Cargando…

Functionality of a bicistronic construction containing HEXA and HEXB genes encoding β-hexosaminidase A for cell-mediated therapy of GM2 gangliosidoses

Tay-Sachs disease and Sandhoff disease are severe hereditary neurodegenerative disorders caused by a deficiency of β-hexosaminidase A (HexA) enzyme, which results in the accumulation of GM2 gangliosides in the nervous system cells. In this work, we analyzed the efficacy and safety of cell-mediated g...

Descripción completa

Detalles Bibliográficos
Autores principales:	Shaimardanova, Alisa A., Chulpanova, Daria S., Solovyeva, Valeriya V., Aimaletdinov, Aleksandr M., Rizvanov, Albert A.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Wolters Kluwer - Medknow 2021
Materias:	Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8451576/ https://www.ncbi.nlm.nih.gov/pubmed/34100447 http://dx.doi.org/10.4103/1673-5374.314310

Ejemplares similares

Increasing β-hexosaminidase A activity using genetically modified mesenchymal stem cells
por: Shaimardanova, Alisa A., et al.
Publicado: (2023)

Identification of a novel HEXB Mutation in an Iranian Family with suspected patient to GM2‐gangliosidoses
por: Mansouri‐Movahed, Fatemeh, et al.
Publicado: (2020)

Serum Cytokine Profile, Beta-Hexosaminidase A Enzymatic Activity and GM(2) Ganglioside Levels in the Plasma of a Tay-Sachs Disease Patient after Cord Blood Cell Transplantation and Curcumin Administration: A Case Report
por: Shaimardanova, Alisa A., et al.
Publicado: (2021)

Current Strategies for the Gene Therapy of Autosomal Recessive Congenital Ichthyosis and Other Types of Inherited Ichthyosis
por: Chulpanova, Daria S., et al.
Publicado: (2022)

Combined replacement effects of human modified β-hexosaminidase B and GM2 activator protein on GM2 gangliosidoses fibroblasts
por: Kitakaze, Keisuke, et al.
Publicado: (2016)

Production and Application of Multicistronic Constructs for Various Human Disease Therapies
por: Shaimardanova, Alisa A., et al.
Publicado: (2019)

New Approaches to Tay-Sachs Disease Therapy
por: Solovyeva, Valeriya V., et al.
Publicado: (2018)

Extracellular vesicles in the diagnosis and treatment of central nervous system diseases
por: Shaimardanova, Alisa A., et al.
Publicado: (2019)

Safety and Efficacy of Intravenous and Intrathecal Delivery of AAV9-Mediated ARSA in Minipigs
por: Mullagulova, Aysilu, et al.
Publicado: (2023)

Metachromatic Leukodystrophy: Diagnosis, Modeling, and Treatment Approaches
por: Shaimardanova, Alisa A., et al.
Publicado: (2020)

Gene and Cell Therapy for Epilepsy: A Mini Review
por: Shaimardanova, Alisa A., et al.
Publicado: (2022)

Cytochalasin B-Induced Membrane Vesicles from Human Mesenchymal Stem Cells Overexpressing IL2 Are Able to Stimulate CD8(+) T-Killers to Kill Human Triple Negative Breast Cancer Cells
por: Chulpanova, Daria S., et al.
Publicado: (2021)

The Role of Cancer Stem Cells and Their Extracellular Vesicles in the Modulation of the Antitumor Immunity
por: Chulpanova, Daria S., et al.
Publicado: (2022)

Gene Therapy of Sphingolipid Metabolic Disorders
por: Shaimardanova, Alisa A., et al.
Publicado: (2023)

Various AAV Serotypes and Their Applications in Gene Therapy: An Overview
por: Issa, Shaza S., et al.
Publicado: (2023)

Thymic Alterations in GM2 Gangliosidoses Model Mice
por: Kanzaki, Seiichi, et al.
Publicado: (2010)

The GM2 gangliosidoses: Unlocking the mysteries of pathogenesis and treatment
por: Toro, Camilo, et al.
Publicado: (2021)

The diagnostic journey for patients with late-onset GM2 Gangliosidoses
por: Lopshire, Mariah C., et al.
Publicado: (2023)

