Development of an AAV-RNAi strategy to silence the dominant variant GNAO1 c.607G>A linked to encephalopathy.
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| Title: | Development of an AAV-RNAi strategy to silence the dominant variant GNAO1 c.607G>A linked to encephalopathy. |
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| Authors: | 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., Klementieva NV; Laboratory of Modeling and Gene Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow, Russia.; Marlin Biotech, Sochi, 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., Volovikov EA; Lopukhin Federal Research and Clinical Center of Physical-Chemical Medicine of the Federal Medical and Biological Agency, Moscow, Russia., Jappy D; Federal Center of Brain Research and Neurotechnologies, Moscow, 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., Svetlova EA; Laboratory of Modeling and Gene Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow, Russia.; Marlin Biotech, Sochi, Russia., 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., Shubina MY; Laboratory of Modeling and Gene Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow, Russia.; Marlin Biotech, Sochi, Russia., Spirin DM; Lopukhin Federal Research and Clinical Center of Physical-Chemical Medicine of the Federal Medical and Biological Agency, Moscow, Russia., Anufrieva KS; Lopukhin Federal Research and Clinical Center of Physical-Chemical Medicine of the Federal Medical and Biological Agency, Moscow, Russia., Lebedev PR; Laboratory of Genetic Technologies and Genome Editing for Biomedicine and Animal Health, Joint Center for Genetic Technologies, Belgorod National Research University, Belgorod, Russia., Pokrovsky VM; Laboratory of Genetic Technologies and Genome Editing for Biomedicine and Animal Health, Joint Center for Genetic Technologies, Belgorod National Research University, Belgorod, Russia., Utkina MV; Endocrinology Research Center, Moscow, Russia., Krut' VG; Federal Center of Brain Research and Neurotechnologies, Moscow, Russia., Sintsov M; Federal Center of Brain Research and Neurotechnologies, Moscow, Russia., Popov S; Endocrinology Research Center, Moscow, Russia., Deykin AV; Laboratory of Genetic Technologies and Genome Editing for Biomedicine and Animal Health, Joint Center for Genetic Technologies, Belgorod National Research University, Belgorod, Russia., Rozov A; Federal Center of Brain Research and Neurotechnologies, Moscow, 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., Bardina MV; Laboratory of Modeling and Gene Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow, Russia. maryana.bardina@gmail.com.; Marlin Biotech, Sochi, Russia. maryana.bardina@gmail.com.; Center for Precision Genome Editing and Genetic Technologies for Biomedicine, Institute of Gene Biology, Russian Academy of Sciences, Moscow, Russia. maryana.bardina@gmail.com. |
| Source: | Gene therapy [Gene Ther] 2025 Jul; Vol. 32 (4), pp. 430-445. Date of Electronic Publication: 2025 Apr 14. |
| Publication Type: | Journal Article; Research Support, Non-U.S. Gov't |
| Journal Info: | Publisher: Nature Publishing Group Country of Publication: England NLM ID: 9421525 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1476-5462 (Electronic) Linking ISSN: 09697128 NLM ISO Abbreviation: Gene Ther Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
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