المؤلفون: Klementieva NV; Laboratory of Modeling and Gene Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow, Russia.; Marlin Biotech LLC, 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 LLC, Sochi, Russia.; Center for Precision Genome Editing and Genetic Technologies for Biomedicine, Institute of Gene Biology, Russian Academy of Sciences, Moscow, Russia., Shmidt AA; Laboratory of Modeling and Gene Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow, Russia.; Marlin Biotech LLC, Sochi, Russia.; Center for Precision Genome Editing and Genetic Technologies for Biomedicine, Institute of Gene Biology, Russian Academy of Sciences, Moscow, Russia., Loseva EM; Marlin Biotech LLC, Sochi, Russia., Savchenko IM; Marlin Biotech LLC, Sochi, Russia.; Center for Precision Genome Editing and Genetic Technologies for Biomedicine, Institute of Gene Biology, Russian Academy of Sciences, Moscow, Russia., Svetlova EA; Laboratory of Modeling and Gene Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow, Russia.; Marlin Biotech LLC, Sochi, Russia., Galkin II; Marlin Biotech LLC, Sochi, Russia.; Center for Precision Genome Editing and Genetic Technologies for Biomedicine, Institute of Gene Biology, Russian Academy of Sciences, Moscow, Russia., Polikarpova AV; Laboratory of Modeling and Gene Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow, Russia.; Marlin Biotech LLC, Sochi, Russia., Usachev EV; Laboratory of Translational Biomedicine, Gamaleya National Research Center for Epidemiology, 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 LLC, Sochi, Russia., Marina VI; Marlin Biotech LLC, Sochi, Russia., Dzhenkova MA; Laboratory of Modeling and Gene Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow, Russia.; Marlin Biotech LLC, Sochi, Russia., Romanova AD; Laboratory of Modeling and Gene Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow, Russia., Agutin AV; State Budgetary Healthcare Institution of Moscow Region 'Balashikha Hospital,' Balashikha, Russia., Timakova AA; Institute for Regenerative Medicine, Sechenov First Moscow State Medical University (Sechenov University), Moscow, Russia., Reshetov DA; Marlin Biotech LLC, 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 LLC, Sochi, Russia., Bardina MV; Laboratory of Modeling and Gene Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow, Russia.; Marlin Biotech LLC, Sochi, Russia.; Center for Precision Genome Editing and Genetic Technologies for Biomedicine, Institute of Gene Biology, Russian Academy of Sciences, Moscow, Russia.

المصدر: Nucleic acid therapeutics [Nucleic Acid Ther] 2024 Apr; Vol. 34 (2), pp. 90-99. Date of Electronic Publication: 2024 Jan 12.

نوع المنشور: Journal Article; Research Support, Non-U.S. Gov't

بيانات الدورية: Publisher: Mary Ann Liebert, Inc Country of Publication: United States NLM ID: 101562758 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2159-3345 (Electronic) Linking ISSN: 21593337 NLM ISO Abbreviation: Nucleic Acid Ther Subsets: MEDLINE

مواضيع طبية MeSH: Genetic Therapy* , Brain Diseases*/genetics, Animals ; Humans ; RNA Interference ; RNA, Small Interfering/pharmacology ; Alleles ; Genetic Vectors/genetics ; GTP-Binding Protein alpha Subunits, Gi-Go/genetics

