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Expert Opinion on Biological Therapy Volume 18, 2018 - Issue 11
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Review

Promise of gene therapy to treat sickle cell disease

Zulema RomeroDepartment of Microbiology, Immunology and Molecular Genetics, University of California Los Angeles, Los Angeles, CA, USAView further author information
,
Mark DeWittInnovative Genomics Initiative, University of California, Berkeley, CA, USAView further author information
&
Mark C. WaltersBlood and Marrow Transplantation Program, UCSF Benioff Children’s Hospital, Oakland, CA, USACorrespondence[email protected]
View further author information
Pages 1123-1136 | Received 26 Mar 2018, Accepted 10 Oct 2018, Published online: 19 Oct 2018

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Martin H Steinberg, Sara Kumar, George J. Murphy & Kim Vanuytsel. (2019) Sickle cell disease in the era of precision medicine: looking to the future. Expert Review of Precision Medicine and Drug Development 4:6, pages 357-367.
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Articles from other publishers (13)

Yogapriya Sundaresan, Sam Yacoub, Bindu Kodati, Charles E. Amankwa, Akash Raola & Gulab Zode. (2023) Therapeutic applications of CRISPR /Cas9 gene editing technology for the treatment of ocular diseases . The FEBS Journal 290:22, pages 5248-5269.
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Turgay Ulas & Mehmet Sinan Dal. (2023) Gene therapy approaches for sickle cell anemia. Transfusion and Apheresis Science 62:2, pages 103677.
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Bing Chen, Haochuan Liu, Zhengang Liu & Fan Yang. (2023) Benefits and limitations of humanized mouse models for human red blood cell-related disease research. Frontiers in Hematology 1.
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Mujeeb ur Rahman, Muhammad Bilal, Junaid Ali Shah, Ajeet Kaushik, Pierre-Louis Teissedre & Małgorzata Kujawska. (2022) CRISPR-Cas9-Based Technology and Its Relevance to Gene Editing in Parkinson’s Disease. Pharmaceutics 14:6, pages 1252.
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Wendy Magis, Mark A. DeWitt, Stacia K. Wyman, Jonathan T. Vu, Seok-Jin Heo, Shirley J. Shao, Finn Hennig, Zulema G. Romero, Beatriz Campo-Fernandez, Suzanne Said, Matthew S. McNeill, Garrett R. Rettig, Yongming Sun, Yu Wang, Mark A. Behlke, Donald B. Kohn, Dario Boffelli, Mark C. Walters, Jacob E. Corn & David I.K. Martin. (2022) High-level correction of the sickle mutation is amplified in vivo during erythroid differentiation. iScience 25:6, pages 104374.
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Mabirizi Vicent, Kawuma Simon & Safari Yonasi. (2022) An algorithm to detect overlapping red blood cells for sickle cell disease diagnosis. IET Image Processing 16:6, pages 1669-1677.
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Kristine E. Yoder, Anthony J. Rabe, Richard Fishel & Ross C. Larue. (2021) Strategies for Targeting Retroviral Integration for Safer Gene Therapy: Advances and Challenges. Frontiers in Molecular Biosciences 8.
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Sherif M. Badawy, Usman Beg, Robert I. Liem, Sonali Chaudhury & Alexis A. Thompson. (2021) A systematic review of quality of life in sickle cell disease and thalassemia after stem cell transplant or gene therapy. Blood Advances 5:2, pages 570-583.
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Yuncheng Man, Erdem Kucukal, Ran An, Quentin D. Watson, Jürgen Bosch, Peter A. Zimmerman, Jane A. Little & Umut A. Gurkan. (2020) Microfluidic assessment of red blood cell mediated microvascular occlusion. Lab on a Chip 20:12, pages 2086-2099.
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Bon Yip. (2020) Recent Advances in CRISPR/Cas9 Delivery Strategies. Biomolecules 10:6, pages 839.
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Beeke Wienert, David N. Nguyen, Alexis Guenther, Sharon J. Feng, Melissa N. Locke, Stacia K. Wyman, Jiyung Shin, Katelynn R. Kazane, Georgia L. Gregory, Matthew A. M. Carter, Francis Wright, Bruce R. Conklin, Alex Marson, Chris D. Richardson & Jacob E. Corn. (2020) Timed inhibition of CDC7 increases CRISPR-Cas9 mediated templated repair. Nature Communications 11:1.
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William H. Peranteau & Alan W. Flake. (2020) The Future of In Utero Gene Therapy. Molecular Diagnosis & Therapy 24:2, pages 135-142.
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Wendy Magis, Mark A. DeWitt, Stacia K. Wyman, Jonathan T. Vu, Seok-Jin Heo, Shirley J. Shao, Fiona Hennig, Zulema G. Romero, Beatriz Campo Fernandez, Suzanne Said, Matthew S. McNeill, Garrett R. Rettig, Yongming Sun, Yu Wang, Mark A. Behlke, Donald B. Donald, Dario Boffelli, Mark C. Walters, Jacob E. E Corn & David IK Martin. (2021) High-Level Correction of the Sickle Mutation is Amplified in Vivo During Erythroid Differentiation. SSRN Electronic Journal.
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