CRISPR/Cas9, a universal tool for genomic engineering

Smirnov, Alexey V.; Юнусова, А. М.; Лукьянчикова, В. А.; Battulin, Nariman

doi:10.18699/vj16.175

Cited by 5 publications

(1 citation statement)

References 115 publications

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“…This system consists of a CRISPR cassette and the gene encoding Cas9 protein. The CRISPR-Cas9 system provides prokaryotic cells resistance to bacteriophages and plasmids containing protospacers, i.e., the sequences which are complimentary to spacers of the CRISPR cassette [9,10]. In this system, the Cas9 nuclease forms a double-stranded DNA break, and the site of this break is determined by the sequence of a singlestranded guide RNA (sgRNA).…”

Section: а(C)dаmentioning

confidence: 99%

Using Genome Editing for Alzheimer’s Disease Therapy: from Experiment to Clinic

Stepanichev

2021

Neurochem. J.

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Here, we review the current achievements in the use of genome editing methods for experimental therapy of neurodegenerative diseases, primarily Alzheimer's disease. The main approaches used for genome editing and editing systems as well as their advantages and disadvantages are considered. Specific examples of the use of genome editing to correct mutations in genes associated with Alzheimer's disease are presented. The existing problems that need to be solved for the introduction of genome editing in the practice of treating neurodegenerative diseases are analyzed.

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Section: а(C)dаmentioning

confidence: 99%

Using Genome Editing for Alzheimer’s Disease Therapy: from Experiment to Clinic

Stepanichev

2021

Neurochem. J.

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A perspective on the application of CRISPR/CAS9 genome editing system to study of molecular-genetic basis of mental disorders

Davydova,

Kazantseva,

Khusnutdinova

2024

Z. nevrol. psikhiatr. im. S.S. Korsakova

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Improvement of the knock-in effciency in the genome of human induced pluripotent stem cells using the CRISPR/Cas9 system

Gridina

2019

Vestn. VOGiS

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Human induced pluripotent stem (hiPS) cells are a powerful tool for biomedical research. The ability to create patient-specific pluripotent cells and their subsequent differentiation into any somatic cell type makes hiPS cells a valuable object for creating in vitro models of human diseases, screening drugs and a future source of cells for regenerative medicine. To realize entirely a potential of hiPScells, effective and precise methods for their genome editing are needed. The CRISPR/Cas9 system is the most widely used method for introducing site-specific double-stranded breaks into DNA. It allows genes of interest to be knocked out with high efficiency. However, knock-in into the target site of the genome is a much more difficult task. Moreover, many researchers have noted a low efficiency of introducing target constructs into the hiPS cells' genome. In this review, I attempt to describe the currently known information regarding the matter of increasing efficiency of targeted in sertions into hiPS cells' genome. Here I will describe the most effective strategies for designing the donor template for homo logy-directed repair, methods to manipulate the double-strand break repair pathways introduced by a nuclease, including control of CRISPR/ Cas9 delivery time. A low survival rate of hiPS cells following genome editing experiments is another difficulty on the way towards successful knock-in, and here several highly effective approaches addressing it are proposed. Finally, I describe the most promising strategies, one-step reprogramming and genome editing, which allows gene-modified integration-free hiPS cells to be efficiently generated directly from somatic cells.Key words: human induced pluripotent stem cells; CRISPR/Cas9 system; genome editing; knock-in efficiency.Индуцированные плюрипотентные стволовые клетки человека (чИПСК) -мощный инструмент для биомедицинских исследований. Возможность создания пациент-специфичных плюрипотентных клеток и последующая их дифференцировка в любой тип соматических клеток делают чИПСК замечательным объектом для создания in vitro моделей заболеваний, скрининга лекарственных препаратов и будущим источником клеточного материала для регенеративной медицины. Для того чтобы потенциал технологии чИПСК можно было реализовать в полном объеме, необходимы эффективные и точные методы редактирования генома этих клеток. В настоящее время система CRISPR/Cas9 -наиболее широко используемый подход для введения в ДНК сайт-специфичных двуцепочечных разрывов. С ее помощью с высокой эффективностью удается реализовать knock-out интересующих исследователя генов. Однако введение в целевое место генома заданной последовательности (knock-in) является существенно более сложной задачей. В зависимости от выбранного для проведения встройки локуса эффективность knock-in в геном чИПСК может составлять от 1 × 10 -5 до 1 × 10 -6 , что на порядок ниже, чем показано для эмбриональных стволовых клеток мышей или перевивных линий клеток. В этом обзоре я делаю попытку объединить и структурировать всю известную информаци...

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CRISPR/Cas9, a universal tool for genomic engineering

Cited by 5 publications

References 115 publications

Using Genome Editing for Alzheimer’s Disease Therapy: from Experiment to Clinic

Using Genome Editing for Alzheimer’s Disease Therapy: from Experiment to Clinic

A perspective on the application of CRISPR/CAS9 genome editing system to study of molecular-genetic basis of mental disorders

Improvement of the knock-in effciency in the genome of human induced pluripotent stem cells using the CRISPR/Cas9 system

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