Primena crISPr/cas9 tehnologije u otkrivanju novih molekularnih terapeutika
Application of crISPr/cas9 technology in the discovery of new molecular therapeutics
Аутори
Skakić, AnitaStojiljković, Maja
Остала ауторства
Pavlović, SonjaBegović, Jelena
Novaković, Ivana
Savić Pavićević, Dušanka
Đorđević, Ana
Поглавље у монографији (Објављена верзија)
Метаподаци
Приказ свих података о документуАпстракт
CRISPR/Cas9 (eng. Clustered Regularly Interspaced Short Palindromic Repeats/CRISPR associated protein 9) je prirodni
alat za editovanje genoma usvojen iz prokariotskog adaptivnog imunskog odbrambenog sistema.
Zbog svoje visoke efikasnosti i preciznosti, protein Cas9 koji pripada CRISPR sistemu klase II, pronašao je
primenu u mnogim poljima nauke. Editovanje genoma zasnovano na tehnologiji CRISPR/Cas9 predstavlja
jedan od najperspektivnijih alata za lečenje humanih bolesti sa genetičkom osnovom, uključujući kardiovaskularne
bolesti, neurodegenerativne poremećaje i različite vrste tumora. Genska terapija zasnovana na
CRISPR/Cas9 opsežno se proučava u pretkliničkim i kliničkim studijama. Editovanje genoma CRISPR/Cas9 je
takođe robustan alat za stvaranje in vitro ćelijskih i životinjskih model sistema za istraživanje i lečenje genetičkih
bolesti, posebno bolesti povezanih sa tačkastim promenama. U ovom radu, osvrnućemo se kratko na
istoriju i mehanizam sistema CRISPR/Cas9, različi...te metodološke pristupe, napraviti pregled primena u biomedicini
i opisati njegovu ulogu u razvoju novih molekularnih terapeutika za retke nasledne bolesti.
Clustered Regularly Interspaced Short Palindromic Repeats/CRISPR associated protein 9 (CRISPR/Cas9) is a
naturally occurring genome editing tool adopted from the prokaryotic adaptive immune defense system.
Due to its high efficiency and precision, the Cas9 protein derived from the type II CRISPR system, has found
applications in many fields of science. Currently, CRISPR/Cas9-based genome editing has become one of
the most promising tools for treating human genetic diseases, including cardiovascular diseases, neurodegenerative
disorders, and various types of tumors. CRISPR/Cas9-based gene therapy is extensively studied
in preclinic and clinic treatments. CRISPR/Cas9 genome editing is also a robust tool to create in vitro cellular
and animal model systems for investigating and treating human genetic disorders, particularly diseases
associated with point mutations. Therefore, in this review, we will present a brief history and mechanism of
the CRISPR/Cas9 system. We will also des...cribe the different methodological approaches, review applications
in biomedicine and describe its role in the development of new molecular therapeutics for rare inherited
diseases.
Кључне речи:
CRISPR/Cas9 / editovanje genoma / genska terapija / in vitro i životinjski model sistemi / retke bolesti / molekularni terapeutici / genome editing / gene therapy / in vitro and animal model systems / rare diseases / molecular therapeuticsИзвор:
Trendovi u molekularnoj Biologiji, 2021, 42-52Издавач:
- Trendovi u molekularnoj Biologiji
Финансирање / пројекти:
- Министарство науке, технолошког развоја и иновација Републике Србије, институционално финансирање - 200042 (Универзитет у Београду, Институт за молекуларну генетику и генетичко инжењерство) (RS-MESTD-inst-2020-200042)
Колекције
Институција/група
Institut za molekularnu genetiku i genetičko inženjerstvoTY - CHAP AU - Skakić, Anita AU - Stojiljković, Maja PY - 2021 UR - https://imagine.imgge.bg.ac.rs/handle/123456789/1728 AB - CRISPR/Cas9 (eng. Clustered Regularly Interspaced Short Palindromic Repeats/CRISPR associated protein 9) je prirodni alat za editovanje genoma usvojen iz prokariotskog adaptivnog imunskog odbrambenog sistema. Zbog svoje visoke efikasnosti i preciznosti, protein Cas9 koji pripada CRISPR sistemu klase II, pronašao je primenu u mnogim poljima nauke. Editovanje genoma zasnovano na tehnologiji CRISPR/Cas9 predstavlja jedan od najperspektivnijih alata za lečenje humanih bolesti sa genetičkom osnovom, uključujući kardiovaskularne bolesti, neurodegenerativne poremećaje i različite vrste tumora. Genska terapija zasnovana na CRISPR/Cas9 opsežno se proučava u pretkliničkim i kliničkim studijama. Editovanje genoma CRISPR/Cas9 je takođe robustan alat za stvaranje in vitro ćelijskih i životinjskih model sistema za istraživanje i lečenje genetičkih bolesti, posebno bolesti povezanih sa tačkastim promenama. U ovom radu, osvrnućemo se kratko na istoriju i mehanizam sistema CRISPR/Cas9, različite metodološke pristupe, napraviti pregled primena u biomedicini i opisati njegovu ulogu u razvoju novih molekularnih terapeutika za retke nasledne bolesti. AB - Clustered Regularly Interspaced Short Palindromic Repeats/CRISPR associated protein 9 (CRISPR/Cas9) is a naturally