CRISPR-Cas9-AAV versus lentivector transduction for genome modification of X-linked severe combined immunodeficiency hematopoietic stem cells
Brault, Julie, Liu, Taylor, Liu, Siyuan, Lawson, Amanda, Choi, Uimook, Kozhushko, Nikita, Bzhilyanskaya, Vera, Pavel-Dinu, Mara, Meis, Ronald J, Eckhaus, Michael A, Burkett, Sandra S, Bosticardo, Marita, Kleinstiver, Benjamin P, Notarangelo, Luigi D, Lazzarotto, Cicera R, Tsai, Shengdar Q, Wu, Xiaolin, Dahl, Gary A, Porteus, Matthew H, Malech, Harry L, De Ravin, Suk See
Published in Frontiers in immunology (04.01.2023)
Published in Frontiers in immunology (04.01.2023)
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Enhanced homology-directed repair for highly efficient gene editing in hematopoietic stem/progenitor cells
De Ravin, Suk See, Brault, Julie, Meis, Ronald J., Liu, Siyuan, Li, Linhong, Pavel-Dinu, Mara, Lazzarotto, Cicera R., Liu, Taylor, Koontz, Sherry M., Choi, Uimook, Sweeney, Colin L., Theobald, Narda, Lee, GaHyun, Clark, Aaron B., Burkett, Sandra S., Kleinstiver, Benjamin P., Porteus, Matthew H., Tsai, Shengdar, Kuhns, Douglas B., Dahl, Gary A., Headey, Stephen, Wu, Xiaolin, Malech, Harry L.
Published in Blood (13.05.2021)
Published in Blood (13.05.2021)
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Correction of X-CGD patient HSPCs by targeted CYBB cDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repair
Sweeney, Colin L., Pavel-Dinu, Mara, Choi, Uimook, Brault, Julie, Liu, Taylor, Koontz, Sherry, Li, Linhong, Theobald, Narda, Lee, Janet, Bello, Ezekiel A., Wu, Xiaolin, Meis, Ronald J., Dahl, Gary A., Porteus, Matthew H., Malech, Harry L., De Ravin, Suk See
Published in Gene therapy (01.06.2021)
Published in Gene therapy (01.06.2021)
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CRISPR-targeted MAGT1 insertion restores XMEN patient hematopoietic stem cells and lymphocytes
Brault, Julie, Liu, Taylor, Bello, Ezekiel, Liu, Siyuan, Sweeney, Colin L., Meis, Ronald J., Koontz, Sherry, Corsino, Cristina, Choi, Uimook, Vayssiere, Guillaume, Bosticardo, Marita, Dowdell, Kennichi, Lazzarotto, Cicera R., Clark, Aaron B., Notarangelo, Luigi D., Ravell, Juan C., Lenardo, Michael J., Kleinstiver, Benjamin P., Tsai, Shengdar Q., Wu, Xiaolin, Dahl, Gary A., Malech, Harry L., De Ravin, Suk See
Published in Blood (30.12.2021)
Published in Blood (30.12.2021)
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High-fidelity PAMless base editing of hematopoietic stem cells to treat chronic granulomatous disease
Bzhilyanskaya, Vera, Ma, Linyuan, Liu, Siyuan, Fox, Lauren R, Whittaker, Madelynn N, Meis, Ronald J, Choi, Uimook, Lawson, Amanda, Ma, Michelle, Theobald, Narda, Burkett, Sandra, Sweeney, Colin L, Lazzarotto, Cicera R, Tsai, Shengdar Q, Lack, Justin B, Wu, Xiaolin, Dahl, Gary A, Malech, Harry L, Kleinstiver, Benjamin P, De Ravin, Suk See
Published in Science translational medicine (16.10.2024)
Published in Science translational medicine (16.10.2024)
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MAGT1 messenger RNA-corrected autologous T and natural killer cells for potential cell therapy in X-linked immunodeficiency with magnesium defect, Epstein-Barr virus infection and neoplasia disease
Brault, Julie, Meis, Ronald J., Li, Linhong, Bello, Ezekiel, Liu, Taylor, Sweeney, Colin L., Koontz, Sherry M., Dowdell, Kennichi, Theobald, Narda, Lee, Janet, Allen, Cornell, Clark, Aaron B., Ravell, Juan C., Lenardo, Michael J., Dahl, Gary A., Malech, Harry L., De Ravin, Suk See
Published in Cytotherapy (Oxford, England) (01.03.2021)
Published in Cytotherapy (Oxford, England) (01.03.2021)
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NADPH oxidase correction by mRNA transfection of apheresis granulocytes in chronic granulomatous disease
De Ravin, Suk See, Brault, Julie, Meis, Ronald J., Li, Linhong, Theobald, Narda, Bonifacino, Aylin C., Lei, Hong, Liu, Taylor Q., Koontz, Sherry, Corsino, Cristina, Zarakas, Marissa A., Desai, Jigar V., Clark, Aaron B., Choi, Uimook, Metzger, Mark E., West, Kamille, Highfill, Steven L., Kang, Elizabeth, Kuhns, Douglas B., Lionakis, Michail S., Stroncek, David F., Dunbar, Cynthia E., Tisdale, John F., Donahue, Robert E., Dahl, Gary A., Malech, Harry L.
Published in Blood advances (08.12.2020)
Published in Blood advances (08.12.2020)
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Extended Detection in Peripheral Blood Following Infusion of Chronic Granulomatous Disease Patient Autologous Granulocytes Corrected By mRNA Transfection in Patients with Chronic Granulomatous Disease
Bzhilyanskaya, Vera, Meis, Ronald J., Theobald, Narda, Estwick, Tyra, Lei, Hong, Highfill, Steven, Stroncek, David F., Chen, Leonard, West-Mitchell, Kamille, Dahl, Gary A., Malech, Harry L, De Ravin, Suk See, Brault, Julie
Published in Blood (15.11.2022)
Published in Blood (15.11.2022)
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Highly Efficient & Specific Repair of MAGT1 Mutation in Xmen Patient T Cells and Hematopoietic Stem Cells
Bzhilyanskaya, Vera, Brault, Julie, Liu, Siyuan, Zea Vera, Andres F., Kozhushko, Nikita, Lawson, Amanda, Choi, Uimook, Clark, Aaron B., Meis, Ronald J., Ma, Michelle, Lazzarotto, Cicera, Tsai, Shengdar, Wu, Xiaolin, Dahl, Gary A., Bergerson, Jenna, Freeman, Alexandra F., Kleinstiver, Benjamin, Malech, Harry L, De Ravin, Suk See
Published in Blood (15.11.2022)
Published in Blood (15.11.2022)
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Gene Editing and mRNA-Based Therapy: Two Complementary Therapeutic Approaches for the Treatment of Patients with Xmen Disease
Brault, Julie, Bello, Ezekiel, Meis, Ronald J., Liu, Taylor, Koontz, Sherry M., Dowdell, Kennichi, Li, Linhong, Allen, Cornell, Theobald, Narda, Lee, Janet, Clark, Aaron B., Ravell, Juan C., Lenardo, Michael, Dahl, Gary A., Malech, Harry L., De Ravin, Suk See
Published in Blood (13.11.2019)
Published in Blood (13.11.2019)
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Further extension of the opine concept: plasmids in Agrobacterium rhizogenes cooperate for opine degradation
Petit, A, David, C, Dahl, G.A, Ellis, J.G, Guyon, P
Published in Molecular & general genetics (01.05.1983)
Published in Molecular & general genetics (01.05.1983)
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