Contribution of Tumor-Derived Extracellular Vesicles to Malignant Transformation of Normal Cells
por: Chulpanova, Daria S., et al.
Publicado: (2022)

Mesenchymal Stem Cell-Based Therapy for Lysosomal Storage Diseases and Other Neurodegenerative Disorders
por: Issa, Shaza S., et al.
Publicado: (2022)

GM2 Gangliosidosis in Shiba Inu Dogs with an In‐Frame Deletion in HEXB
por: Kolicheski, A., et al.
Publicado: (2017)

GM2 Gangliosidoses: Clinical Features, Pathophysiological Aspects, and Current Therapies
por: Leal, Andrés Felipe, et al.
Publicado: (2020)

Microglia-Specific Expression of HEXA and HEXB Leads to Poor Prognosis in Glioblastoma Patients
por: Jia, Mengxian, et al.
Publicado: (2021)

Therapeutic Prospects of Extracellular Vesicles in Cancer Treatment
por: Chulpanova, Daria S., et al.
Publicado: (2018)

Mouse Tumor Models for Advanced Cancer Immunotherapy
por: Chulpanova, Daria S., et al.
Publicado: (2020)

Emerging Gene Therapy Approaches in the Management of Spinal Muscular Atrophy (SMA): An Overview of Clinical Trials and Patent Landscape
por: Ponomarev, Aleksei S., et al.
Publicado: (2023)

Molecular Aspects and Future Perspectives of Cytokine-Based Anti-cancer Immunotherapy
por: Chulpanova, Daria S., et al.
Publicado: (2020)

Comparative Analysis of Natural and Cytochalasin B-Induced Membrane Vesicles from Tumor Cells and Mesenchymal Stem Cells
por: Gilazieva, Zarema, et al.
Publicado: (2022)

Natural history study of glycan accumulation in large animal models of GM2 gangliosidoses
por: Cavender, Catlyn, et al.
Publicado: (2020)

L-Arginine Ameliorates Defective Autophagy in GM2 Gangliosidoses by mTOR Modulation
por: Castejón-Vega, Beatriz, et al.
Publicado: (2021)

Identification of mutations in HEXA and HEXB in Sandhoff and Tay-Sachs diseases: a new large deletion caused by Alu elements in HEXA
por: Dastsooz, Hassan, et al.
Publicado: (2018)

Application of Mesenchymal Stem Cells for Therapeutic Agent Delivery in Anti-tumor Treatment
por: Chulpanova, Daria S., et al.
Publicado: (2018)

Analysis of the Interaction of Human Neuroblastoma Cell-Derived Cytochalasin B Induced Membrane Vesicles with Mesenchymal Stem Cells Using Imaging Flow Cytometry
por: Solovyeva, Valeriya V., et al.
Publicado: (2022)

In situ detection of GM1 and GM2 gangliosides using immunohistochemical and immunofluorescent techniques for auxiliary diagnosis of canine and feline gangliosidoses
por: Kohyama, Moeko, et al.
Publicado: (2016)

Recombinant Viruses for Cancer Therapy
por: Chulpanova, Daria S., et al.
Publicado: (2018)

Chitotriosidase as a biomarker for gangliosidoses
por: Kim, Sarah, et al.
Publicado: (2021)

Construction of a hybrid β-hexosaminidase subunit capable of forming stable homodimers that hydrolyze GM2 ganglioside in vivo
por: Tropak, Michael B, et al.
Publicado: (2016)

Canine GM2‐Gangliosidosis Sandhoff Disease Associated with a 3‐Base Pair Deletion in the HEXB Gene
por: Wang, P., et al.
Publicado: (2017)

Plasma neurofilament light, glial fibrillary acidic protein and lysosphingolipid biomarkers for pharmacodynamics and disease monitoring of GM2 and GM1 gangliosidoses patients
por: Welford, Richard W.D., et al.
Publicado: (2022)

Cytochalasin B-Induced Membrane Vesicles from TRAIL-Overexpressing Mesenchymal Stem Cells Induce Extrinsic Pathway of Apoptosis in Breast Cancer Mouse Model
por: Chulpanova, Daria S., et al.
Publicado: (2023)

Cannot write session to /tmp/vufind_sessions/sess_mpdm71aovpb9rjgei4138ibak4