Zobrazeno 1 - 8
of 8
pro vyhledávání: '"Svetlana G. Vassilieva"'
Autor:
Tatiana V. Egorova, Anna V. Polikarpova, Svetlana G. Vassilieva, Marina A. Dzhenkova, Irina M. Savchenko, Oleg A. Velyaev, Anna A. Shmidt, Vladislav O. Soldatov, Mikhail V. Pokrovskii, Alexey V. Deykin, Maryana V. Bardina
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 30, Iss , Pp 161-180 (2023)
Duchenne muscular dystrophy (DMD) is a severe hereditary disease caused by a deficiency in the dystrophin protein. The most frequent types of disease-causing mutations in the DMD gene are frameshift deletions of one or more exons. Precision genome ed
Externí odkaz:
https://doaj.org/article/86a0dbc1beb14091855dc8b5f24c41bb
Autor:
Anna V. Polikarpova, Tatiana V. Egorova, Evgenii A. Lunev, Alexandra A. Tsitrina, Svetlana G. Vassilieva, Irina M. Savchenko, Yuliya Y. Silaeva, Alexey V. Deykin, Maryana V. Bardina
Publikováno v:
Frontiers in Genome Editing, Vol 5 (2023)
The development of personalized medicine for genetic diseases requires preclinical testing in the appropriate animal models. GNAO1 encephalopathy is a severe neurodevelopmental disorder caused by heterozygous de novo mutations in the GNAO1 gene. GNAO
Externí odkaz:
https://doaj.org/article/23346d150ced45478ada50824f70075c
Autor:
Anna V. Starikova, Victoria V. Skopenkova, Anna V. Polikarpova, Denis A. Reshetov, Svetlana G. Vassilieva, Oleg A. Velyaev, Anna A. Shmidt, Irina M. Savchenko, Vladislav O. Soldatov, Tatiana V. Egorova, Maryana V. Bardina
Publikováno v:
Scientific Reports, Vol 12, Iss 1, Pp 1-13 (2022)
Abstract High expectations have been set on gene therapy with an AAV-delivered shortened version of dystrophin (µDys) for Duchenne muscular dystrophy (DMD), with several drug candidates currently undergoing clinical trials. Safety concerns with this
Externí odkaz:
https://doaj.org/article/e55381cec5004eccbc9982cbd02611bd
Autor:
Tatiana V. Egorova, Ivan I. Galkin, Oleg A. Velyaev, Svetlana G. Vassilieva, Irina M. Savchenko, Vyacheslav A. Loginov, Marina A. Dzhenkova, Diana S. Korshunova, Olga S. Kozlova, Dmitry N. Ivankov, Anna V. Polikarpova
Publikováno v:
International Journal of Molecular Sciences, Vol 24, Iss 11, p 9117 (2023)
Mutations that prevent the production of proteins in the DMD gene cause Duchenne muscular dystrophy. Most frequently, these are deletions leading to reading-frame shift. The “reading-frame rule” states that deletions that preserve ORF result in a
Externí odkaz:
https://doaj.org/article/3c824123831c446a8fd35bb5fc2799af
Autor:
Tatiana V. Egorova, Evgenia D. Zotova, Denis A. Reshetov, Anna V. Polikarpova, Svetlana G. Vassilieva, Dmitry V. Vlodavets, Alexey A. Gavrilov, Sergey V. Ulianov, Vladimir L. Buchman, Alexei V. Deykin
Publikováno v:
Disease Models & Mechanisms, Vol 12, Iss 4 (2019)
Exon skipping is a promising strategy for Duchenne muscular dystrophy (DMD) disease-modifying therapy. To make this approach safe, ensuring that excluding one or more exons will restore the reading frame and that the resulting protein will retain cri
Externí odkaz:
https://doaj.org/article/316b1ecf089f4dba993bcc8630736791
Autor:
Svetlana G. Vassilieva, Tatiana V. Egorova, Alexandra A Tsitrina, Yuri Kotelevtsev, Kirill A. Danilov, Sergei N. Orlov, Ivan I. Galkin, A. Shmidt, A. V. Polikarpova, Anna V. Starikova
Publikováno v:
Experimental cell research. 392(2)
AAV-delivered microdystrophin genes hold great promise for Duchenne muscular dystrophy (DMD) treatment. It is anticipated that the optimization of engineered dystrophin genes will be required to increase the efficacy and reduce the immunogenicity of
Publikováno v:
BIOTECHNOLOGY: STATE OF THE ART AND PERSPECTIVES. :239-240
In the present work we validated neonatal intracerebroventricular injections for AAV-mediated transgene delivery into GNAO1-rich regions of the murine brain. This administration route can be used for testing efficacy of AAV- based gene therapy for GN
Autor:
Svetlana G. Vassilieva, Dmitry V. Vlodavets, A. V. Polikarpova, Alexey A. Gavrilov, Denis A. Reshetov, Sergey V. Ulianov, Vladimir L. Buchman, Evgenia D. Zotova, Alexei V. Deykin, Tatiana V. Egorova
Publikováno v:
Disease Models & Mechanisms, Vol 12, Iss 4 (2019)
Disease Models & Mechanisms
Disease Models & Mechanisms
Exon skipping is a promising strategy for Duchenne muscular dystrophy (DMD) disease-modifying therapy. To make this approach safe, ensuring that excluding one or more exons will restore the reading frame and that the resulting protein will retain cri