المؤلفون: Sayitoglu, Ece Canan, Luca, Bogdan A., Boss, Allison Paige, Thomas, Benjamin Craig, Freeborn, Robert Arthur, Uyeda, Molly Javier^{Aff1, Aff3}, Chen, Pauline Ping, Nakauchi, Yusuke, Waichler, Colin, Lacayo, Norman, Bacchetta, Rosa^{Aff1, Aff5}, Majeti, Ravindra^{Aff3, Aff4, Aff6}, Gentles, Andrew J., Cepika, Alma-Martina^{Aff1, IDs41375024022551_cor14}, Roncarolo, Maria Grazia^{Aff1, Aff3, Aff5, IDs41375024022551_cor15}

المصدر: Leukemia. 38(6):1246-1255

المؤلفون: Bacchetta, Rosa, Roncarolo, Maria Grazia

المصدر: In The Journal of Allergy and Clinical Immunology March 2024 153(3):595-605

المؤلفون: Mochly-Rosen, Daria^Aff3, Grimes, Kevin^Aff3, Hannoush, Rami N.^Aff4, Koch, Bruce^Aff5, Ehrenkaufer, Gretchen^Aff6, Erlanson, Daniel A.^Aff7, Saiki, Julie^Aff8, Wilson, Jennifer L.^Aff9, Aldred, Shelley Force^Aff10, Garcia, Adriana A.^Aff6, Li, Jin Billy^Aff11, Bacchetta, Rosa^Aff12, Roncarolo, Maria Grazia^Aff12, Cepika, Alma-Martina^Aff12, Greenberg, Harry^Aff13, Goodman, Steven N.^Aff14, Kohn, Michael A.^Aff15

المساهمون: Mochly-Rosen, Daria, editor^Aff1, Grimes, Kevin, editor^Aff2

المصدر: A Practical Guide to Drug Development in Academia : The SPARK Approach. :33-100

المؤلفون: Sato, Yohei, Nathan, Abinaya, Shipp, Suzette, Wright, John Fraser, Tate, Keri Marie, Wani, Prachi, Roncarolo, Maria-Grazia, Bacchetta, Rosa

المصدر: In Molecular Therapy - Methods & Clinical Development 14 December 2023 31

المؤلفون: Ferrua, Francesca, Marangoni, Francesco, Aiuti, Alessandro, Roncarolo, Maria Grazia

المصدر: Journal of Allergy and Clinical Immunology. 146(2)

مصطلحات موضوعية: Biomedical and Clinical Sciences, Immunology, Genetic Therapy, Genetic Vectors, Hematopoietic Stem Cells, Humans, Male, Transplantation Conditioning, Wiskott-Aldrich Syndrome, Gene therapy, Wiskott-Aldrich syndrome, lentiviral vector, hematopoietic stem/progenitor cell, reduced-intensity conditioning, Allergy

وصف الملف: application/pdf

URL الوصول: https://escholarship.org/uc/item/4zn8f8bg

المؤلفون: Pavel-Dinu, Mara, Wiebking, Volker, Dejene, Beruh T, Srifa, Waracharee, Mantri, Sruthi, Nicolas, Carmencita E, Lee, Ciaran, Bao, Gang, Kildebeck, Eric J, Punjya, Niraj, Sindhu, Camille, Inlay, Matthew A, Saxena, Nivedita, DeRavin, Suk See, Malech, Harry, Roncarolo, Maria Grazia, Weinberg, Kenneth I, Porteus, Matthew H

المصدر: Nature communications. 10(1)

وصف الملف: application/pdf

URL الوصول: https://escholarship.org/uc/item/7bd8h6qn

المؤلفون: Pavel-Dinu, Mara, Wiebking, Volker, Dejene, Beruh T, Srifa, Waracharee, Mantri, Sruthi, Nicolas, Carmencita E, Lee, Ciaran, Bao, Gang, Kildebeck, Eric J, Punjya, Niraj, Sindhu, Camille, Inlay, Matthew A, Saxena, Nivedita, DeRavin, Suk See, Malech, Harry, Roncarolo, Maria Grazia, Weinberg, Kenneth I, Porteus, Matthew H

المصدر: Nature communications. 10(1)

