Concepedia

Promoterless gene targeting without nucleases ameliorates haemophilia B in mice

Adi Barzel, Nicole K. Paulk, Yun Shi, Yuanhua Huang, K. Chu, F. Zhang, Paul N. Valdmanis, Laura Spector, Matthew H. Porteus, K M Gaensler,

Nature · 2014 · 262 citations · 30 references

DOI

Concepts

References

30

High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells

Yanfang Fu, Jennifer A Foden, Cyd Khayter et al. · Nature Biotechnology · 2013 · 3.3K citations · Full text

, , +16

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

Amit C. Nathwani, Edward G. D. Tuddenham, Savita Rangarajan et al. · New England Journal of Medicine · 2011 · 1.8K citations · Full text

, , +13

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

Salima Hacein‐Bey‐Abina, Alexandrine Garrigue, Gary P. Wang et al. · Journal of Clinical Investigation · 2008 · 1.8K citations · Full text

, , +14

Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways

Dirk Grimm, Konrad L. Streetz, Catherine L. Jopling et al. · Nature · 2006 · 1.7K citations

, , +8

High Cleavage Efficiency of a 2A Peptide Derived from Porcine Teschovirus-1 in Human Cell Lines, Zebrafish and Mice

Jin Hee Kim, Sang Rok Lee, Li-Hua Li et al. · PLoS ONE · 2011 · 1.4K citations · Full text

, , +14