Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Comment
. 2015 Jan;23(1):4-6.
doi: 10.1038/mt.2014.230.

Hitting the target without pulling the trigger

Thierry VandenDriessche  1 , Marinee K Chuah  1
Affiliations
Comment

Hitting the target without pulling the trigger

Thierry VandenDriessche et al. Mol Ther. 2015 Jan.
No abstract available

PubMed Disclaimer

Figures

Figure 1
Figure 1
Nuclease-free adeno-associated virus (AAV)-mediated site-specific integration into the albumin locus. See text for details. cDNA, complementary DNA; FIX, coagulation factor IX; ssAAV8, single-stranded AAV serotype 8; UTR, untranslated region.
See this image and copyright information in PMC

Comment on

Similar articles

See all similar articles

Cited by

References

    1. Barzel A, Paulk NK, Shi Y, Huang Y, Chu K, Zhang F.et al. (2014Promoterless gene targeting without nucleases ameliorates haemophilia B in mice Naturee-pub ahead of print 29 October 2014. - PMC - PubMed
    1. Lombardo A, Cesana D, Genovese P, Di Stefano B, Provasi E, Colombo DF.et al. (2011Site-specific integration and tailoring of cassette design for sustainable gene transfer Nat Methods 8861–869. - PubMed
    1. Sadelain M, Papapetrou EP., and, Bushman FD. Safe harbours for the integration of new DNA in the human genome. Nat Rev Cancer . 2011;12:51–58. - PubMed
    1. Hacein-Bey-Abina S, Von Kalle C, Schmidt M, McCormack MP, Wulffraat N, Leboulch P.et al. (2003LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1 Science 302415–419. - PubMed
    1. Donsante A, Miller DG, Li Y, Vogler C, Brunt EM, Russell DW.et al. (2007AAV vector integration sites in mouse hepatocellular carcinoma Science 317477. - PubMed