occurring genome editing tool adopted from the prokaryotic adaptive immune defense system. Due to its high efficiency and precision, the Cas9 protein derived from the type II CRISPR system, has found applications in many fields of science. Currently, CRISPR/Cas9-based genome editing has become one of the most promising tools for treating human genetic diseases, including cardiovascular diseases, neurodegenerative disorders, and various types of tumors. CRISPR/Cas9-based gene therapy is extensively studied in preclinic and clinic treatments. CRISPR/Cas9 genome editing is also a robust tool to create in vitro cellular and animal model systems for investigating and treating human genetic disorders, particularly diseases associated with point mutations. Therefore, in this review, we will present a brief history and mechanism of the CRISPR/Cas9 system. We will also describe the different methodological approaches, review applications in biomedicine and describe its role in the development of new molecular therapeutics for rare inherited diseases. PB - Trendovi u molekularnoj Biologiji T2 - Trendovi u molekularnoj Biologiji T1 - Primena crISPr/cas9 tehnologije u otkrivanju novih molekularnih terapeutika T1 - Application of crISPr/cas9 technology in the discovery of new molecular therapeutics EP - 52 SP - 42 UR - https://hdl.handle.net/21.15107/rcub_imagine_1728 ER -
@inbook{ author = "Skakić, Anita and Stojiljković, Maja", year = "2021", abstract = "CRISPR/Cas9 (eng. Clustered Regularly Interspaced Short Palindromic Repeats/CRISPR associated protein 9) je prirodni alat za editovanje genoma usvojen iz prokariotskog adaptivnog imunskog odbrambenog sistema. Zbog svoje visoke efikasnosti i preciznosti, protein Cas9 koji pripada CRISPR sistemu klase II, pronašao je primenu u mnogim poljima nauke. Editovanje genoma zasnovano na tehnologiji CRISPR/Cas9 predstavlja jedan od najperspektivnijih alata za lečenje humanih bolesti sa genetičkom osnovom, uključujući kardiovaskularne bolesti, neurodegenerativne poremećaje i različite vrste tumora. Genska terapija zasnovana na CRISPR/Cas9 opsežno se proučava u pretkliničkim i kliničkim studijama. Editovanje genoma CRISPR/Cas9 je takođe robustan alat za stvaranje in vitro ćelijskih i životinjskih model sistema za istraživanje i lečenje genetičkih bolesti, posebno bolesti povezanih sa tačkastim promenama. U ovom radu, osvrnućemo se kratko na istoriju i mehanizam sistema CRISPR/Cas9, različite metodološke pristupe, napraviti pregled primena u biomedicini i opisati njegovu ulogu u razvoju novih molekularnih terapeutika za retke nasledne bolesti., Clustered Regularly Interspaced Short Palindromic Repeats/CRISPR associated protein 9 (CRISPR/Cas9) is a naturally occurring genome editing tool adopted from the prokaryotic adaptive immune defense system. Due to its high efficiency and precision, the Cas9 protein derived from the type II CRISPR system, has found applications in many fields of science. Currently, CRISPR/Cas9-based genome editing has become one of the most promising tools for treating human genetic diseases, including cardiovascular diseases, neurodegenerative disorders, and various types of tumors. CRISPR/Cas9-based gene therapy is extensively studied in preclinic and clinic treatments. CRISPR/Cas9 genome editing is also a robust tool to create in vitro cellular and animal model systems for investigating and treating human genetic disorders, particularly diseases associated with point mutations. Therefore, in this review, we will present a brief history and mechanism of the CRISPR/Cas9 system. We will also describe the different methodological approaches, review applications in biomedicine and describe its role in the development of new molecular therapeutics for rare inherited diseases.", publisher = "Trendovi u molekularnoj Biologiji", journal = "Trendovi u molekularnoj Biologiji", booktitle = "Primena crISPr/cas9 tehnologije u otkrivanju novih molekularnih terapeutika, Application of crISPr/cas9 technology in the discovery of new molecular therapeutics", pages = "52-42", url = "https://hdl.handle.net/21.15107/rcub_imagine_1728" }
Skakić, A.,& Stojiljković, M.. (2021). Primena crISPr/cas9 tehnologije u otkrivanju novih molekularnih terapeutika. in Trendovi u molekularnoj Biologiji Trendovi u molekularnoj Biologiji., 42-52. https://hdl.handle.net/21.15107/rcub_imagine_1728
Skakić A, Stojiljković M. Primena crISPr/cas9 tehnologije u otkrivanju novih molekularnih terapeutika. in Trendovi u molekularnoj Biologiji. 2021;:42-52. https://hdl.handle.net/21.15107/rcub_imagine_1728 .
Skakić, Anita, Stojiljković, Maja, "Primena crISPr/cas9 tehnologije u otkrivanju novih molekularnih terapeutika" in Trendovi u molekularnoj Biologiji (2021):42-52, https://hdl.handle.net/21.15107/rcub_imagine_1728 .