The paper develops a modular platform for synthesizing bio-orthogonal macromolecular conjugates (BMCs) to enable targeted delivery of macromolecular therapeutics with improved efficiency and specificity.

Bioconjugate Chemistry · Nov 10, 2025Peer-reviewedBiochemistry

Efficient Cas9 nuclease-based editing in skeletal muscle via lipid nanoparticle delivery

Sukanya Iyer, Katelyn Daman, Yehui Sun, Amanda A. Tutto, Sarah E. Holbrook, Anya T. Joynt, Jing Yan, Prajakta Prasad Ambegaokar, Dongsheng Guo, Pengpeng Liu, Jennifer E. Stauffer, Stacy A. Maitland, Sang M. Lee, Thomas L. Gallagher, Gregory A. Cox, , Scot A. Wolfe

SORT LNPs enable efficient Cas9-mediated repair of a pathogenic allele in skeletal muscle in a mouse model of LGMDR7.

bioRxiv · May 12, 2025 · PreprintMolecular Biology

Gene editing without ex vivo culture evades genotoxicity in human hematopoietic stem cells

Jing Zeng, My Anh Nguyen, Pengpeng Liu, Lucas Ferreira da Silva, Sébastien Levesque, Linda Yingqi Lin, David G. Justus, Karl Petri, Kendell Clement, Shaina N. Porter, Archana Verma, Nola R. Neri, Tolulope O. Rosanwo, Marioara-Felicia Ciuculescu, Daniela Abriss, , Daniel E. Bauer

This study shows that gene editing of HSCs without ex vivo culture reduces genotoxicity while maintaining therapeutic efficacy for beta-hemoglobinopathy treatment.

Cell stem cell · Dec 12, 2024Peer-reviewedGenetics

PRC1.6 localizes on chromatin with the human silencing hub (HUSH) complex for promoter-specific silencing

Tomás Rodríguez, Leonid Yurkovetskiy, Karthika Nagalekshmi, Oscar Lam, Eva Jazbec, Stacy A. Maitland, Scot A. Wolfe, Erik J. Sontheimer, Jeremy Luban

The paper identifies PRC1.6 as a partner of the HUSH complex in promoter-specific provirus silencing through chromatin colocalization and functional assays.

bioRxiv · Jul 12, 2024 · PreprintMicrobiology

Human genetic diversity modifies therapeutic gene editing off-target potential

Samuele Cancellieri, Jing Zeng, Linda Yingqi Lin, Manuel Tognon, My Anh Nguyen, Jiecong Lin, Nicola Bombieri, Stacy A. Maitland, Marioara-Felicia Ciuculescu, Varun Katta, Shengdar Q. Tsai, Myriam A. Armant, Scot A. Wolfe, Rosalba Giugno, Daniel E. Bauer, Luca Pinello

The paper develops CRISPRme, a tool that integrates genetic diversity data to assess off-target risks of gene editing, finding allele-specific off-target effects in clinical gRNAs.

Nature Genetics · Dec 15, 2022Peer-reviewedGenetics

Optimization of NLS Composition Improves CRISPR-Cas12a Editing Rates in Human Primary Cells

Kevin Luk, Pengpeng Liu, Jing Zeng, Yetao Wang, Stacy A. Maitland, Feston Idrizi, Karthikeyan Ponnienselvan, Lihua Julie Zhu, Jeremy Luban, Daniel E. Bauer, Scot A. Wolfe

This study optimizes the nuclear localization signal (NLS) of Cas12a to enhance editing efficiency in human primary cells without compromising specificity.

bioRxiv · Feb 1, 2022 · PreprintMolecular Biology

Precision Cas9 Genome Editing in vivo with All-in-one, Self-targeting AAV Vectors

Raed Ibraheim, Phillip W.L. Tai, Aamir Mir, Nida Javeed, Jiaming Wang, Tomás Rodríguez, Suk Namkung, Samantha J. Nelson, Eraj Shafiq Khokhar, Esther Mintzer, Stacy A. Maitland, Zexiang Chen, Yueying Cao, Emmanouela Tsagkaraki, Scot A. Wolfe, , Erik J. Sontheimer

This paper develops single-AAV vectors for precise in vivo genome editing using Nme2Cas9 with HDR templates or multiple sgRNAs, showing efficacy in treating HT-I and MPS-I mouse models.

Nature Communications · Nov 1, 2021Peer-reviewedMolecular Biology