AAV-vectored base editor trans-splicing delivers dystrophin repair

por Li, Zhen, Gonçalves, Manuel A.F.V.
Publicado 2023

Engineered Viruses as Genome Editing Devices

por Chen, Xiaoyu, Gonçalves, Manuel A F V
Publicado 2016

DNA, RNA, and Protein Tools for Editing the Genetic Information in Human Cells

por Chen, Xiaoyu, Gonçalves, Manuel A.F.V.
Publicado 2018

The emerging role of viral vectors as vehicles for DMD gene editing

por Maggio, Ignazio, Chen, Xiaoyu, Gonçalves, Manuel A. F. V.
Publicado 2016

Adenoviral Vectors Meet Gene Editing: A Rising Partnership for the Genomic Engineering of Human Stem Cells and Their Progeny

por Tasca, Francesca, Wang, Qian, Gonçalves, Manuel A.F.V.
Publicado 2020

Novel Therapeutic Approaches for the Treatment of Retinal Degenerative Diseases: Focus on CRISPR/Cas-Based Gene Editing

por Gallego, Carmen, Gonçalves, Manuel A. F. V., Wijnholds, Jan
Publicado 2020

A primer to gene therapy: Progress, prospects, and problems

por Zittersteijn, Hidde A., Gonçalves, Manuel A.F.V., Hoeben, Rob C.
Publicado 2020

Precise homology-directed installation of large genomic edits in human cells with cleaving and nicking high-specificity Cas9 variants

por Wang, Qian, Liu, Jin, Janssen, Josephine M, Gonçalves, Manuel A F V
Publicado 2023

Nonspaced inverted DNA repeats are preferential targets for homology-directed gene repair in mammalian cells

por Holkers, Maarten, de Vries, Antoine A. F., Gonçalves, Manuel A. F. V.
Publicado 2012

The Chromatin Structure Differentially Impacts High-Specificity CRISPR-Cas9 Nuclease Strategies

por Chen, Xiaoyu, Liu, Jin, Janssen, Josephine M., Gonçalves, Manuel A.F.V.
Publicado 2017

The Chromatin Structure of CRISPR-Cas9 Target DNA Controls the Balance between Mutagenic and Homology-Directed Gene-Editing Events

por Janssen, Josephine M., Chen, Xiaoyu, Liu, Jin, Gonçalves, Manuel A.F.V.
Publicado 2019

High-Capacity Adenoviral Vectors Permit Robust and Versatile Testing of DMD Gene Repair Tools and Strategies in Human Cells

por Brescia, Marcella, Janssen, Josephine M., Liu, Jin, Gonçalves, Manuel A. F. V.
Publicado 2020

Adenoviral vectors encoding CRISPR/Cas9 multiplexes rescue dystrophin synthesis in unselected populations of DMD muscle cells

por Maggio, Ignazio, Liu, Jin, Janssen, Josephine M., Chen, Xiaoyu, Gonçalves, Manuel A. F. V.
Publicado 2016

Rapid and Sensitive Lentivirus Vector-Based Conditional Gene Expression Assay to Monitor and Quantify Cell Fusion Activity

por Gonçalves, Manuel A. F. V., Janssen, Josephine M., Holkers, Maarten, de Vries, Antoine A. F.
Publicado 2010

High-capacity adenovector delivery of forced CRISPR-Cas9 heterodimers fosters precise chromosomal deletions in human cells

por Tasca, Francesca, Brescia, Marcella, Liu, Jin, Janssen, Josephine M., Mamchaoui, Kamel, Gonçalves, Manuel A.F.V.
Publicado 2023

Concerted nicking of donor and chromosomal acceptor DNA promotes homology-directed gene targeting in human cells

por Gonçalves, Manuel A. F. V., van Nierop, Gijsbert P., Holkers, Maarten, de Vries, Antoine A. F.
Publicado 2012

Adenoviral vector delivery of RNA-guided CRISPR/Cas9 nuclease complexes induces targeted mutagenesis in a diverse array of human cells

por Maggio, Ignazio, Holkers, Maarten, Liu, Jin, Janssen, Josephine M., Chen, Xiaoyu, Gonçalves, Manuel A. F. V.
Publicado 2014

Probing the impact of chromatin conformation on genome editing tools

por Chen, Xiaoyu, Rinsma, Marrit, Janssen, Josephine M., Liu, Jin, Maggio, Ignazio, Gonçalves, Manuel A.F.V.
Publicado 2016

Broadening the reach and investigating the potential of prime editors through fully viral gene-deleted adenoviral vector delivery

por Wang, Qian, Liu, Jin, Janssen, Josephine M, Tasca, Francesca, Mei, Hailiang, Gonçalves, Manuel A F V
Publicado 2021

Selection-free gene repair after adenoviral vector transduction of designer nucleases: rescue of dystrophin synthesis in DMD muscle cell populations

por Maggio, Ignazio, Stefanucci, Luca, Janssen, Josephine M., Liu, Jin, Chen, Xiaoyu, Mouly, Vincent, Gonçalves, Manuel A.F.V.
Publicado 2016