مصطلحات موضوعية: Hematopoietic Stem Cells, Cell Line, Fetal Blood, Animals, Transplantation Chimera, Humans, Mice, Parvovirinae, DNA, Complementary, Codon, Initiator, Antigens, CD34, Hematopoietic Stem Cell Transplantation, Transplantation, Heterologous, Transduction, Genetic, Mutation, Genetic Vectors, Exons, Time Factors, Male, Interleukin Receptor Common gamma Subunit, X-Linked Combined Immunodeficiency Diseases, Primary Cell Culture, Healthy Volunteers, CRISPR-Cas Systems, Gene Editing, Transplantation, Stem Cell Research - Nonembryonic - Non-Human, Rare Diseases, Stem Cell Research, Hematology, Genetics, Biotechnology, Stem Cell Research - Nonembryonic - Human, Gene Therapy, Regenerative Medicine, 5.2 Cellular and gene therapies

وصف الملف: application/pdf

URL الوصول: https://escholarship.org/uc/item/2kf11376

المؤلفون: Wilkes, Mark C., Scanlon, Vanessa, Shibuya, Aya, Cepika, Alma-Martina, Eskin, Ascia, Chen, Zugen, Narla, Anupama, Glader, Bert, Roncarolo, Maria Grazia, Nelson, Stanley F., Sakamoto, Kathleen M.

المصدر: In Experimental Hematology July 2022 111:66-78

المؤلفون: Mochly-Rosen, Daria^Aff3, Grimes, Kevin^Aff3, Hannoush, Rami N.^Aff4, Koch, Bruce^Aff5, Ehrenkaufer, Gretchen^Aff3, Erlanson, Daniel A.^Aff6, Saiki, Julie^Aff7, Wilson, Jennifer L.^Aff8, Aldred, Shelley Force^Aff9, Garcia, Adriana A.^Aff10, Li, Jin Billy^Aff11, Bacchetta, Rosa^Aff12, Roncarolo, Maria Grazia^Aff12, Cepika, Alma-Martina^Aff12, Greenberg, Harry^Aff13, Goodman, Steven N.^Aff14, Kohn, Michael A.^Aff15

المساهمون: Mochly-Rosen, Daria, editor^Aff1, Grimes, Kevin, editor^Aff2

المصدر: A Practical Guide to Drug Development in Academia : The SPARK Approach. :C1-C1

المؤلفون: Roth, Theodore L, Puig-Saus, Cristina, Yu, Ruby, Shifrut, Eric, Carnevale, Julia, Li, P Jonathan, Hiatt, Joseph, Saco, Justin, Krystofinski, Paige, Li, Han, Tobin, Victoria, Nguyen, David N, Lee, Michael R, Putnam, Amy L, Ferris, Andrea L, Chen, Jeff W, Schickel, Jean-Nicolas, Pellerin, Laurence, Carmody, David, Alkorta-Aranburu, Gorka, Del Gaudio, Daniela, Matsumoto, Hiroyuki, Morell, Montse, Mao, Ying, Cho, Min, Quadros, Rolen M, Gurumurthy, Channabasavaiah B, Smith, Baz, Haugwitz, Michael, Hughes, Stephen H, Weissman, Jonathan S, Schumann, Kathrin, Esensten, Jonathan H, May, Andrew P, Ashworth, Alan, Kupfer, Gary M, Greeley, Siri Atma W, Bacchetta, Rosa, Meffre, Eric, Roncarolo, Maria Grazia, Romberg, Neil, Herold, Kevan C, Ribas, Antoni, Leonetti, Manuel D, Marson, Alexander

المصدر: Nature. 559(7714)

مصطلحات موضوعية: T-Lymphocytes, Cells, Cultured, Animals, Humans, Mice, Receptors, Antigen, T-Cell, Protein Engineering, Neoplasm Transplantation, Autoimmunity, Genome, Human, Male, Interleukin-2 Receptor alpha Subunit, CRISPR-Cas Systems, Cellular Reprogramming, Gene Editing, Human Genome, Biotechnology, Genetics, Gene Therapy, 5.2 Cellular and gene therapies, 5.1 Pharmaceuticals, Inflammatory and immune system, Cancer, General Science & Technology

وصف الملف: application/pdf

URL الوصول: https://escholarship.org/uc/item/5rx2j6kz