المؤلفون: Egorova TV; Laboratory of Modeling and Therapy of Hereditary Diseases, Institute of Gene Biology, Russian Academy of Sciences, Moscow 119334, Russia.; Marlin Biotech LLC, Sochi 354340, Russia., Polikarpova AV; Laboratory of Modeling and Therapy of Hereditary Diseases, Institute of Gene Biology, Russian Academy of Sciences, Moscow 119334, Russia.; Marlin Biotech LLC, Sochi 354340, Russia., Vassilieva SG; Laboratory of Modeling and Therapy of Hereditary Diseases, Institute of Gene Biology, Russian Academy of Sciences, Moscow 119334, Russia., Dzhenkova MA; Laboratory of Modeling and Therapy of Hereditary Diseases, Institute of Gene Biology, Russian Academy of Sciences, Moscow 119334, Russia., Savchenko IM; Center for Precision Genome Editing and Genetic Technologies for Biomedicine, Institute of Gene Biology Russian Academy of Sciences, Moscow 119334, Russia., Velyaev OA; Laboratory of Modeling and Therapy of Hereditary Diseases, Institute of Gene Biology, Russian Academy of Sciences, Moscow 119334, Russia., Shmidt AA; Laboratory of Modeling and Therapy of Hereditary Diseases, Institute of Gene Biology, Russian Academy of Sciences, Moscow 119334, Russia.; Center for Precision Genome Editing and Genetic Technologies for Biomedicine, Institute of Gene Biology Russian Academy of Sciences, Moscow 119334, Russia., Soldatov VO; Research Institute of Living Systems Pharmacology, Belgorod National Research University, Belgorod 308007, Russia., Pokrovskii MV; Research Institute of Living Systems Pharmacology, Belgorod National Research University, Belgorod 308007, Russia., Deykin AV; Marlin Biotech LLC, Sochi 354340, Russia.; Joint Center for Genetic Technologies, Laboratory of Genetic Technologies and Gene Editing for Biomedicine and Veterinary Medicine, Department of Pharmacology and Clinical Pharmacology, Belgorod National Research University, Belgorod 308015, Russia., Bardina MV; Laboratory of Modeling and Therapy of Hereditary Diseases, Institute of Gene Biology, Russian Academy of Sciences, Moscow 119334, Russia.; Marlin Biotech LLC, Sochi 354340, Russia.; Center for Precision Genome Editing and Genetic Technologies for Biomedicine, Institute of Gene Biology Russian Academy of Sciences, Moscow 119334, Russia.

المصدر: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2023 Jun 17; Vol. 30, pp. 161-180. Date of Electronic Publication: 2023 Jun 17 (Print Publication: 2023).

نوع المنشور: Journal Article

بيانات الدورية: Publisher: Cell Press Country of Publication: United States NLM ID: 101624857 Publication Model: eCollection Cited Medium: Print ISSN: 2329-0501 (Print) Linking ISSN: 23290501 NLM ISO Abbreviation: Mol Ther Methods Clin Dev Subsets: PubMed not MEDLINE

المؤلفون: Egorova TV; Laboratory of Modeling and Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow 119334, Russia.; Marlin Biotech LLC, Sochi 354340, Russia., Galkin II; Marlin Biotech LLC, Sochi 354340, Russia.; Center for Precision Genome Editing and Genetic Technologies for Biomedicine, Institute of Gene Biology Russian Academy of Sciences, Moscow 119334, Russia.; Belozersky Institute of Physico-Chemical Biology, Lomonosov Moscow State University, Moscow 119991, Russia., Velyaev OA; Laboratory of Modeling and Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow 119334, Russia., Vassilieva SG; Laboratory of Modeling and Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow 119334, Russia., Savchenko IM; Laboratory of Modeling and Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow 119334, Russia.; Center for Precision Genome Editing and Genetic Technologies for Biomedicine, Institute of Gene Biology Russian Academy of Sciences, Moscow 119334, Russia., Loginov VA; Laboratory of Modeling and Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow 119334, Russia., Dzhenkova MA; Laboratory of Modeling and Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow 119334, Russia., Korshunova DS; Core Facilities, Institute of Gene Biology Russian Academy of Sciences, Moscow 119334, Russia., Kozlova OS; Laboratory of Modeling and Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow 119334, Russia., Ivankov DN; Center for Molecular and Cellular Biology, Skolkovo Institute of Science and Technology, Moscow 121205, Russia., Polikarpova AV; Laboratory of Modeling and Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow 119334, Russia.; Marlin Biotech LLC, Sochi 354340, Russia.

المصدر: International journal of molecular sciences [Int J Mol Sci] 2023 May 23; Vol. 24 (11). Date of Electronic Publication: 2023 May 23.

نوع المنشور: Journal Article

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

مواضيع طبية MeSH: Dystrophin*/genetics , Muscular Dystrophy, Duchenne*/metabolism, Mice ; Animals ; Mice, Inbred CBA ; Phenotype ; Exons/genetics ; Gene Deletion