Nizar Y. Saad

Biochemistry, Genetics and Molecular Biology · Life Sciences

8h-index579citations25works0.52yr avg
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Research Topics

Muscle Physiology and Disorders(8), RNA Research and Splicing(6), RNA and protein synthesis mechanisms(5), Extracellular vesicles in disease(5), RNA modifications and cancer(5)

Publications25 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
REpair of heterozygous Mutations independent of Exogenous Donor template with high efficiency (REMEDY) using allele specific CRISPR targeting and HDR enhancers
2025Open Access
Meet the <i>Nucleic Acids Insights Editorial Board</i>: Nizar Y Saad
Nucleic Acid Insights·2025
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
Comparative analysis of nucleic acid delivery systems for gene therapy: assessing viral and non-viral approaches with emphasis on extracellular vesicles
Nucleic Acid Insights·2024· 1 citedOpen Access
P65 Development of therapeutic extracellular vesicle enveloped-AAV vectors for muscle gene therapy
Neuromuscular Disorders·2023· 2 cited
Post‐Translational Modifications of the <scp>DUX4</scp> Protein Impact Toxic Function in FSHD Cell Models
Annals of Neurology·2023· 4 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
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
P.51Identification of the DUX4-targeted miRNome from a library of 1,881 natural human miRNAs
Neuromuscular Disorders·2019
AAV-mediated follistatin gene therapy improves functional outcomes in the TIC-DUX4 mouse model of FSHD
JCI Insight·2018· 74 citedOpen Access
FSHD / OPMD / EDMD / DMI
Neuromuscular Disorders·2018
FSHD / OPMD / EDMD / DMI
Neuromuscular Disorders·2018
Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD
Molecular Therapy — Methods & Clinical Development·2017· 65 citedOpen Access
A ribonucleopeptide world at the origin of life
Journal of Systematics and Evolution·2017· 32 citedOpen Access
A <i>glyS</i> T-box riboswitch with species-specific structural features responding to both proteinogenic and nonproteinogenic tRNA<sup>Gly</sup> isoacceptors
RNA·2015· 21 citedOpen Access
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Frequent Co-authors

Scott Q. Harper(14), Lindsay M. Wallace(7), Jocelyn O. Eidahl(5), Afrooz Rashnonejad(5), Nettie K. Pyne(4), Mustafa Al-Kharsan(4), Sara E. Garwick-Coppens(4), Allison M. Fowler(3), Gholamhossein Amini Chermahini(3), Jacqueline S. Domire(3), Louise R. Rodino‐Klapac(3), H. D. Becker(3), Carlee R. Giesige(2), Kristin N. Heller(2), S. Guckes(2), K. John McLaughlin(2), Zarife Sahenk(2), Mélanie Brayé(2), Denis Drainas(2), Constantinos Stathopoulos(2)