Abstract

The Cell and Virus Core is designed to support two major activities of the NGEC: cell separations and analysis, and construction and production of self inactivating non-integrating lentiviral (NIL) vectors and viral stocks. AutoMACs and flow cytometry and sorting will be heavily utilized in Components 2-5 for LHE design, and in Components 6-8 for analysis of fluorescent reporter assays and immune function in in vivo gene targeting and repair experiments. NIL vectors will be used for delivery of LHE's and donor templates in selected experiments from Component 1, and in virtually all experiments proposed in Components 6-8.

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
National Heart, Lung, and Blood Institute (NHLBI)
Type
Linked Center Core Grant (PL1)
Project #
5PL1HL092557-05
Application #
8121472
Study Section
Special Emphasis Panel (ZRR1-SRC (99))
Program Officer
Thomas, John
Project Start
2007-09-30
Project End
2013-06-30
Budget Start
2011-07-01
Budget End
2013-06-30
Support Year
5
Fiscal Year
2011
Total Cost
$845,477
Indirect Cost

  Institution

Name
Seattle Children's Hospital
Department
Type
DUNS #
048682157
City
Seattle
State
WA
Country
United States
Zip Code
98105

  Publications

Romano Ibarra, Guillermo S; Paul, Biswajit; Sather, Blythe D et al. (2016) Efficient Modification of the CCR5 Locus in Primary Human T Cells With megaTAL Nuclease Establishes HIV-1 Resistance. Mol Ther Nucleic Acids 5:e352
Wang, Xuefeng; Shin, Simon C; Chiang, Andy F J et al. (2015) Intraosseous delivery of lentiviral vectors targeting factor VIII expression in platelets corrects murine hemophilia A. Mol Ther 23:617-26
Sather, Blythe D; Romano Ibarra, Guillermo S; Sommer, Karen et al. (2015) Efficient modification of CCR5 in primary human hematopoietic cells using a megaTAL nuclease and AAV donor template. Sci Transl Med 7:307ra156
Wang, Cathy X; Sather, Blythe D; Wang, Xuefeng et al. (2014) Rapamycin relieves lentiviral vector transduction resistance in human and mouse hematopoietic stem cells. Blood 124:913-23
Wang, Yupeng; Khan, Iram F; Boissel, Sandrine et al. (2014) Progressive engineering of a homing endonuclease genome editing reagent for the murine X-linked immunodeficiency locus. Nucleic Acids Res 42:6463-75
Boissel, Sandrine; Jarjour, Jordan; Astrakhan, Alexander et al. (2014) megaTALs: a rare-cleaving nuclease architecture for therapeutic genome engineering. Nucleic Acids Res 42:2591-601
Kuhar, Ryan; Gwiazda, Kamila S; Humbert, Olivier et al. (2014) Novel fluorescent genome editing reporters for monitoring DNA repair pathway utilization at endonuclease-induced breaks. Nucleic Acids Res 42:e4
Certo, Michael T; Gwiazda, Kamila S; Kuhar, Ryan et al. (2012) Coupling endonucleases with DNA end-processing enzymes to drive gene disruption. Nat Methods 9:973-5
Certo, Michael T; Ryu, Byoung Y; Annis, James E et al. (2011) Tracking genome engineering outcome at individual DNA breakpoints. Nat Methods 8:671-6
Aubert, Martine; Ryu, Byoung Y; Banks, Lindsey et al. (2011) Successful targeting and disruption of an integrated reporter lentivirus using the engineered homing endonuclease Y2 I-AniI. PLoS One 6:e16825

Showing the most recent 10 out of 12 publications