Efficient modification of CCR5 in primary human hematopoietic cells using a megaTAL nuclease and AAV donor template

Blythe D. Sather; Guillermo S. Romano Ibarra; Karen Sommer; Gabrielle Curinga; Malika Hale; Iram F. Khan; Swati Singh; Yumei Song; Kamila Gwiazda; Jaya Sahni; Jordan Jarjour; Alexander Astrakhan; Thor A. Wagner; Andrew M. Scharenberg; David J. Rawlings

doi:10.1126/scitranslmed.aac5530

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

Blythe D. Sather

Center for Immunity and Immunotherapies and Program for Cell and Gene Therapy, Seattle Children’s Research Institute, Seattle, WA 98101, USA.

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Guillermo S. Romano Ibarra

Center for Immunity and Immunotherapies and Program for Cell and Gene Therapy, Seattle Children’s Research Institute, Seattle, WA 98101, USA.

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Karen Sommer

Center for Immunity and Immunotherapies and Program for Cell and Gene Therapy, Seattle Children’s Research Institute, Seattle, WA 98101, USA.

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Gabrielle Curinga

Center for Immunity and Immunotherapies and Program for Cell and Gene Therapy, Seattle Children’s Research Institute, Seattle, WA 98101, USA.

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Malika Hale

Center for Immunity and Immunotherapies and Program for Cell and Gene Therapy, Seattle Children’s Research Institute, Seattle, WA 98101, USA.

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Iram F. Khan

Center for Immunity and Immunotherapies and Program for Cell and Gene Therapy, Seattle Children’s Research Institute, Seattle, WA 98101, USA.

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Swati Singh

Center for Immunity and Immunotherapies and Program for Cell and Gene Therapy, Seattle Children’s Research Institute, Seattle, WA 98101, USA.

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Yumei Song

Center for Immunity and Immunotherapies and Program for Cell and Gene Therapy, Seattle Children’s Research Institute, Seattle, WA 98101, USA.

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Kamila Gwiazda

Center for Immunity and Immunotherapies and Program for Cell and Gene Therapy, Seattle Children’s Research Institute, Seattle, WA 98101, USA.

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Jaya Sahni

Center for Immunity and Immunotherapies and Program for Cell and Gene Therapy, Seattle Children’s Research Institute, Seattle, WA 98101, USA.

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Jordan Jarjour

Bluebird Bio, Seattle, WA 98102, USA.

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Alexander Astrakhan

Bluebird Bio, Seattle, WA 98102, USA.

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Thor A. Wagner

Center for Global Infectious Disease Research, Seattle Children’s Research Institute, Seattle, WA 98101, USA.

Department of Pediatrics, University of Washington, Seattle, WA 98101, USA.

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Andrew M. Scharenberg

Center for Immunity and Immunotherapies and Program for Cell and Gene Therapy, Seattle Children’s Research Institute, Seattle, WA 98101, USA.

Department of Pediatrics, University of Washington, Seattle, WA 98101, USA.

Department of Immunology, University of Washington, Seattle, WA 98101, USA.

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For correspondence: andrewms@u.washington.edu drawling@uw.edu

David J. Rawlings

Center for Immunity and Immunotherapies and Program for Cell and Gene Therapy, Seattle Children’s Research Institute, Seattle, WA 98101, USA.

Department of Pediatrics, University of Washington, Seattle, WA 98101, USA.

Department of Immunology, University of Washington, Seattle, WA 98101, USA.

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For correspondence: andrewms@u.washington.edu drawling@uw.edu

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Therapeutic coding sequences can be targeted to the CCR5 locus of primary human T cells with high efficiency by using megaTAL nuclease and an AAV donor template.

Efficient modification of CCR5 in primary human hematopoietic cells using a megaTAL nuclease and AAV donor template

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