Abstract:With the advent of endonuclease methods of genome editing, particularly CRISPR/Cas9, it has become possible to obtain genetically modified rabbits by microinjection of zygotes. These highly effective human disease models can be used for various purposes. The present review aims to consider modern achievements in the creation of rabbit biomodels of human diseases using the technologies of genetic editing. It is concluded that Russian laboratories should intensify research in the development of genetically modif… Show more
“…Появление эндонуклеазных методов и технологий редактирования генома позволило получать крупных генетически модифицированных животных -кроликов. Более ранний метод получения таких животных -классический трансгенез -был низкоэффективным [4].…”
Дегенерации сетчатки представляют собой обширную и гетерогенную группу заболеваний (дистрофий) сетчатки, которые приводят к прогрессирующей потере зрения. Одновременно с развитием новых методов экспериментальной терапии (генной и клеточной терапии, регенеративной офтальмохирургии) возрастает потребность в экспериментальных моделях. В обзоре рассматриваются различные животные модели дегенеративных заболеваний сетчатки глаза человека. Индуцированные и генетические экспериментальные модели имеют свои преимущества и недостатки, освещающиеся в обзоре. Адекватный выбор экспериментальной модели может способствовать патогенетическому подходу лечения заболеваний сетчатки.
“…Появление эндонуклеазных методов и технологий редактирования генома позволило получать крупных генетически модифицированных животных -кроликов. Более ранний метод получения таких животных -классический трансгенез -был низкоэффективным [4].…”
Дегенерации сетчатки представляют собой обширную и гетерогенную группу заболеваний (дистрофий) сетчатки, которые приводят к прогрессирующей потере зрения. Одновременно с развитием новых методов экспериментальной терапии (генной и клеточной терапии, регенеративной офтальмохирургии) возрастает потребность в экспериментальных моделях. В обзоре рассматриваются различные животные модели дегенеративных заболеваний сетчатки глаза человека. Индуцированные и генетические экспериментальные модели имеют свои преимущества и недостатки, освещающиеся в обзоре. Адекватный выбор экспериментальной модели может способствовать патогенетическому подходу лечения заболеваний сетчатки.
“…In recent years, using CRISPR/Cas9 technology there were produced GM rabbitsmodels of such human diseases as: cardiovascular (hypertrophic cardiomyopathy, atherosclerosis, arterial thrombosis); immune diseases, including immunodeficiency; infectious diseases; metabolic syndromes due to both nutrition and heredity; Xchromosome-related diseases -hypophosphatemia (a common cause of inherited rickets) and Duchenne Muscular Dystrophy; progeria syndrome; hermaphroditism; ectodermal dysplasia; cleft palate; Wilson's disease (copper metabolism disorder, leading to severe hereditary diseases of the Central nervous system and internal organs); models were created to study the metabolism of lipids and lipoproteins [14].…”
Genetically modified (GM) animals are necessary to solve the global problems of humanity related to nutrition and health. Rabbits, as laboratory, domestic and farm animals, occupy a special niche in research. GM rabbits are promising as bioreactors for producing biologically active (BA) proteins with milk or blood, and are in demand in Biomedicine as biomodels of diseases. To date, many GM rabbits-biomodels, producers of recombinant proteins have been created in the world using CRISPR/Cas9 technology. All-Russian Research Institute of Animal Physiology, Biochemistry and Nutrition has experience in obtaining transgenic rabbitsproducers of human BA proteins with milk by microinjecting recombinant DNA into zygote pronuclei. The possibility of site-specific modification of the rabbit whey acidic protein (WAP) gene using CRISPR/Cas9 technology is discussed. A DNA matrix containing homology arms to the WAP rabbit gene and site-specific CRISPR/Cas9 components in plasmid form were obtained. Microinjections of rabbit zygotes were performed and embryo survival was evaluated in vitro. The efficiency of using the green fluorescent protein gene under the cytomegalovirus promoter in the DNA matrix as an indicator of homologically directed repair was evaluated. This work can be useful for obtaining rabbits that produce with milk BA protein instead of WAP.
Monoclonal antibodies are widely used in all fields of biology and medicine. The emergence and development of the technology for their production revolutionized immunology and allowed the creation of not only new diagnostic methods, but also many effective drugs. The purpose of our review is to analyze and summarize relevant data concerning the technology of obtaining monoclonal antibodies. We have analyzed information from 70 modern literary sources devoted to various methods of obtaining them. In this review, we tried to cover the entire range of methods used to obtain monoclonal antibodies today.
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