In Vitro and In Vivo Genetic Disease Modeling via NHEJ-Precise Deletions Using CRISPR-Cas9

Sergio López-Manzaneda; Isabel Ojeda-Pérez; Nerea Zabaleta; Aída García-Torralba; Omaira Alberquilla; Raúl Torres; Rebeca Sánchez-Domínguez; Laura Torella; Emmanuel Olivier; Joanne Mountford; Juan C. Ramírez; Juan A. Bueren; Gloria González-Aseguinolaza; Jose Carlos Segovia

doi:10.1016/j.omtm.2020.10.007

In Vitro and In Vivo Genetic Disease Modeling via NHEJ-Precise Deletions Using CRISPR-Cas9

Sergio López-Manzaneda, Isabel Ojeda-Pérez, Nerea Zabaleta, Aída García-Torralba, Omaira Alberquilla, Raúl Torres, Rebeca Sánchez-Domínguez, Laura Torella, Emmanuel Olivier, Joanne Mountford, Juan C. Ramírez, Juan A. Bueren, Gloria González-Aseguinolaza, Jose Carlos Segovia

Research output: Contribution to journal › Article › peer-review

4 Scopus citations

Abstract

A CRISPR-Cas9 system generates precise deletions (up to 90% efficiency) when two guides are used simultaneously by means of non-homologous end joining. The PAM-in/PAM-in Cas9 orientation is the best combination to generate precise deletions. This gene-editing procedure facilitates the generation of homogeneously genetically defined cell and animal models.

Original language	English (US)
Pages (from-to)	426-437
Number of pages	12
Journal	Molecular Therapy - Methods and Clinical Development
Volume	19
DOIs	https://doi.org/10.1016/j.omtm.2020.10.007
State	Published - Dec 11 2020
Externally published	Yes

ASJC Scopus subject areas

Molecular Medicine
Molecular Biology
Genetics

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10.1016/j.omtm.2020.10.007

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López-Manzaneda, S., Ojeda-Pérez, I., Zabaleta, N., García-Torralba, A., Alberquilla, O., Torres, R., Sánchez-Domínguez, R., Torella, L., Olivier, E., Mountford, J., Ramírez, J. C., Bueren, J. A., González-Aseguinolaza, G., & Segovia, J. C. (2020). In Vitro and In Vivo Genetic Disease Modeling via NHEJ-Precise Deletions Using CRISPR-Cas9. Molecular Therapy - Methods and Clinical Development, 19, 426-437. https://doi.org/10.1016/j.omtm.2020.10.007

López-Manzaneda, S, Ojeda-Pérez, I, Zabaleta, N, García-Torralba, A, Alberquilla, O, Torres, R, Sánchez-Domínguez, R, Torella, L, Olivier, E, Mountford, J, Ramírez, JC, Bueren, JA, González-Aseguinolaza, G & Segovia, JC 2020, 'In Vitro and In Vivo Genetic Disease Modeling via NHEJ-Precise Deletions Using CRISPR-Cas9', Molecular Therapy - Methods and Clinical Development, vol. 19, pp. 426-437. https://doi.org/10.1016/j.omtm.2020.10.007

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title = "In Vitro and In Vivo Genetic Disease Modeling via NHEJ-Precise Deletions Using CRISPR-Cas9",

abstract = "A CRISPR-Cas9 system generates precise deletions (up to 90% efficiency) when two guides are used simultaneously by means of non-homologous end joining. The PAM-in/PAM-in Cas9 orientation is the best combination to generate precise deletions. This gene-editing procedure facilitates the generation of homogeneously genetically defined cell and animal models.",

author = "Sergio L{\'o}pez-Manzaneda and Isabel Ojeda-P{\'e}rez and Nerea Zabaleta and A{\'i}da Garc{\'i}a-Torralba and Omaira Alberquilla and Ra{\'u}l Torres and Rebeca S{\'a}nchez-Dom{\'i}nguez and Laura Torella and Emmanuel Olivier and Joanne Mountford and Ram{\'i}rez, {Juan C.} and Bueren, {Juan A.} and Gloria Gonz{\'a}lez-Aseguinolaza and Segovia, {Jose Carlos}",

