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Fludarabine increases nuclease-free AAV- and CRISPR/Cas9-mediated homologous recombination in mice.
Tsuji S, Stephens CJ, Bortolussi G, Zhang F, Baj G, Jang H, de Alencastro G, Muro AF, Pekrun K, Kay MA. Tsuji S, et al. Nat Biotechnol. 2022 Aug;40(8):1285-1294. doi: 10.1038/s41587-022-01240-2. Epub 2022 Apr 7. Nat Biotechnol. 2022. PMID: 35393561
Here, we tested a series of small-molecule compounds and found that ribonucleotide reductase (RNR) inhibitors substantially enhance AAV-HR efficiency in mouse and human liver cell lines approximately threefold. Short-term administration of the RNR inhibitor fludarabine inc …
Here, we tested a series of small-molecule compounds and found that ribonucleotide reductase (RNR) inhibitors substantially enhance AAV-HR e …
Long-term safety and efficacy of factor IX gene therapy in hemophilia B.
Nathwani AC, Reiss UM, Tuddenham EG, Rosales C, Chowdary P, McIntosh J, Della Peruta M, Lheriteau E, Patel N, Raj D, Riddell A, Pie J, Rangarajan S, Bevan D, Recht M, Shen YM, Halka KG, Basner-Tschakarjan E, Mingozzi F, High KA, Allay J, Kay MA, Ng CY, Zhou J, Cancio M, Morton CL, Gray JT, Srivastava D, Nienhuis AW, Davidoff AM. Nathwani AC, et al. N Engl J Med. 2014 Nov 20;371(21):1994-2004. doi: 10.1056/NEJMoa1407309. N Engl J Med. 2014. PMID: 25409372 Free PMC article. Clinical Trial.
METHODS: We evaluated the stability of transgene expression and long-term safety in 10 patients with severe hemophilia B: 6 patients who had been enrolled in an initial phase 1 dose-escalation trial, with 2 patients each receiving a low, intermediate, or high dose, and 4 a …
METHODS: We evaluated the stability of transgene expression and long-term safety in 10 patients with severe hemophilia B: 6 patients …
Organ size control is dominant over Rb family inactivation to restrict proliferation in vivo.
Ehmer U, Zmoos AF, Auerbach RK, Vaka D, Butte AJ, Kay MA, Sage J. Ehmer U, et al. Cell Rep. 2014 Jul 24;8(2):371-81. doi: 10.1016/j.celrep.2014.06.025. Epub 2014 Jul 10. Cell Rep. 2014. PMID: 25017070 Free PMC article.
Here, we show that, in contrast, deletion of the entire Rb gene family in mature hepatocytes is not sufficient for their long-term proliferation. The cell cycle block in Rb family mutant hepatocytes is independent of the Arf/p53/p21 checkpoint but can be abrogated upon dec …
Here, we show that, in contrast, deletion of the entire Rb gene family in mature hepatocytes is not sufficient for their long-term pr …
Ribosomal DNA integrating rAAV-rDNA vectors allow for stable transgene expression.
Lisowski L, Lau A, Wang Z, Zhang Y, Zhang F, Grompe M, Kay MA. Lisowski L, et al. Mol Ther. 2012 Oct;20(10):1912-23. doi: 10.1038/mt.2012.164. Epub 2012 Sep 18. Mol Ther. 2012. PMID: 22990671 Free PMC article.
Because of the relative safety of integration in the rDNA locus, these vectors expand the usage of rAAV for therapeutics requiring long-term gene transfer into dividing cells....
Because of the relative safety of integration in the rDNA locus, these vectors expand the usage of rAAV for therapeutics requiring long-t …
Prevention of spontaneous bleeding in dogs with haemophilia A and haemophilia B.
Nichols TC, Raymer RA, Franck HW, Merricks EP, Bellinger DA, DeFriess N, Margaritis P, Arruda VR, Kay MA, High KA. Nichols TC, et al. Haemophilia. 2010 May;16 Suppl 3(Suppl 3):19-23. doi: 10.1111/j.1365-2516.2010.02255.x. Haemophilia. 2010. PMID: 20586797 Free PMC article. Review.
The phenotypic and genotypic characteristics of haemophilic dogs have been well-described, and such dogs are suitable for testing prophylactic protein replacement therapy and gene transfer strategies. In dogs with haemophilia, long-term effects on spontaneous bleeding freq …
The phenotypic and genotypic characteristics of haemophilic dogs have been well-described, and such dogs are suitable for testing prophylact …