دورية أكاديمية
أعرض في EDS

RNA-Seq Analysis of Trans-Differentiated ARPE-19 Cells Transduced by AAV9-AIPL1 Vectors.

التفاصيل البيبلوغرافية
العنوان: RNA-Seq Analysis of Trans-Differentiated ARPE-19 Cells Transduced by AAV9-AIPL1 Vectors.
المؤلفون: Galieva A; Gene Therapy Department, Science Center for Translational Medicine, Sirius University of Science and Technology, 354340 Sirius, Russia., Egorov A; Gene Therapy Department, Science Center for Translational Medicine, Sirius University of Science and Technology, 354340 Sirius, Russia., Malogolovkin A; Gene Therapy Department, Science Center for Translational Medicine, Sirius University of Science and Technology, 354340 Sirius, Russia.; Molecular Virology Laboratory, First Moscow State Medical University (Sechenov University), 119991 Moscow, Russia., Brovin A; Gene Therapy Department, Science Center for Translational Medicine, Sirius University of Science and Technology, 354340 Sirius, Russia., Karabelsky A; Gene Therapy Department, Science Center for Translational Medicine, Sirius University of Science and Technology, 354340 Sirius, Russia.
المصدر: International journal of molecular sciences [Int J Mol Sci] 2023 Dec 22; Vol. 25 (1). Date of Electronic Publication: 2023 Dec 22.
نوع المنشور: Journal Article
اللغة: English
بيانات الدورية: Publisher: MDPI Country of Publication: Switzerland NLM ID: 101092791 Publication Model: Electronic Cited Medium: Internet ISSN: 1422-0067 (Electronic) Linking ISSN: 14220067 NLM ISO Abbreviation: Int J Mol Sci Subsets: MEDLINE
أسماء مطبوعة: Original Publication: Basel, Switzerland : MDPI, [2000-
مواضيع طبية MeSH: Neurons* , Dependovirus*/genetics, Animals ; Humans ; RNA-Seq ; Cell Differentiation ; Sequence Analysis, RNA ; Antiviral Agents ; Adaptor Proteins, Signal Transducing
مستخلص: Inherited retinal disorders (IRD) have become a primary focus of gene therapy research since the success of adeno-associated virus-based therapeutics (voretigene neparvovec-rzyl) for Leber congenital amaurosis type 2 (LCA2). Dozens of monogenic IRDs could be potentially treated with a similar approach using an adeno-associated virus (AAV) to transfer a functional gene into the retina. Here, we present the results of the design, production, and in vitro testing of the AAV serotype 9 (AAV9) vector carrying the codon-optimized (co) copy of aryl hydrocarbon receptor-interacting protein like-1 ( AIPL1 ) as a possible treatment for LCA4. The pAAV-AIPL1co was able to successfully transduce retinal pigment epithelium cells (ARPE-19) and initiate the expression of human AIPL1 . Intriguingly, cells transduced with AAV9-AIPL1co showed much less antiviral response than AAV9-AIPL1wt (wild-type AIPL1 ) transduced. RNA-sequencing (RNA-seq) analysis of trans-differentiated ARPE-19 cells transduced with AAV9-AIPL1co demonstrated significant differences in the expression of genes involved in the innate immune response. In contrast, AAV9-AIPL1wt induced the prominent activation of multiple interferon-stimulated genes. The key part of the possible regulatory molecular mechanism is the activation of dsRNA-responsive antiviral oligoadenylate synthetases, and a significant increase in the level of histone coding genes' transcripts overrepresented in RNA-seq data (i.e., H1, H2A, H2B, H3, and H4). The RNA-seq data suggests that AAV9-AIPL1co exhibiting less immunogenicity than AAV9-AIPL1wt can be used for potency testing, using relevant animal models to develop future therapeutics for LCA4.
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معلومات مُعتمدة: 075-10-2021-093 Ministry of Science and Higher Education of the Russian Federation
فهرسة مساهمة: Keywords: AAV; AAV9; AIPL1; ARPE-19; LCA4; Leber congenital amaurosis; RNA-seq; gene therapy
المشرفين على المادة: 0 (Antiviral Agents)
0 (AIPL1 protein, human)
0 (Adaptor Proteins, Signal Transducing)
تواريخ الأحداث: Date Created: 20240111 Date Completed: 20240112 Latest Revision: 20240113
رمز التحديث: 20240113
مُعرف محوري في PubMed: PMC10778816
DOI: 10.3390/ijms25010197
PMID: 38203368
قاعدة البيانات: MEDLINE
الوصف
تدمد:1422-0067
DOI:10.3390/ijms25010197