note = "Funding Information: The authors would like to thank Miguel A. Martin for the careful maintenance of NSG mice, and Aurora de la Cal, Mar{\'i}a del Carmen S{\'a}nchez, Soledad Moreno, Nadia Abu-sabha, Montserrat Aldea, and Sergio Losada for dedicated administrative help. This work was supported by grants from the Ministerio de Econom{\'i}a, Comercio y Competitividad y Fondo Europeo de Desarrollo Regional (FEDER) ( SAF2017-84248-P ), the Fondo de Investigaciones Sanitarias, Instituto de Salud Carlos III ( Red TERCEL ; RD16/0011/0011 ), and the Comunidad de Madrid ( AvanCell , B2017/BMD-3692 ). The authors also thank Fundaci{\'o}n Bot{\'i}n for promoting translational research at the Hematopoietic Innovative Therapies Division of the CIEMAT. CIBERER is an initiative of the Instituto de Salud Carlos III and the Fondo Europeo de Desarrollo Regional (FEDER). Funding Information: The authors would like to thank Miguel A. Martin for the careful maintenance of NSG mice, and Aurora de la Cal, Mar?a del Carmen S?nchez, Soledad Moreno, Nadia Abu-sabha, Montserrat Aldea, and Sergio Losada for dedicated administrative help. This work was supported by grants from the Ministerio de Econom?a, Comercio y Competitividad y Fondo Europeo de Desarrollo Regional (FEDER) (SAF2017-84248-P), the Fondo de Investigaciones Sanitarias, Instituto de Salud Carlos III (Red TERCEL; RD16/0011/0011), and the Comunidad de Madrid (AvanCell, B2017/BMD-3692). The authors also thank Fundaci?n Bot?n for promoting translational research at the Hematopoietic Innovative Therapies Division of the CIEMAT. CIBERER is an initiative of the Instituto de Salud Carlos III and the Fondo Europeo de Desarrollo Regional (FEDER). Publisher Copyright: {\textcopyright} 2020 The Authors",

year = "2020",

month = dec,

day = "11",

doi = "10.1016/j.omtm.2020.10.007",

language = "English (US)",

volume = "19",

pages = "426--437",

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T1 - In Vitro and In Vivo Genetic Disease Modeling via NHEJ-Precise Deletions Using CRISPR-Cas9

AU - López-Manzaneda, Sergio

AU - Ojeda-Pérez, Isabel

AU - Zabaleta, Nerea

AU - García-Torralba, Aída

AU - Alberquilla, Omaira

AU - Torres, Raúl

AU - Sánchez-Domínguez, Rebeca

AU - Torella, Laura

AU - Olivier, Emmanuel

AU - Mountford, Joanne

AU - Ramírez, Juan C.

AU - Bueren, Juan A.

AU - González-Aseguinolaza, Gloria

AU - Segovia, Jose Carlos

N1 - Funding Information: The authors would like to thank Miguel A. Martin for the careful maintenance of NSG mice, and Aurora de la Cal, María del Carmen Sánchez, Soledad Moreno, Nadia Abu-sabha, Montserrat Aldea, and Sergio Losada for dedicated administrative help. This work was supported by grants from the Ministerio de Economía, Comercio y Competitividad y Fondo Europeo de Desarrollo Regional (FEDER) ( SAF2017-84248-P ), the Fondo de Investigaciones Sanitarias, Instituto de Salud Carlos III ( Red TERCEL ; RD16/0011/0011 ), and the Comunidad de Madrid ( AvanCell , B2017/BMD-3692 ). The authors also thank Fundación Botín for promoting translational research at the Hematopoietic Innovative Therapies Division of the CIEMAT. CIBERER is an initiative of the Instituto de Salud Carlos III and the Fondo Europeo de Desarrollo Regional (FEDER). Funding Information: The authors would like to thank Miguel A. Martin for the careful maintenance of NSG mice, and Aurora de la Cal, Mar?a del Carmen S?nchez, Soledad Moreno, Nadia Abu-sabha, Montserrat Aldea, and Sergio Losada for dedicated administrative help. This work was supported by grants from the Ministerio de Econom?a, Comercio y Competitividad y Fondo Europeo de Desarrollo Regional (FEDER) (SAF2017-84248-P), the Fondo de Investigaciones Sanitarias, Instituto de Salud Carlos III (Red TERCEL; RD16/0011/0011), and the Comunidad de Madrid (AvanCell, B2017/BMD-3692). The authors also thank Fundaci?n Bot?n for promoting translational research at the Hematopoietic Innovative Therapies Division of the CIEMAT. CIBERER is an initiative of the Instituto de Salud Carlos III and the Fondo Europeo de Desarrollo Regional (FEDER). Publisher Copyright: © 2020 The Authors

PY - 2020/12/11

Y1 - 2020/12/11

N2 - A CRISPR-Cas9 system generates precise deletions (up to 90% efficiency) when two guides are used simultaneously by means of non-homologous end joining. The PAM-in/PAM-in Cas9 orientation is the best combination to generate precise deletions. This gene-editing procedure facilitates the generation of homogeneously genetically defined cell and animal models.

AB - A CRISPR-Cas9 system generates precise deletions (up to 90% efficiency) when two guides are used simultaneously by means of non-homologous end joining. The PAM-in/PAM-in Cas9 orientation is the best combination to generate precise deletions. This gene-editing procedure facilitates the generation of homogeneously genetically defined cell and animal models.

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JO - Molecular Therapy - Methods and Clinical Development

JF - Molecular Therapy - Methods and Clinical Development

ER -

In Vitro and In Vivo Genetic Disease Modeling via NHEJ-Precise Deletions Using CRISPR-Cas9

Abstract

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