Author

Sunetra Sase

Children's Hospital of Philadelphia

Listed by name: these papers carry no ORCID for this author, so different people with the same name may be mixed together.

Biology Disciplines

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Neuroscience· 2 Genetics· 1 Molecular Biology· 1 Synthetic Biology· 1

Signature Assays, Organisms & Methods

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Research Focus:drug response / drug discovery· 4 Approach:experimental assay· 4 Organism:human· 3 Research Focus:differential expression· 2 Organism:mouse· 2 Assay:bulk RNA-seq· 1 Research Focus:cell type annotation· 1 Research Focus:cell-cell communication· 1

Frequent Co-Authors

Adeline L. VanderveriD· 5 shared Asako TakanohashiiD· 3 shared Julia Liao HackeriD· 3 shared Akshata Almad· 2 shared Anjali Bhagavatula· 2 shared Judith B. GrinspaniD· 2 shared Prabhat R. Napit· 2 shared Sarah WoidilliD· 2 shared

Activity by Year

2020-2026 · peak 3/yr

Co-Author Collaboration Network

10 top collaborators · 36 mutual co-author links

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5 shared papersNeuroscience

Adeline L. VanderverORCID

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Author profile →

5 papers

Recent·Most cited

A Mammalian High-Throughput Screen for AI-Designed Peptide-Guided Protein Degraders

Lin Zhao, Anuoluwapo A Mattix, Aastha Pal, Tong Chen, Sophia Vincoff, Lauren Hong, Diana Renteria, Sunetra Sase, Adeline L. Vanderver, Daniel Matson, Pranam Chatterjee

This paper presents a high-throughput platform for screening peptide-guided protein degraders in mammalian cells, demonstrating their ability to reduce target protein abundance and function.

bioRxiv · Aug 26, 2026 · PreprintSynthetic Biology

Deletion of TUBB4A mitigates the oligodendrocyte and neuronal deficits in human iPSCs derived from individuals affected by H-ABC

Zhongqi Hou, Rolf Dale Bates, Prabhat R. Napit, Luis Garcia, Anjali Bhagavatula, Julia Liao Hacker, Teshawn Johnson, Alyssa L. Gagne, Jean Ann Maguire, Asako Takanohashi, Deborah L. French, Akshata Almad, Judith B. Grinspan, Adeline L. Vanderver, Sunetra Sase

This study shows that deleting TUBB4A in human iPSCs reduces cell-autonomous deficits in oligodendrocytes and neurons associated with H-ABC, suggesting it could be a therapeutic strategy for TUBB4A-related leukodystrophy.

bioRxiv · Aug 6, 2026 · PreprintNeuroscience

Therapeutic suppression of Tubb4a rescues H-ABC leukodystrophy

Sunetra Sase, Julia Liao Hacker, Prabhat R. Napit, Anjali Bhagavatula, Sarah Woidill, Annemarie D’Alessandro, Marisa A. Jeffries, Akshata Almad, Asako Takanohashi, Quasar Saleem Padiath, Judith B. Grinspan, Eric D. Marsh, Adeline L. Vanderver

This study shows that suppressing Tubb4a with an ASO improves symptoms and extends lifespan in a mouse model of H-ABC leukodystrophy.

Molecular Therapy · Jan 20, 2026Peer-reviewedNeuroscience

Effective Gene Therapy for Metachromatic Leukodystrophy Achieved with Minimal Lentiviral Genomic Integrations

Lucas Tricoli, Sunetra Sase, Julia Liao Hacker, Vi Pham, Maxwell Chappell, Laura Breda, Stephanie N. Hurwitz, Naoto Tanaka, Carlo Castruccio Castracani, Amaliris Guerra, Zhongqi Hou, Lars Schlotawa, Karthikeyan Radhakrishnan, Matthew Hogenauer, Aoife M. Roche, , Stefano Rivella

This paper develops a novel lentiviral vector for gene therapy of MLD that achieves high ARSA activity with minimal genomic integrations, improving safety and efficacy in preclinical models.

Molecular Therapy — Nucleic Acids · Jan 25, 2025Peer-reviewedMolecular Biology

Type II Alexander disease caused by splicing errors and aberrant overexpression of an uncharacterized GFAP isoform.

Guy Helman, Asako Takanohashi, Tracy L. Hagemann, Ming Der Perng, Marzena Walkiewicz, Sarah Woidill, Sunetra Sase, Zachary Cross, Yangzhu Du, Ling Zhao, Amy Tara Waldman, Bret C. Haake, Ali S. Fatemi, Michael Brenner, Omar Sherbini, , Cas Simons

Human Mutation · Mar 3, 2020Peer-reviewedGenetics