Genetic treatment of a molecular disorder: gene therapy approaches to sickle cell disease von Hoban, Megan D., Orkin, Stuart H., Bauer, Daniel E.

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Analyzing CRISPR genome-editing experiments with CRISPResso von Pinello, Luca, Canver, Matthew C, Hoban, Megan D, Orkin, Stuart H, Kohn, Donald B, Bauer, Daniel E, Yuan, Guo-Cheng

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CRISPR/Cas9-Mediated Correction of the Sickle Mutation in Human CD34+ cells von Hoban, Megan D, Lumaquin, Dianne, Kuo, Caroline Y, Romero, Zulema, Long, Joseph, Ho, Michelle, Young, Courtney S, Mojadidi, Michelle, Fitz-Gibbon, Sorel, Cooper, Aaron R, Lill, Georgia R, Urbinati, Fabrizia, Campo-Fernandez, Beatriz, Bjurstrom, Carmen F, Pellegrini, Matteo, Hollis, Roger P, Kohn, Donald B

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Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells von Hoban, Megan D., Cost, Gregory J., Mendel, Matthew C., Romero, Zulema, Kaufman, Michael L., Joglekar, Alok V., Ho, Michelle, Lumaquin, Dianne, Gray, David, Lill, Georgia R., Cooper, Aaron R., Urbinati, Fabrizia, Senadheera, Shantha, Zhu, Allen, Liu, Pei-Qi, Paschon, David E., Zhang, Lei, Rebar, Edward J., Wilber, Andrew, Wang, Xiaoyan, Gregory, Philip D., Holmes, Michael C., Reik, Andreas, Hollis, Roger P., Kohn, Donald B.

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Site-Specific Gene Editing of Human Hematopoietic Stem Cells for X-Linked Hyper-IgM Syndrome von Kuo, Caroline Y., Long, Joseph D., Campo-Fernandez, Beatriz, de Oliveira, Satiro, Cooper, Aaron R., Romero, Zulema, Hoban, Megan D., Joglekar, Alok V., Lill, Georgia R., Kaufman, Michael L., Fitz-Gibbon, Sorel, Wang, Xiaoyan, Hollis, Roger P., Kohn, Donald B.

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Editing the Sickle Cell Disease Mutation in Human Hematopoietic Stem Cells: Comparison of Endonucleases and Homologous Donor Templates von Romero, Zulema, Lomova, Anastasia, Said, Suzanne, Miggelbrink, Alexandra, Kuo, Caroline Y., Campo-Fernandez, Beatriz, Hoban, Megan D., Masiuk, Katelyn E., Clark, Danielle N., Long, Joseph, Sanchez, Julie M., Velez, Miriam, Miyahira, Eric, Zhang, Ruixue, Brown, Devin, Wang, Xiaoyan, Kurmangaliyev, Yerbol Z., Hollis, Roger P., Kohn, Donald B.

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A genome editing primer for the hematologist von Hoban, Megan D., Bauer, Daniel E.

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Fertility-preserving myeloablative conditioning using single-dose CD117 antibody-drug conjugate in a rhesus gene therapy model von Uchida, Naoya, Stasula, Ulana, Demirci, Selami, Germino-Watnick, Paula, Hinds, Malikiya, Le, Anh, Chu, Rebecca, Berg, Alexander, Liu, Xiong, Su, Ling, Wu, Xiaolin, Krouse, Allen E., Linde, N. Seth, Bonifacino, Aylin, Hong, So Gun, Dunbar, Cynthia E., Lanieri, Leanne, Bhat, Anjali, Palchaudhuri, Rahul, Bennet, Bindu, Hoban, Megan, Bertelsen, Kirk, Olson, Lisa M., Donahue, Robert E., Tisdale, John F.

