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Research ArticleGenome Editing
Efficient modification of CCR5 in primary human hematopoietic cells using a megaTAL nuclease and AAV donor template
- Blythe D. Sather1,*,
- Guillermo S. Romano Ibarra1,*,
- Karen Sommer1,
- Gabrielle Curinga1,
- Malika Hale1,
- Iram F. Khan1,
- Swati Singh1,
- Yumei Song1,
- Kamila Gwiazda1,
- Jaya Sahni1,
- Jordan Jarjour2,
- Alexander Astrakhan2,
- Thor A. Wagner3,4,
- Andrew M. Scharenberg1,4,5,† and
- David J. Rawlings1,4,5,†
- 1Center for Immunity and Immunotherapies and Program for Cell and Gene Therapy, Seattle Children’s Research Institute, Seattle, WA 98101, USA.
- 2Bluebird Bio, Seattle, WA 98102, USA.
- 3Center for Global Infectious Disease Research, Seattle Children’s Research Institute, Seattle, WA 98101, USA.
- 4Department of Pediatrics, University of Washington, Seattle, WA 98101, USA.
- 5Department of Immunology, University of Washington, Seattle, WA 98101, USA.
- ↵†Corresponding author. E-mail: andrewms{at}u.washington.edu (A.M.S.); drawling{at}uw.edu (D.J.R.)
↵* These authors contributed equally.
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Science Translational Medicine 30 Sep 2015:
Vol. 7, Issue 307, pp. 307ra156
DOI: 10.1126/scitranslmed.aac5530
Vol. 7, Issue 307, pp. 307ra156
DOI: 10.1126/scitranslmed.aac5530
Blythe D. Sather
1Center for Immunity and Immunotherapies and Program for Cell and Gene Therapy, Seattle Children’s Research Institute, Seattle, WA 98101, USA.
Guillermo S. Romano Ibarra
1Center for Immunity and Immunotherapies and Program for Cell and Gene Therapy, Seattle Children’s Research Institute, Seattle, WA 98101, USA.
Karen Sommer
1Center for Immunity and Immunotherapies and Program for Cell and Gene Therapy, Seattle Children’s Research Institute, Seattle, WA 98101, USA.
Gabrielle Curinga
1Center for Immunity and Immunotherapies and Program for Cell and Gene Therapy, Seattle Children’s Research Institute, Seattle, WA 98101, USA.
Malika Hale
1Center for Immunity and Immunotherapies and Program for Cell and Gene Therapy, Seattle Children’s Research Institute, Seattle, WA 98101, USA.
Iram F. Khan
1Center for Immunity and Immunotherapies and Program for Cell and Gene Therapy, Seattle Children’s Research Institute, Seattle, WA 98101, USA.
Swati Singh
1Center for Immunity and Immunotherapies and Program for Cell and Gene Therapy, Seattle Children’s Research Institute, Seattle, WA 98101, USA.
Yumei Song
1Center for Immunity and Immunotherapies and Program for Cell and Gene Therapy, Seattle Children’s Research Institute, Seattle, WA 98101, USA.
Kamila Gwiazda
1Center for Immunity and Immunotherapies and Program for Cell and Gene Therapy, Seattle Children’s Research Institute, Seattle, WA 98101, USA.
Jaya Sahni
1Center for Immunity and Immunotherapies and Program for Cell and Gene Therapy, Seattle Children’s Research Institute, Seattle, WA 98101, USA.
Jordan Jarjour
2Bluebird Bio, Seattle, WA 98102, USA.
Alexander Astrakhan
2Bluebird Bio, Seattle, WA 98102, USA.
Thor A. Wagner
3Center for Global Infectious Disease Research, Seattle Children’s Research Institute, Seattle, WA 98101, USA.
4Department of Pediatrics, University of Washington, Seattle, WA 98101, USA.
Andrew M. Scharenberg
1Center for Immunity and Immunotherapies and Program for Cell and Gene Therapy, Seattle Children’s Research Institute, Seattle, WA 98101, USA.
4Department of Pediatrics, University of Washington, Seattle, WA 98101, USA.
5Department of Immunology, University of Washington, Seattle, WA 98101, USA.
David J. Rawlings
1Center for Immunity and Immunotherapies and Program for Cell and Gene Therapy, Seattle Children’s Research Institute, Seattle, WA 98101, USA.
4Department of Pediatrics, University of Washington, Seattle, WA 98101, USA.
5Department of Immunology, University of Washington, Seattle, WA 98101, USA.
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