Dual Adeno-Associated Virus 9 with Codon-Optimized DYSF Gene Promotes In Vivo Muscle Regeneration and May Decrease Inflammatory Response in Limb Girdle Muscular Dystrophy Type R2.

Autor: Yakovlev IA; Genotarget LLC, Skolkovo Innovation Center, 121205 Moscow, Russia.; PJSC Human Stem Cells Institute, 129110 Moscow, Russia., Emelin AM; Department of Pathological Anatomy, I. I. Mechnikov North-West State Medical University, Ministry of Health of the Russian Federation, 191036 St. Petersburg, Russia., Slesarenko YS; Genotarget LLC, Skolkovo Innovation Center, 121205 Moscow, Russia., Limaev IS; Department of Pathological Anatomy, I. I. Mechnikov North-West State Medical University, Ministry of Health of the Russian Federation, 191036 St. Petersburg, Russia., Vetrova IA; Department of Pathological Anatomy, I. I. Mechnikov North-West State Medical University, Ministry of Health of the Russian Federation, 191036 St. Petersburg, Russia., Belikova LD; Lopukhin Federal Research and Clinical Center of Physical-Chemical Medicine of Federal Medical Biological Agency, 119435 Moscow, Russia.; Faculty of Bioengineering and Bioinformatics, Lomonosov Moscow State University, 119992 Moscow, Russia., Grafskaia EN; Lopukhin Federal Research and Clinical Center of Physical-Chemical Medicine of Federal Medical Biological Agency, 119435 Moscow, Russia., Bobrovsky PA; Lopukhin Federal Research and Clinical Center of Physical-Chemical Medicine of Federal Medical Biological Agency, 119435 Moscow, Russia., Pokrovsky MV; Laboratory for Modeling and Gene Therapy of Human Diseases, Belgorod State National Research University, 308015 Belgorod, Russia., Kuzubova EV; Laboratory for Modeling and Gene Therapy of Human Diseases, Belgorod State National Research University, 308015 Belgorod, Russia., Pokrovsky VM; Laboratory for Modeling and Gene Therapy of Human Diseases, Belgorod State National Research University, 308015 Belgorod, Russia., Lebedev PA; Laboratory for Modeling and Gene Therapy of Human Diseases, Belgorod State National Research University, 308015 Belgorod, Russia., Bardakov SN; PJSC Human Stem Cells Institute, 129110 Moscow, Russia., Isaev AA; PJSC Human Stem Cells Institute, 129110 Moscow, Russia., Deev RV; Genotarget LLC, Skolkovo Innovation Center, 121205 Moscow, Russia.; PJSC Human Stem Cells Institute, 129110 Moscow, Russia.; Department of Pathological Anatomy, I. I. Mechnikov North-West State Medical University, Ministry of Health of the Russian Federation, 191036 St. Petersburg, Russia.

Publikováno v: International journal of molecular sciences [Int J Mol Sci] 2023 Aug 31; Vol. 24 (17). Date of Electronic Publication: 2023 Aug 31.

P.F508del editing in cells from cystic fibrosis patients.

Autor: Smirnikhina SA; Laboratory of Genome Editing, Research Centre for Medical Genetics, Moscow, Russian Federation., Kondrateva EV; Laboratory of Genome Editing, Research Centre for Medical Genetics, Moscow, Russian Federation., Adilgereeva EP; Laboratory of Genome Editing, Research Centre for Medical Genetics, Moscow, Russian Federation., Anuchina AA; Laboratory of Genome Editing, Research Centre for Medical Genetics, Moscow, Russian Federation., Zaynitdinova MI; Laboratory of Genome Editing, Research Centre for Medical Genetics, Moscow, Russian Federation., Slesarenko YS; Laboratory of Genome Editing, Research Centre for Medical Genetics, Moscow, Russian Federation., Ershova AS; Nazarbayev University, School of Science and Technology, Nur-Sultan, Republic of Kazakhstan., Ustinov KD; Laboratory of Genome Editing, Research Centre for Medical Genetics, Moscow, Russian Federation., Yasinovsky MI; Laboratory of Genome Editing, Research Centre for Medical Genetics, Moscow, Russian Federation., Amelina EL; Laboratory of Cystic Fibrosis, Research Institute of Pulmonology, Moscow, Russian Federation., Voronina ES; Laboratory of Genome Editing, Research Centre for Medical Genetics, Moscow, Russian Federation., Yakushina VD; Laboratory of Genome Editing, Research Centre for Medical Genetics, Moscow, Russian Federation., Tabakov VY; Laboratory of Genome Editing, Research Centre for Medical Genetics, Moscow, Russian Federation., Lavrov AV; Laboratory of Genome Editing, Research Centre for Medical Genetics, Moscow, Russian Federation.

Publikováno v: PloS one [PLoS One] 2020 Nov 11; Vol. 15 (11), pp. e0242094. Date of Electronic Publication: 2020 Nov 11 (Print Publication: 2020).