Personal information

Biography

Dr. Rashnonejad’s core expertise is in developing AAV-based gene therapies for rare neurodegenerative and neuromuscular diseases. During her Ph.D., she investigated intra-uterine (aka prenatal or fetal) AAV gene therapy for Spinal Muscular Atrophy (SMA). During her postdoc, she developed CRISPR-Cas13-mediated DUX4-silencing and U7snRNA-mediated exon-skipping approaches for treating Facioscapulohumeral muscular dystrophy (FSHD) as well as characterizing a new FSHD mouse model.

In her own translational lab, Dr. Rashnonejad continues developing novel AAV-based gene therapies for rare pediatric disorders with the goal of translating these research programs into the clinic. AAV vectors appear to be the safest and most effective delivery vehicles; however, they do not come with an innate tropism toward a specific tissue/cell type. Targeting the peripheral nerve system (PNS) is still challenging using AAV vectors and is the most significant barrier to developing gene therapy for peripheral neuropathies. Her team is working to address this need by designing next-generation AAV vectors to achieve tissue- and cell-specific treatments for PNS disorders. Her current research focus has also included evaluating novel gene therapies for Charcot-Marie-Tooth type 1B (CMT1B), mitochondrial DNA depletion syndrome, and Nemaline Myopathy. Rashnonejad lab is also interested in investigating in-utero gene therapies for severe inherited diseases that start prenatally. The team employs cutting-edge technologies such as RNAi and artificial miRNA, and CRISPR approaches, advanced molecular methods, DNA/RNA sequencing, and testing therapies in animal models.

Activities

Works (19)

Posttranslational modifications of the DUX4 protein impact toxic function

biorxiv.
2022-07-22 | Preprint
Contributors: Renatta N. Knox; Jocelyn O. Eidahl; Lindsay Wallace; Sarah Choudury; Afrooz Rashnonejad; Nizar Y. Saad; Michael E. Hoover; Liwen Zhang; Owen E. Branson; Michael A. Freitas et al.
Source: Self-asserted source
Afrooz RASHNONEJAD

Designed U7 snRNAs inhibit DUX4 expression and improve FSHD-associated outcomes in DUX4 overexpressing cells and FSHD patient myotubes

Molecular Therapy - Nucleic Acids
2021 | Journal article
EID:

2-s2.0-85099137967

Part of ISSN: 21622531
Contributors: Rashnonejad, A.; Amini-Chermahini, G.; Taylor, N.K.; Wein, N.; Harper, S.Q.
Source: Self-asserted source
Afrooz RASHNONEJAD via Scopus - Elsevier

DUX4 RNA SILENCING USING RNA TARGETING CRISPR-CAS13B

WIPO IP Portal patentscope
2020-09-07 | Patent
Source: Self-asserted source
Afrooz RASHNONEJAD

Fetal Gene Therapy Using a Single Injection of Recombinant AAV9 Rescued SMA Phenotype in Mice

Molecular Therapy
2019 | Journal article
EID:

2-s2.0-85072269426

Part of ISSN: 15250024 15250016
Contributors: Rashnonejad, A.; Amini Chermahini, G.; Gündüz, C.; Onay, H.; Aykut, A.; Durmaz, B.; Baka, M.; Su, Q.; Gao, G.; Özkınay, F.
Source: Self-asserted source
Afrooz RASHNONEJAD via Scopus - Elsevier

RNAscope in situ hybridization-based method for detecting DUX4 RNA expression in vitro

RNA
2019 | Journal article
EID:

2-s2.0-85071706040

Part of ISSN: 14699001 13558382
Contributors: Chermahini, G.A.; Rashnonejad, A.; Harper, S.Q.
Source: Self-asserted source
Afrooz RASHNONEJAD via Scopus - Elsevier

O.8DUX4 mRNA silencing with CRISPR-Cas13 gene therapy as a prospective treatment for Facioscapulohumeral muscular dystrophy

Neuromuscular Disorders
2019-10 | Conference abstract
Part of ISSN: 0960-8966
Source: Self-asserted source
Afrooz RASHNONEJAD

P.50An in situ hybridization-based method for detecting DUX4 RNA expression in vitro

Neuromuscular Disorders
2019-10 | Conference paper
Part of ISSN: 0960-8966
Source: Self-asserted source
Afrooz RASHNONEJAD

AAV-mediated follistatin gene therapy improves functional outcomes in the TIC-DUX4 mouse model of FSHD

JCI insight
2018 | Journal article
EID:

