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 journalArticlepeer-review

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 languageEnglish (US)
Pages (from-to)426-437
Number of pages12
JournalMolecular Therapy - Methods and Clinical Development
Volume19
DOIs
StatePublished - Dec 11 2020
Externally publishedYes

ASJC Scopus subject areas

  • Molecular Medicine
  • Molecular Biology
  • Genetics

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