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Characterization of Gene Alterations following Editing of the β-Globin Gene Locus in Hematopoietic Stem/Progenitor Cells von Long, Joseph, Hoban, Megan D., Cooper, Aaron R., Kaufman, Michael L., Kuo, Caroline Y., Campo-Fernandez, Beatriz, Lumaquin, Dianne, Hollis, Roger P., Wang, Xiaoyan, Kohn, Donald B., Romero, Zulema

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Site Specific Gene Correction of Defects in CD40 Ligand Using the Crispr/Cas9 Genome Editing Platform von Kuo, Caroline Y., M.D, Hoban, Megan D., M.S, Joglekar, Alok V., Ph.D, Kohn, Donald B., M.D

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Dissecting the mechanism of histone deacetylase inhibitors to enhance the activity of zinc finger nucleases delivered by integrase-defective lentiviral vectors von Joglekar, Alok V, Stein, Libby, Ho, Michelle, Hoban, Megan D, Hollis, Roger P, Kohn, Donald B

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Delivery of Genome Editing Reagents to Hematopoietic Stem/Progenitor Cells von Hoban, Megan D., Romero, Zulema, Cost, Gregory J., Mendel, Matthew, Holmes, Michael, Kohn, Donald B.

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Identifying mechanisms that limit efficient site-specific gene modification by homology-directed repair in hematopoietic stem cells von Bjurström, Carmen F, Mojadidi, Michelle, Campo, Beatriz, Reik, Andreas, Holmes, Michael C, Cost, Greg, Gregory, Philip D, Hoban, Megan D, Hollis, Roger P, Kohn, Donald B

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The human ankyrin 1 promoter insulator sustains gene expression in a β-globin lentiviral vector in hematopoietic stem cells von Romero, Zulema, Campo-Fernandez, Beatriz, Wherley, Jennifer, Kaufman, Michael L, Urbinati, Fabrizia, Cooper, Aaron R, Hoban, Megan D, Baldwin, Kismet M, Lumaquin, Dianne, Wang, Xiaoyan, Senadheera, Shantha, Hollis, Roger P, Kohn, Donald B

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Ex Vivo Expansion with E478 Increases the Rate of CRISPR-Mediated Homology Directed Repair of the Beta-Globin Gene in Human Hematopoietic Stem Cells By >10-Fold and Improves In Viv... von Goncalves, Kevin A., Hoban, Megan D., Hyzy, Sharon L., George, Katia S., Boitano, Anthony E., Cooke, Michael P.

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CD117 Antibody-Drug Conjugate Conditioning Allows Efficient Engraftment of Gene-Modified CD34+ Cells with Fertility Preservation in a Rhesus Gene Therapy Model von Uchida, Naoya, Demirci, Selami, Le, Anh, Chu, Rebecca, Liu, Xiong, Su, Ling, Wu, Xiaolin, Krouse, Allen, Linde, N Seth, Bonifacino, Aylin, Hong, So Gun, Dunbar, Cynthia E., Lanieri, Leanne, Bhat, Anjali, Palchaudhuri, Rahul, Bennet, Bindu, Hoban, Megan D., Bertelsen, Kirk, Olson, Lisa, Donahue, Robert, Tisdale, John F.

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Aryl Hydrocarbon Receptor Antagonists Expand Adult Hematopoietic Stem Cells from Mobilized Peripheral Blood and Bone Marrow and Increase the Dose of CRISPR/Cas9 Gene-Edited NSG-Rep... von Hoban, Megan D., Goncalves, Kevin A., Proctor, Jennifer L., Adams, Hillary L., Hyzy, Sharon L., George, Katia S., Boitano, Anthony E., Cooke, Michael P.

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Phenotype Does Not Always Equal Function: HDAC Inhibitors and UM171, but Not SR1, Lead to Rapid Upregulation of CD90 on Non-Engrafting CD34+CD90-Negative Human Cells von Goncalves, Kevin A., Hoban, Megan D., Proctor, Jennifer L., Adams, Hillary L., Hyzy, Sharon L., Boitano, Anthony E., Cooke, Michael P.

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Non-Genotoxic Conditioning for Hematopoietic Stem Cell Transplant Using a Human Antibody Drug Conjugate Targeting C-KIT von Hartigan, Adam J., Pearse, Bradley R., McDonough, Sean M., Proctor, Jennifer L., Adams, Hillary L., McShea, Molly A., Hoban, Megan D., Panwar, Rajiv, Hyzy, Sharon L., Goncalves, Kevin A., Palchaudhuri, Rahul, Boitano, Anthony E., Cooke, Michael P.

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121. Targeted Gene Therapy in the Treatment of X-Linked Hyper IgM Syndrome von Kuo, Caroline Y., Hoban, Megan D., Joglekar, Alok V., Young, Courtney S., Kleinman, John, Abele, Alison N., Kohn, Donald B.

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