2-s2.0-85063241811

Part of ISSN: 23793708
Contributors: Giesige, C.R.; Wallace, L.M.; Heller, K.N.; Eidahl, J.O.; Saad, N.Y.; Fowler, A.M.; Pyne, N.K.; Al-Kharsan, M.; Rashnonejad, A.; Chermahini, G.A. et al.
Source: Self-asserted source
Afrooz RASHNONEJAD via Scopus - Elsevier

Comparative analysis of human UCB and adipose tissue derived mesenchymal stem cells for their differentiation potential into brown and white adipocytes

Molecular Biology Reports
2018 | Journal article
EID:

2-s2.0-85042105117

Part of ISSN: 15734978 03014851
Contributors: Rashnonejad, A.; Ercan, G.; Gunduz, C.; Akdemir, A.; Tiftikcioglu, Y.O.
Source: Self-asserted source
Afrooz RASHNONEJAD via Scopus - Elsevier

The Effect of Pre-Postnatal Nicotine Exposure on Types and Levels of Plasma Sialic Acids in Rats

Proceedings
2018-12-06 | Journal article
Part of ISSN: 2504-3900
Source: Self-asserted source
Afrooz RASHNONEJAD

FSHD / gene therapy / trnagenic mouse model / AAV

Neuromuscular Disorders
2018-10 | Conference abstract
Part of ISSN: 0960-8966
Source: Self-asserted source
Afrooz RASHNONEJAD

In vitro gene manipulation of spinal muscular atrophy fibroblast cell line using gene-targeting fragment for restoration of SMN protein expression

Gene Therapy
2016 | Journal article
EID:

2-s2.0-84955203170

Contributors: Rashnonejad, A.; Gündüz, C.; Süslüer, S.Y.; Onay, H.; Durmaz, B.; Bandehpour, M.; Özkinay, F.
Source: Self-asserted source
Afrooz RASHNONEJAD via Scopus - Elsevier

Large-Scale Production of Adeno-Associated Viral Vector Serotype-9 Carrying the Human Survival Motor Neuron Gene

Molecular Biotechnology
2016 | Journal article
EID:

2-s2.0-84954376750

Contributors: Rashnonejad, A.; Chermahini, G.A.; Li, S.; Ozkinay, F.; Gao, G.
Source: Self-asserted source
Afrooz RASHNONEJAD via Scopus - Elsevier

Molecular genetic analysis of survival motor neuron gene in 460 Turkish cases with suspicious spinal muscular atrophy disease

Iranian Journal of Child Neurology
2016 | Journal article
EID:

2-s2.0-84990041214

Contributors: Rashnonejad, A.; Onay, H.; Atik, T.; Atan Sahin, O.; Gokben, S.; Tekgul, H.; Ozkinay, F.
Source: Self-asserted source
Afrooz RASHNONEJAD via Scopus - Elsevier

Hematologic Malignancies in Children with Down Syndrome: Transient Myeloproliferative Disease and Acute Megakaryoblastic Leukemia

The Journal of Pediatric Research
2015-03-05 | Journal article
Part of ISSN: 2147-9445
Source: Self-asserted source
Afrooz RASHNONEJAD

Principles of Gene Therapy and Recent Advance (Gen tedavisinin temel ilkeleri ve son gelişmeler)

Ege Tıp Dergisi
2014-12-01 | Journal article
Part of ISSN: 1016-9113
Source: Self-asserted source
Afrooz RASHNONEJAD

Genetically Modified Stem Cells as a New Strategy for Treatment of Diseases Afrooz RASHNONEJAD

Unpublished
2013 | Conference abstract
Source: Self-asserted source
Afrooz RASHNONEJAD

Pyocyanine biosynthetic genes in clinical and environmental isolates of Pseudomonas aeruginosa and detection of pyocyanine's antimicrobial effects with or without colloidal silver nanoparticles

Cell Journal
2012 | Journal article
EID:

2-s2.0-84862537553

Contributors: Nowroozi, J.; Akhavan Sepahi, A.; Rashnonejad, A.
Source: Self-asserted source
Afrooz RASHNONEJAD via Scopus - Elsevier

Pyocyanine Biosynthetic Genes in Clinical and Environmental Isolates of Pseudomonas aeruginosa and Detection of Pyocyanine\textquoterights Antimicrobial Effects with or without Colloidal Silver Nanoparticles

2012 | Journal article
URI:

https://doaj.org/article/94d79d5198944098bb558756b0d2fe50

Source: Self-asserted source
Afrooz RASHNONEJAD

Peer review (3 reviews for 3 publications/grants)

Review activity for Cellular and molecular life sciences (1)
Review activity for JoVE. (1)
Review activity for Molecular biotechnology. (1)