CRISPR-Cas9 correction in the DMD mouse model is accompanied by upregulation of Dp71f protein

Autor: Egorova, Tatiana V., Polikarpova, Anna V., Vassilieva, Svetlana G., Dzhenkova, Marina A., Savchenko, Irina M., Velyaev, Oleg A., Shmidt, Anna A., Soldatov, Vladislav O., Pokrovskii, Mikhail V., Deykin, Alexey V., Bardina, Maryana V.

Publikováno v: In Molecular Therapy - Methods & Clinical Development 14 September 2023 30:161-180

RNA Interference Effectors Selectively Silence the Pathogenic Variant GNAO1 c.607 G > A In Vitro.

Autor: Klementieva, Natalia V., Lunev, Evgenii A., Shmidt, Anna A., Loseva, Elizaveta M., Savchenko, Irina M., Svetlova, Ekaterina A., Galkin, Ivan I., Polikarpova, Anna V., Usachev, Evgeny V., Vassilieva, Svetlana G., Marina, Valeria I., Dzhenkova, Marina A., Romanova, Anna D., Agutin, Anton V., Timakova, Anna A., Reshetov, Denis A., Egorova, Tatiana V., Bardina, Maryana V.

Publikováno v: Nucleic Acid Therapeutics; Apr2024, Vol. 34 Issue 2, p90-99, 10p

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K zobrazení výsledku je třeba se přihlásit.

CRISPR/Cas9-generated mouse model with humanizing single-base substitution in the Gnao1 for safety studies of RNA therapeutics.

Autor: Polikarpova AV; Laboratory of Modeling and Gene Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow, Russia.; Marlin Biotech, Sochi, Russia., Egorova TV; Laboratory of Modeling and Gene Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow, Russia.; Marlin Biotech, Sochi, Russia., Lunev EA; Laboratory of Modeling and Gene Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow, Russia.; Marlin Biotech, Sochi, Russia.; Center for Precision Genome Editing and Genetic Technologies for Biomedicine, Institute of Gene Biology, Russian Academy of Sciences, Moscow, Russia., Tsitrina AA; Koltzov Institute of Developmental Biology Russian Academy of Sciences, Moscow, Russia., Vassilieva SG; Laboratory of Modeling and Gene Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow, Russia.; Marlin Biotech, Sochi, Russia., Savchenko IM; Marlin Biotech, Sochi, Russia.; Center for Precision Genome Editing and Genetic Technologies for Biomedicine, Institute of Gene Biology, Russian Academy of Sciences, Moscow, Russia., Silaeva YY; Core Facility Center, Institute of Gene Biology Russian Academy of Sciences, Moscow, Russia., Deykin AV; Marlin Biotech, Sochi, Russia.; Core Facility Center, Institute of Gene Biology Russian Academy of Sciences, Moscow, Russia.; Laboratory of Genetic Technologies and Genome Editing for Biomedicine and Animal Health, Joint Center for Genetic Technologies, Belgorod National Research University, Belgorod, Russia., Bardina MV; Laboratory of Modeling and Gene Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow, Russia.; Marlin Biotech, Sochi, Russia.; Center for Precision Genome Editing and Genetic Technologies for Biomedicine, Institute of Gene Biology, Russian Academy of Sciences, Moscow, Russia.

Publikováno v: Frontiers in genome editing [Front Genome Ed] 2023 Apr 03; Vol. 5, pp. 1034720. Date of Electronic Publication: 2023 Apr 03 (Print Publication: 2023).