Article in the Genes and Cells Journal
The Genes and Cells Journal (Impact Factor 0.914, Q3) published an article by researchers of FBSI RCMG Genome Editing Laboratory (Lab Head is S.A. Smirnikhina, Candidate of med.sci.):
"Gene therapy approaches to Duchenne muscular dystrophy treatment"
M.I. Zainitdinova, S.A. Smirnikhina, A.V. Lavrov, I.I. Eremin, A.A. Pulin
The main experimental therapeutic approaches to the disease treatment based on gene therapy are considered. The Article shows that the most promising should be the CRISPR / Cas9 method, an indirect exon skipping. It allows making necessary changes in the genome after a single application and with great efficiency.
The Genes & Cells: Vol. XIV, No.4, 2019, p.: 6-18