Systemic delivery of triplex-forming PNA and donor DNA by nanoparticles mediates site-specific genome editing of human hematopoietic cells in vivo-Reference-Cited by-同舟云学术

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Systemic delivery of triplex-forming PNA and donor DNA by nanoparticles mediates site-specific genome editing of human hematopoietic cells in vivo

Published:2012-10-18 Issue:6 Volume:20 Page:658-669
ISSN:0969-7128
Container-title:Gene Therapy
language:en
Short-container-title:Gene Ther

Author:

McNeer N A,Schleifman E B,Cuthbert A,Brehm M,Jackson A,Cheng C,Anandalingam K,Kumar P,Shultz L D,Greiner D L,Mark Saltzman W,Glazer P M

Publisher

Springer Science and Business Media LLC

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.nature.com/articles/gt201282.pdf

Reference37 articles.

1. Woods NB, Bottero V, Schmidt M, von Kalle C, Verma IM . Gene therapy: therapeutic gene causing lymphoma. Nature 2006; 440: 1123.

2. McNeer NA, Chin JY, Schleifman EB, Fields RJ, Glazer PM, Saltzman WM . Nanoparticles deliver triplex-forming PNAs for site-specific genomic recombination in CD34(+) human hematopoietic progenitors. Mol Ther 2011; 19: 172–180.

3. Holt N, Wang J, Kim K, Friedman G, Wang X, Taupin V et al. Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo. Nat Biotechnol 28: 839–847.

4. Maurisse R, De Semir D, Emamekhoo H, Bedayat B, Abdolmohammadi A, Parsi H et al. Comparative transfection of DNA into primary and transformed mammalian cells from different lineages. BMC Biotechnol 2010; 10: 9.

5. Vasquez KM, Narayanan L, Glazer PM . Specific mutations induced by triplex-forming oligonucleotides in mice. Science 2000; 290: 530–533.

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