Jenny M Tam, Maha H Jabbar, Bhubanananda Sahu, Janelle F Adeniran, Manish Muhuri, Phillip WL Tai, Jun Xie, Tyler B Krause, Andyna Vernet, Matthew Pezone, Ru Xiao, Tina Liu, Wei Wang, Henry J Kaplan, Guangping Gao, Andrew D Dick, Federico Mingozzi, Maureen A McCall, Constance L Cepko, George M Church

Science Translational Medicine · 2021

Nrf2 overexpression rescues the RPE in mouse models of retinitis pigmentosa

David M Wu, Xuke Ji, Maryna V Ivanchenko, Michelle Chung, Mary Piper, Parimal Rana, Sean K Wang, Yunlu Xue, Emma West, Sophia R Zhao, Hongbin Xu, Marcelo Cicconet, Wenjun Xiong, Constance L Cepko

JCI Insight · 2021

Circumventing cellular immunity by miR142-mediated regulation sufficiently supports rAAV-delivered OVA expression without activating humoral immunity

Yuanyuan Xiao, Manish Muhuri, Shaoyong Li, Wanru Qin, Guangchao Xu, Li Luo, Jia Li, Alexander J Letizia, Sean K Wang, Ying Kai Chan, Chunmei Wang, Sebastian P Fuchs, Dan Wang, Qin Su, M Abu Nahid, George M Church, Michael Farzan, Li Yang, Yuquan Wei, Ronald C Desrosiers, Christian Mueller, Phillip WL Tai, Guangping Gao

JCI Insight · 2019

AAV cis-regulatory sequences are correlated with ocular toxicity

Wenjun Xiong, David M Wu, Yunlu Xue, Sean K Wang, Michelle J Chung, Xuke Ji, Parimal Rana, Sophia R Zhao, Shuyi Mai, Constance L Cepko

Proceedings of the National Academy of Sciences · 2019

Hoxb5 marks long-term haematopoietic stem cells and reveals a homogenous perivascular niche

James Y Chen, Masanori Miyanishi, Sean K Wang, Satoshi Yamazaki, Rahul Sinha, Kevin S Kao, Jun Seita, Debashis Sahoo, Hiromitsu Nakauchi, Irving L Weissman

Nature · 2016

For a full list of publications, see Google Scholar.