Afrooz Rashnonejad
Biochemistry, Genetics and Molecular Biology · The Ohio State University
Publications
26
Citations
284
Est. group size
~1
Recurring co-author estimate
Active years
14
Publishing since 2012
Afrooz Rashnonejad's research focuses on developing gene and RNA-based therapies for inherited neuromuscular diseases, including facioscapulohumeral muscular dystrophy (FSHD), spinal muscular atrophy (SMA), Charcot-Marie-Tooth disease type 1B (CMT1B), and nemaline myopathy. Work spans CRISPR-Cas gene editing and silencing, viral (AAV)-delivered gene therapy, RNA sequencing to identify disease-causing mutations, and stem cell modeling using patient-derived induced pluripotent stem cells (iPSCs). This research aims to understand disease mechanisms at the molecular level and to test experimental therapies, primarily in cell and animal models.
Publication output has been variable over the last decade, with quiet years (e.g., 2017, 2021) interspersed with more active years, and a notable increase in output in 2025.
Generated by claude-sonnet-5 from public bibliographic data · Jul 20, 2026
- Generation of two induced pluripotent stem cell lines from Charcot-Marie-Tooth type 1B patients harboring autosomal dominant mutations in myelin protein zero gene
Stem Cell Research · 2025
- Sustained efficacy of CRISPR-Cas13b gene therapy for FSHD is challenged by immune response to Cas13b
bioRxiv (Cold Spring Harbor Laboratory) · 2025
- Generation of an induced pluripotent stem cell line (NCHi023-A) from a 41-year-old male with nemaline myopathy carrying autosomal dominant ACTA1 c.809-10C>A mutation
Stem Cell Research · 2025
- REpair of heterozygous Mutations independent of Exogenous Donor template with high efficiency (REMEDY) using allele specific CRISPR targeting and HDR enhancers
bioRxiv (Cold Spring Harbor Laboratory) · 2025
- Like father, like son: RNA-sequencing from a 30-year-old muscle biopsy identifies a novel splice variant in ACTA1 as the cause of an attenuated nemaline myopathy phenotype
Neuromuscular Disorders · 2025
- Navigating the Landscape of CMT1B: Understanding Genetic Pathways, Disease Models, and Potential Therapeutic Approaches
International Journal of Molecular Sciences · 2024
- 18O Breaking ground in CMT1B treatment: AAV9-mediated dual RNAi and gene replacement therapy targeting schwann cells improves myelination and peripheral nerve function in mice
Neuromuscular Disorders · 2024
- Post‐Translational Modifications of the <scp>DUX4</scp> Protein Impact Toxic Function in FSHD Cell Models
Annals of Neurology · 2023
- FP.29 AAV-CRISPR-Cas13 gene therapy for FSHD: DUX4 gene silencing efficacy and immune responses to Cas13b protein
Neuromuscular Disorders · 2022
- Posttranslational modifications of the DUX4 protein impact toxic function
bioRxiv (Cold Spring Harbor Laboratory) · 2022
- Designed U7 snRNAs inhibit DUX4 expression and improve FSHD-associated outcomes in DUX4 overexpressing cells and FSHD patient myotubes
Molecular Therapy — Nucleic Acids · 2020
- Fetal Gene Therapy Using a Single Injection of Recombinant AAV9 Rescued SMA Phenotype in Mice
Molecular Therapy · 2019
- RNAscope in situ hybridization-based method for detecting <i>DUX4</i> RNA expression in vitro
RNA · 2019
- O.8DUX4 mRNA silencing with CRISPR-Cas13 gene therapy as a prospective treatment for Facioscapulohumeral muscular dystrophy
Neuromuscular Disorders · 2019
- P.50An in situ hybridization-based method for detecting DUX4 RNA expression in vitro
Neuromuscular Disorders · 2019
- Neuromuscular Disorders×6
- bioRxiv (Cold Spring Harbor Laboratory)×3
- Stem Cell Research×2
- JCI Insight×1
- Molecular Therapy×1
- Kevin M. Flanigan
Biochemistry, Genetics and Molecular Biology · The Ohio State University
- Louise R. Rodino‐Klapac
Biochemistry, Genetics and Molecular Biology · The Ohio State University
- Scott Q. Harper
Biochemistry, Genetics and Molecular Biology · The Ohio State University
- K. Flanigan
Biochemistry, Genetics and Molecular Biology · The Ohio State University
- Tatyana A. Vetter
Biochemistry, Genetics and Molecular Biology · The Ohio State University
This profile was generated automatically from public scholarly data (OpenAlex). Group size and activity levels are estimates derived from co-authorship patterns.
Last updated Jul 19, 2026.
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