Scott Q. Harper

Biochemistry, Genetics and Molecular Biology · Life Sciences

31h-index5.8kcitations100works5.12yr avg
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Research Topics

Muscle Physiology and Disorders(56), RNA Research and Splicing(27), Virus-based gene therapy research(19), RNA Interference and Gene Delivery(15), Neurogenetic and Muscular Disorders Research(15)

Publications100 total

Safety, efficacy, and distal nerve Schwann cell biodistribution in mice and NHPs to support translation of AAV9 RNAi therapy for CMT1A
Molecular Therapy — Nucleic Acids·2026Open Access
Synaptic dysregulation in a mouse model of GRIN2D developmental and epileptic encephalopathy
Brain·2025· 4 cited
Sustained efficacy of CRISPR-Cas13b gene therapy for FSHD is challenged by immune response to Cas13b
2025· 1 citedOpen Access
Identification of disease-specific extracellular vesicle-associated plasma protein biomarkers for Duchenne Muscular Dystrophy and Facioscapulohumeral Muscular Dystrophy
2024· 2 citedOpen Access
294P Identification of disease-specific extracellular vesicle-associated plasma biomarkers for Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD)
Neuromuscular Disorders·2024
Effective knockdown-replace gene therapy in a novel mouse model of DNM1 developmental and epileptic encephalopathy
Molecular Therapy·2024· 14 citedOpen Access
A self-complementary AAV proviral plasmid that reduces cross-packaging and ITR promoter activity in AAV vector preparations
Molecular Therapy — Methods & Clinical Development·2024· 14 citedOpen Access
P308 Development of a new DUX4-responsive reporter mouse
Neuromuscular Disorders·2023
VP289 Allele specific knockdown for LGMDD1
Neuromuscular Disorders·2023
Single-cell and spatial transcriptomics identify a macrophage population associated with skeletal muscle fibrosis
Science Advances·2023· 111 citedOpen Access
Post‐Translational Modifications of the <scp>DUX4</scp> Protein Impact Toxic Function in FSHD Cell Models
Annals of Neurology·2023· 4 citedOpen Access
Single-cell and spatial transcriptomics identify a macrophage population associated with skeletal muscle fibrosis
2023· 9 citedOpen Access
P.143 Investigation of human bone marrow mesenchymal stem cell-derived extracellular vesicles as therapeutic agents for Facioscapulohumeral muscular dystrophy
Neuromuscular Disorders·2022
Posttranslational modifications of the DUX4 protein impact toxic function
2022Open Access
The <scp>DUX4</scp> protein is a co‐repressor of the progesterone and glucocorticoid nuclear receptors
FEBS Letters·2022· 8 cited
The DUX4 protein is a co-repressor of the progesterone and glucocorticoid nuclear receptors
Academic Production·2022
A translatable RNAi-driven gene therapy silences PMP22/Pmp22 genes and improves neuropathy in CMT1A mice
Journal of Clinical Investigation·2022· 45 citedOpen Access
Meeting report: the 2021 FSHD International Research Congress
Skeletal Muscle·2022· 24 citedOpen Access
Human miRNA miR-675 inhibits DUX4 expression and may be exploited as a potential treatment for Facioscapulohumeral muscular dystrophy
Nature Communications·2021· 50 citedOpen Access
A stromal progenitor and ILC2 niche promotes muscle eosinophilia and fibrosis-associated gene expression
Cell Reports·2021· 46 citedOpen Access
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Frequent Co-authors

Lindsay M. Wallace(15), Beverly L. Davidson(9), Sara E. Garwick-Coppens(6), Allison M. Fowler(6), Jeffrey S. Chamberlain(5), Jacqueline S. Domire(5), Jocelyn O. Eidahl(5), Nettie K. Pyne(5), Alexandra Belayew(4), S. Guckes(4), Carlee R. Giesige(4), Nizar Y. Saad(4), Patrick D. Staber(3), Henry L. Paulson(3), Dongsheng Duan(3), Frédérique Coppée(3), Jian Liu(3), Gérald Coulis(3), Jenna M. Kastenschmidt(3), Philip K. Farahat(3)