Cas-Based Systems for RNA Editing in Gene Therapy of Monogenic Diseases

Standard

Cas-Based Systems for RNA Editing in Gene Therapy of Monogenic Diseases : In Vitro and in Vivo Application and Translational Potential. / Reshetnikov, Vasiliy V.; Chirinskaite, Angelina V.; Sopova, Julia V.; Ivanov, Roman A.; Leonova, Elena I.

In: Frontiers in Cell and Developmental Biology, Vol. 10, 903812, 16.06.2022.

Research output: Contribution to journal › Review article › peer-review

BibTeX

@article{7634d420c12b4958a5002790e001c5be,

title = "Cas-Based Systems for RNA Editing in Gene Therapy of Monogenic Diseases: In Vitro and in Vivo Application and Translational Potential",

abstract = "Rare genetic diseases reduce quality of life and can significantly shorten the lifespan. There are few effective treatment options for these diseases, and existing therapeutic strategies often represent only supportive or palliative care. Therefore, designing genetic-engineering technologies for the treatment of genetic diseases is urgently needed. Rapid advances in genetic editing technologies based on programmable nucleases and in the engineering of gene delivery systems have made it possible to conduct several dozen successful clinical trials; however, the risk of numerous side effects caused by off-target double-strand breaks limits the use of these technologies in the clinic. Development of adenine-to-inosine (A-to-I) and cytosine-to-uracil (C-to-U) RNA-editing systems based on dCas13 enables editing at the transcriptional level without double-strand breaks in DNA. In this review, we discuss recent progress in the application of these technologies in in vitro and in vivo experiments. The main strategies for improving RNA-editing tools by increasing their efficiency and specificity are described as well. These data allow us to outline the prospects of base-editing systems for clinical application.",

keywords = "cure, dCas13, gene therapy, monogenic disease, repair, rescue, RNA editing",

author = "Reshetnikov, {Vasiliy V.} and Chirinskaite, {Angelina V.} and Sopova, {Julia V.} and Ivanov, {Roman A.} and Leonova, {Elena I.}",

note = "Publisher Copyright: Copyright {\textcopyright} 2022 Reshetnikov, Chirinskaite, Sopova, Ivanov and Leonova.",

year = "2022",

month = jun,

day = "16",

doi = "10.3389/fcell.2022.903812",

language = "English",

volume = "10",

journal = "Frontiers in Cell and Developmental Biology",

issn = "2296-634X",

publisher = "JMIR PUBLICATIONS, INC",

}

RIS

TY - JOUR

T1 - Cas-Based Systems for RNA Editing in Gene Therapy of Monogenic Diseases

T2 - In Vitro and in Vivo Application and Translational Potential

AU - Reshetnikov, Vasiliy V.

AU - Chirinskaite, Angelina V.

AU - Sopova, Julia V.

AU - Ivanov, Roman A.

AU - Leonova, Elena I.

PY - 2022/6/16

Y1 - 2022/6/16

N2 - Rare genetic diseases reduce quality of life and can significantly shorten the lifespan. There are few effective treatment options for these diseases, and existing therapeutic strategies often represent only supportive or palliative care. Therefore, designing genetic-engineering technologies for the treatment of genetic diseases is urgently needed. Rapid advances in genetic editing technologies based on programmable nucleases and in the engineering of gene delivery systems have made it possible to conduct several dozen successful clinical trials; however, the risk of numerous side effects caused by off-target double-strand breaks limits the use of these technologies in the clinic. Development of adenine-to-inosine (A-to-I) and cytosine-to-uracil (C-to-U) RNA-editing systems based on dCas13 enables editing at the transcriptional level without double-strand breaks in DNA. In this review, we discuss recent progress in the application of these technologies in in vitro and in vivo experiments. The main strategies for improving RNA-editing tools by increasing their efficiency and specificity are described as well. These data allow us to outline the prospects of base-editing systems for clinical application.

AB - Rare genetic diseases reduce quality of life and can significantly shorten the lifespan. There are few effective treatment options for these diseases, and existing therapeutic strategies often represent only supportive or palliative care. Therefore, designing genetic-engineering technologies for the treatment of genetic diseases is urgently needed. Rapid advances in genetic editing technologies based on programmable nucleases and in the engineering of gene delivery systems have made it possible to conduct several dozen successful clinical trials; however, the risk of numerous side effects caused by off-target double-strand breaks limits the use of these technologies in the clinic. Development of adenine-to-inosine (A-to-I) and cytosine-to-uracil (C-to-U) RNA-editing systems based on dCas13 enables editing at the transcriptional level without double-strand breaks in DNA. In this review, we discuss recent progress in the application of these technologies in in vitro and in vivo experiments. The main strategies for improving RNA-editing tools by increasing their efficiency and specificity are described as well. These data allow us to outline the prospects of base-editing systems for clinical application.

KW - cure

KW - dCas13

KW - gene therapy

KW - monogenic disease

KW - repair

KW - rescue

KW - RNA editing

UR - http://www.scopus.com/inward/record.url?scp=85133505521&partnerID=8YFLogxK

UR - https://www.mendeley.com/catalogue/bffa179d-e45c-3f01-a3b0-653422c909ab/

U2 - 10.3389/fcell.2022.903812

DO - 10.3389/fcell.2022.903812

M3 - Review article

AN - SCOPUS:85133505521

VL - 10

JO - Frontiers in Cell and Developmental Biology

JF - Frontiers in Cell and Developmental Biology

SN - 2296-634X

M1 - 903812

ER -

ID: 97181087

Cas-Based Systems for RNA Editing in Gene Therapy of Monogenic Diseases: In Vitro and in Vivo Application and Translational Potential

Standard

Harvard

APA

Vancouver

Author

BibTeX

RIS