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FiCAT gene editing: allowing genes to be efficiently transferred without size restrictions

 Last Update: 2021-12-29
 Source: Internet
 Author: User

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From left to right: Marc Güell, Dimitrije Ivani, Avencia Sánchez-Mejías and Maria Pallarès

An international multidisciplinary research team from the Translational Synthetic Biology Laboratory of the University of Pompei Fabra in Barcelona, Spain, led by Dr.

UPF Translational Synthetic Biology Laboratory has been committed to gene editing and synthetic biology applied to gene therapy since 2017

"With the development of new editing tools, human genome engineering has made significant progress in the past decade, but there is still a technological gap-how to allow therapeutic genes to be effectively transferred without size restrictions

In this work, the researchers developed an efficient and precise programmable gene editing technology based on the combination of the modified protein CRISPR-cas and porcine Bac transposase (PB).

"In this way, FiCAT technology allows us to precisely insert large pieces of DNA into the genome

The researchers tested the technology in human and mouse cell lines, and the study proved that the proportion of off-target insertion was very small, and the efficiency reached 5-22%, and demonstrated targeted gene transfer in the mouse liver and germ cells of the mouse model.

"We have been modifying enzymes so that they obtain the functions we are looking for, and choosing those with better functions

UPF transferred FiCAT technology through its subsidiary Integra Therapeutics

Reference article:

Pallarès-Masmitjà, M; Ivan?i?, D; Mir-Pedrol, J; Jaraba-Wallace, J; Tagliani, T; Oliva, B; Sánchez-Mejías, A; Güell, M.

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