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

Autor: 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.

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