Author

Kerstin A. Effenberger

PTC Therapeutics (United States)

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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Biochemistry· 1 Bioinformatics· 1

Signature Assays, Organisms & Methods

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Research Focus:differential expression· 2 Assay:bulk RNA-seq· 1 Research Focus:cell type annotation· 1 Research Focus:cell-cell communication· 1 Approach:deep learning· 1 Approach:experimental assay· 1 Research Focus:gene regulatory network inference· 1 Organism:human· 1

Frequent Co-Authors

Christopher R. TrottaiD· 2 shared Amal Dakka· 1 shared Anil K. ChekuriiD· 1 shared Aram J. KrausoniD· 1 shared Ashok RagavendraniD· 1 shared Brian Kosmyna· 1 shared Dadi GaoiD· 1 shared Elisabetta MoriniiD· 1 shared

Activity by Year

2020-2026 · peak 1/yr

Co-Author Collaboration Network

10 top collaborators · 37 mutual co-author links

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2 shared papersBiochemistry

Christopher R. TrottaORCID

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2 papers

Recent·Most cited

BiochemistryNature CommunicationsPeer-reviewedJul 16, 2026

The U1 snRNP protein U1C and Helix H of U1 snRNA are critical for small molecule splicing modulator function

Zhiling Kuang, Brian Kosmyna, Xueni Li…·Rui Zhao

This study shows that U1C and Helix H of U1 snRNA are essential for branaplam's effect on weak 5 splice site recognition, while risdiplam requires additional factors for activity.

2 citations

spatial transcriptomicsexperimental assay

𝕏 PostBluesky

BioinformaticsbioRxivPreprintFeb 3, 2020

A deep learning approach to identify new gene targets of a novel therapeutic for human splicing disorders

Dadi Gao, Elisabetta Morini, Monica Salani…·Susan A. Slaugenhaupt

The paper uses deep learning to identify new gene targets for a splicing modulator drug, validating predictions with in vitro assays.

8 citations

humanbulk RNA-seq

𝕏 PostBluesky