CRISPR/Cas9-generated mouse model of Duchenne muscular dystrophy recapitulating a newly identified large 430 kb deletion in the human <i>DMD</i> gene.

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第一作者： Tatiana V,Egorova

第一单位： Laboratory of Modeling and Gene Therapy of Hereditary Diseases, Institute of Gene Biology, Russian Academy of Sciences, Moscow, 119334, Russia t.dimitrieva@marlinbiotech.com.

作者： Tatiana V,Egorova ^[1] ; Evgenia D,Zotova ^[2] ; Denis A,Reshetov ^[2] ; Anna V,Polikarpova ^[3] ; Svetlana G,Vassilieva ^[4] ; Dmitry V,Vlodavets ^[2] ; Alexey A,Gavrilov ^[4] ; Sergey V,Ulianov ^[2] ; Vladimir L,Buchman ^[5] ; Alexei V,Deykin ^[6]

作者单位： Laboratory of Modeling and Gene Therapy of Hereditary Diseases, Institute of Gene Biology, Russian Academy of Sciences, Moscow, 119334, Russia t.dimitrieva@marlinbiotech.com. ^[1] Marlin Biotech LLC, Moscow, 143026, Russia. ^[2] Research Centre for Genetic Medicine, Moscow, 117292, Russia. ^[3] Laboratory of Modeling and Gene Therapy of Hereditary Diseases, Institute of Gene Biology, Russian Academy of Sciences, Moscow, 119334, Russia. ^[4] Veltischev Scientific Research Clinical Paediatric Institute, Moscow, 125412, Russia. ^[5] Group of Genome Spatial Organization, Institute of Gene Biology, Russian Academy of Sciences, Moscow, 119334, Russia. ^[6] Laboratory of Structural and Functional Organization of Chromosomes, Institute of Gene Biology, Russian Academy of Sciences, Moscow, 119334, Russia. ^[7] Faculty of Biology, M.V. Lomonosov Moscow State University, Moscow, 119991, Russia. ^[8] School of Biosciences, Cardiff University, Cardiff, CF10 3AX, UK. ^[9] Core Facilities, Institute of Gene Biology, Russian Academy of Sciences, Moscow, 119334, Russia. ^[10]

关键词 DMD Exon skipping Genome editing Mouse model Muscle degeneration New deletion