Author

Scott Q. Harper

Nationwide Children's HospitalORCID 0000-0001-5135-4317Unclaimed ORCID profile · follow to get notified of new preprints

Biology Disciplines

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Bioengineering· 1 Molecular Biology· 1 Neuroscience· 1

Signature Assays, Organisms & Methods

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Research Focus:benchmarking· 2 Research Focus:drug response / drug discovery· 2 Approach:experimental assay· 2 Organism:human· 2 Research Focus:cell type annotation· 1 Assay:CRISPR screen· 1 Research Focus:differential expression· 1 Assay:imaging· 1

Frequent Co-Authors

Afrooz RashnonejadiD· 2 shared Nizar Y. SaadiD· 2 shared Allison M. Fowler· 1 shared Bilal Bayazit· 1 shared Chiranth K. NagarajiD· 1 shared Eduardo ReáteguiiD· 1 shared Gérald CoulisiD· 1 shared Gholamhossein Amini Chermahini· 1 shared

Activity by Year

2022-2026 · peak 1/yr

Co-Author Collaboration Network

10 top collaborators · 22 mutual co-author links

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2 shared papersMolecular Biology

Afrooz RashnonejadORCID

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3 papers

Recent·Most cited

BioengineeringbioRxivPreprintMay 29, 2026

Single Particle Adsorption and Response Quantification for Functional AAVx Titer and Dose Analysis

Chiranth K. Nagaraj, Truc Kim Nugyen, Kwang Joo Kwak…·Eduardo Reátegui

This paper introduces SPARQ, a method to quantify functional AAV vectors at single-particle resolution, revealing functional heterogeneity and improving dose analysis in gene therapy.

DOI 10.64898/2026.05.28.728583

imagingmicroscopy & imaging analysis

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Molecular BiologybioRxivPreprintDec 18, 2024

Sustained efficacy of CRISPR-Cas13b gene therapy for FSHD is challenged by immune response to Cas13b

Afrooz Rashnonejad, Manal Farea, Gholamhossein Amini Chermahini…·Scott Q. Harper

This study shows CRISPR-Cas13b effectively reduces DUX4 in FSHD but is limited by immune responses that decrease Cas13b levels over time.

4 citations

mousehuman

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NeurosciencebioRxivPreprintJul 22, 2022

Posttranslational modifications of the DUX4 protein impact toxic function

Renatta N. Knox, Jocelyn O. Eidahl, Lindsay M. Wallace…·Scott Q. Harper

This study identifies posttranslational modifications of DUX4 that regulate its toxic function in FSHD and suggests potential therapeutic targets.

DOI 10.1101/2022.07.22.501148

humanexperimental assay

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