Genes & CellsGenes & CellsГены и КлеткиGenes and Cells2313-18292500-2562Human Stem Cells Institute12057010.23868/gc120570ArticlesСтатьиReview ArticlePreand posttranscriptional genetic information modification in muscular dystrophy treatmentПРЕД- И ПОСТТРАНСКРИПЦИОННАЯ МОДИФИКАЦИЯ ГЕНЕТИЧЕСКОЙ ИНФОРМАЦИИ В ПРОГРАММЕ ЛЕЧЕНИЯ МЫШЕЧНЫХ ДИСТРОФИЙYakovlevI. AЯковлевИ. Аivan@ivan-ya.ruDeevR. VДеевР. ВSolovyevaV. VСоловьеваВ. ВRizvanovA. AРизвановА. АIsaevA. AИсаевА. АHuman Stem Cells InstituteИнститут стволовых клеток человекаKazan (Volga region) Federal UniversityКазанский (Приволжский) федеральный университет15062016112VOL 11, NO2 (2016)ТОМ 11, №2 (2016)425205012023Copyright ©; 2016, Eco-VectorCopyright ©; 2016, Эко-Вектор2016Eco-VectorЭко-Векторhttps://genescells.ru/2313-1829/article/view/120570

Nowadays, a whole range of genetherapeutic methods is being used to restore a lost protein function due to mutation, a big number of preclinical and clinical studies of potential drugs that may allow to implement an etiotropic approach is being performed. 0ne of the most prevalent and socially significant groups of genetic pathologies is muscular dystrophy, including such diseases as Duchenne muscular dystrophy and dysfelinopathy. Despite a large number of studies in this field, there is no effective method of gene therapy for these diseases yet. This work is intended to review main genetherapeutic methods in myodystrophy treatment, especially pre- and posttranscriptional genetic (biosynthetic) information modification, and analyze most optimal of them.

Сегодня имеется целый ряд геннотерапевтических методов для восстановления функции белка утраченной в результате мутации, проводится большое количество как доклинических, так и клинических исследований безопасности и эффективности препаратов-кандидатов, которые должны позволить осуществить этиотропный подход к лечению наследственных заболеваний. Одной из самых распространенных и социально значимых групп генетических болезней является мышечная дистрофия, включающая в себя такие заболевания как миодистрофия Дюшенна и дисферлинопатия. Для этих заболеваний пока не найдено эффективного метода генной терапии, несмотря на значительный объем исследований в этой области. Настоящий обзор призван рассмотреть основные геннотераветические стратегии в лечении миодистрофий, а именно пред- и посттранскрипционную модификацию биосинтетической (генетической) информации и проанализировать наиболее перспективные из них.

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