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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.
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
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  Data: Development of an AAV-RNAi strategy to silence the dominant variant GNAO1 c.607G>A linked to encephalopathy.
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  Data: <searchLink fieldCode="AU" term="%22Lunev+EA%22">Lunev EA</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Klementieva+NV%22">Klementieva NV</searchLink>; Laboratory of Modeling and Gene Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow, Russia.; Marlin Biotech, Sochi, Russia.<br /><searchLink fieldCode="AU" term="%22Vassilieva+SG%22">Vassilieva SG</searchLink>; Laboratory of Modeling and Gene Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow, Russia.; Marlin Biotech, Sochi, Russia.<br /><searchLink fieldCode="AU" term="%22Volovikov+EA%22">Volovikov EA</searchLink>; Lopukhin Federal Research and Clinical Center of Physical-Chemical Medicine of the Federal Medical and Biological Agency, Moscow, Russia.<br /><searchLink fieldCode="AU" term="%22Jappy+D%22">Jappy D</searchLink>; Federal Center of Brain Research and Neurotechnologies, Moscow, Russia.<br /><searchLink fieldCode="AU" term="%22Savchenko+IM%22">Savchenko IM</searchLink>; Marlin Biotech, Sochi, Russia.; Center for Precision Genome Editing and Genetic Technologies for Biomedicine, Institute of Gene Biology, Russian Academy of Sciences, Moscow, Russia.<br /><searchLink fieldCode="AU" term="%22Svetlova+EA%22">Svetlova EA</searchLink>; Laboratory of Modeling and Gene Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow, Russia.; Marlin Biotech, Sochi, Russia.<br /><searchLink fieldCode="AU" term="%22Polikarpova+AV%22">Polikarpova AV</searchLink>; Laboratory of Modeling and Gene Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow, Russia.; Marlin Biotech, Sochi, Russia.<br /><searchLink fieldCode="AU" term="%22Shubina+MY%22">Shubina MY</searchLink>; Laboratory of Modeling and Gene Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow, Russia.; Marlin Biotech, Sochi, Russia.<br /><searchLink fieldCode="AU" term="%22Spirin+DM%22">Spirin DM</searchLink>; Lopukhin Federal Research and Clinical Center of Physical-Chemical Medicine of the Federal Medical and Biological Agency, Moscow, Russia.<br /><searchLink fieldCode="AU" term="%22Anufrieva+KS%22">Anufrieva KS</searchLink>; Lopukhin Federal Research and Clinical Center of Physical-Chemical Medicine of the Federal Medical and Biological Agency, Moscow, Russia.<br /><searchLink fieldCode="AU" term="%22Lebedev+PR%22">Lebedev PR</searchLink>; Laboratory of Genetic Technologies and Genome Editing for Biomedicine and Animal Health, Joint Center for Genetic Technologies, Belgorod National Research University, Belgorod, Russia.<br /><searchLink fieldCode="AU" term="%22Pokrovsky+VM%22">Pokrovsky VM</searchLink>; Laboratory of Genetic Technologies and Genome Editing for Biomedicine and Animal Health, Joint Center for Genetic Technologies, Belgorod National Research University, Belgorod, Russia.<br /><searchLink fieldCode="AU" term="%22Utkina+MV%22">Utkina MV</searchLink>; Endocrinology Research Center, Moscow, Russia.<br /><searchLink fieldCode="AU" term="%22Krut'+VG%22">Krut' VG</searchLink>; Federal Center of Brain Research and Neurotechnologies, Moscow, Russia.<br /><searchLink fieldCode="AU" term="%22Sintsov+M%22">Sintsov M</searchLink>; Federal Center of Brain Research and Neurotechnologies, Moscow, Russia.<br /><searchLink fieldCode="AU" term="%22Popov+S%22">Popov S</searchLink>; Endocrinology Research Center, Moscow, Russia.<br /><searchLink fieldCode="AU" term="%22Deykin+AV%22">Deykin AV</searchLink>; Laboratory of Genetic Technologies and Genome Editing for Biomedicine and Animal Health, Joint Center for Genetic Technologies, Belgorod National Research University, Belgorod, Russia.<br /><searchLink fieldCode="AU" term="%22Rozov+A%22">Rozov A</searchLink>; Federal Center of Brain Research and Neurotechnologies, Moscow, Russia.<br /><searchLink fieldCode="AU" term="%22Egorova+TV%22">Egorova TV</searchLink>; Laboratory of Modeling and Gene Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow, Russia.; Marlin Biotech, Sochi, Russia.<br /><searchLink fieldCode="AU" term="%22Bardina+MV%22">Bardina MV</searchLink>; 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.
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              Text: 2025 Jul
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