Author

Amal Dakka

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 Molecular Biology· 1

Signature Assays, Organisms & Methods

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

Frequent Co-Authors

Anil K. ChekuriiD· 2 shared Aram J. KrausoniD· 2 shared Elisabetta MoriniiD· 2 shared Emily M. Logan· 2 shared Jana NarasimhaniD· 2 shared Marla L. WeetalliD· 2 shared Matthew G. Woll· 2 shared Monica Salani· 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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2 shared papersGenetics

Anil K. ChekuriORCID

Mutual co-authors in cluster

Author profile →

3 papers

Recent·Most cited

rAAV-Delivered Bicistronic Artificial microRNAs for Allele-Specific Silencing Improve Motor and Molecular Outcomes in Spinocerebellar Ataxia Type 3

Ana Carolina da Silva, Carina Henriques, Diana Duarte Lobo, Ana Rita Fernandes, Miguel Monteiro Lopes, Kevin Leandro, Dina Pereira, Sónia Duarte, Sara Lopes, Magda M. Santana, Amal Dakka, Steve De Marco, Marla L. Weetall, Jana Narasimhan, Anu Bhattacharyya, , Luís Pereira de Almeida

This paper develops and tests rAAV-delivered bicistronic artificial microRNAs to selectively silence mutant ATXN3 in SCA3 mouse models, showing improved motor function and reduced mutant protein aggregates.

bioRxiv · Oct 2, 2025 · PreprintMolecular Biology

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