Author

Matthew G. Woll

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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Bioinformatics· 1 Genetics· 1 Neuroscience· 1

Signature Assays, Organisms & Methods

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

Frequent Co-Authors

Elisabetta MoriniiD· 3 shared Amal Dakka· 2 shared Anil K. ChekuriiD· 2 shared Aram J. KrausoniD· 2 shared Christopher R. TrottaiD· 2 shared Emily M. Logan· 2 shared Jana NarasimhaniD· 2 shared Marla L. WeetalliD· 2 shared

Activity by Year

2020-2025 · peak 1/yr

Co-Author Collaboration Network

10 top collaborators · 45 mutual co-author links

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3 shared papersNeuroscience

Elisabetta MoriniORCID

Mutual co-authors in cluster

Author profile →

3 papers

Recent·Most cited

MAPT Splicing Modulators as a Therapeutic Strategy for Tauopathies

M. Catarina Silva, Hannah Lindmeier, Paolo Pigini, Jennifer Laughlin, Yong Yu, Christie Morrill, Michael A. Arnold, Scott J. Barraza, Khalil Saadipour, Monal Dieterich, Angela Minnella, Kausiki Datta, Nanjing Zhang, Jana Narasimhan, Christopher R. Trotta, , Elisabetta Morini

Discovery of SMCs that correct MAPT splicing, reduce 4R tau, and rescue pathology in patient-derived neuronal and in vivo models of 4R tauopathies.

bioRxiv · May 6, 2025 · PreprintNeuroscience

Development of a novel oral treatment that rescues gait ataxia and retinal degeneration in a phenotypic mouse model of familial dysautonomia.

Elisabetta Morini, Anil K. Chekuri, Emily M. Logan, Jessica Bolduc, Emily G. Kirchner, Monica Salani, Aram J. Krauson, Jana Narasimhan, Vijayalakshmi Gabbeta, Shivani Grover, Amal Dakka, Anna Mollin, Stephen P. Jung, Xin Zhao, Nanjing Zhang, , Susan Slaugenhaupt

This paper develops an oral treatment, PTC258, that restores ELP1 splicing and improves symptoms in a mouse model of familial dysautonomia.

The American Journal of Human Genetics · Feb 20, 2023Peer-reviewedGenetics

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

Dadi Gao, Elisabetta Morini, Monica Salani, Aram J. Krauson, Ashok Ragavendran, Serkan Erdin, Emily M. Logan, Anil K. Chekuri, Wencheng Li, Amal Dakka, Nikolai A. Naryshkin, Christopher R. Trotta, Kerstin A. Effenberger, Matthew G. Woll, Vijayalakshmi Gabbeta, , Susan A. Slaugenhaupt

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

bioRxiv · Feb 3, 2020 · PreprintBioinformatics