Gonçalves MAFV - Search Results

1

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

Zittersteijn HA, Gonçalves MAFV, Hoeben RC. Zittersteijn HA, et al. Among authors: goncalves mafv. J Inherit Metab Dis. 2021 Jan;44(1):54-71. doi: 10.1002/jimd.12270. Epub 2020 Jul 20. J Inherit Metab Dis. 2021. PMID: 32510617 Free PMC article. Review.

2

A concise peer into the background, initial thoughts and practices of human gene therapy.

Gonçalves MA. Gonçalves MA. Bioessays. 2005 May;27(5):506-17. doi: 10.1002/bies.20218. Bioessays. 2005. PMID: 15832383 Review.

3

Adenoviral vectors stimulate glucagon transcription in human mesenchymal stem cells expressing pancreatic transcription factors.

Zaldumbide A, Carlotti F, Gonçalves MA, Knaän-Shanzer S, Cramer SJ, Roep BO, Wiertz EJ, Hoeben RC. Zaldumbide A, et al. PLoS One. 2012;7(10):e48093. doi: 10.1371/journal.pone.0048093. Epub 2012 Oct 26. PLoS One. 2012. PMID: 23110179 Free PMC article.

4

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

Maggio I, Holkers M, Liu J, Janssen JM, Chen X, Gonçalves MA. Maggio I, et al. Sci Rep. 2014 May 29;4:5105. doi: 10.1038/srep05105. Sci Rep. 2014. PMID: 24870050 Free PMC article.

5

Engineered Viruses as Genome Editing Devices.

Chen X, Gonçalves MA. Chen X, et al. Mol Ther. 2016 Mar;24(3):447-57. doi: 10.1038/mt.2015.164. Epub 2015 Sep 4. Mol Ther. 2016. PMID: 26336974 Free PMC article. Review.

6

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

Maggio I, Stefanucci L, Janssen JM, Liu J, Chen X, Mouly V, Gonçalves MA. Maggio I, et al. Nucleic Acids Res. 2016 Feb 18;44(3):1449-70. doi: 10.1093/nar/gkv1540. Epub 2016 Jan 13. Nucleic Acids Res. 2016. PMID: 26762977 Free PMC article.

7

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

Maggio I, Chen X, Gonçalves MA. Maggio I, et al. Genome Med. 2016 May 23;8(1):59. doi: 10.1186/s13073-016-0316-x. Genome Med. 2016. PMID: 27215286 Free PMC article. Review.

8

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

Maggio I, Liu J, Janssen JM, Chen X, Gonçalves MA. Maggio I, et al. Sci Rep. 2016 Nov 15;6:37051. doi: 10.1038/srep37051. Sci Rep. 2016. PMID: 27845387 Free PMC article.

9

In trans paired nicking triggers seamless genome editing without double-stranded DNA cutting.

Chen X, Janssen JM, Liu J, Maggio I, 't Jong AEJ, Mikkers HMM, Gonçalves MAFV. Chen X, et al. Among authors: goncalves mafv. Nat Commun. 2017 Sep 22;8(1):657. doi: 10.1038/s41467-017-00687-1. Nat Commun. 2017. PMID: 28939824 Free PMC article.

10

Intronic SMCHD1 variants in FSHD: testing the potential for CRISPR-Cas9 genome editing.

Goossens R, van den Boogaard ML, Lemmers RJLF, Balog J, van der Vliet PJ, Willemsen IM, Schouten J, Maggio I, van der Stoep N, Hoeben RC, Tapscott SJ, Geijsen N, Gonçalves MAFV, Sacconi S, Tawil R, van der Maarel SM. Goossens R, et al. Among authors: goncalves mafv. J Med Genet. 2019 Dec;56(12):828-837. doi: 10.1136/jmedgenet-2019-106402. Epub 2019 Nov 1. J Med Genet. 2019. PMID: 31676591

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