المؤلفون: 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

المصدر: Cell Reports. 23(9)

مصطلحات موضوعية: Biological Sciences, Stem Cell Research, Biotechnology, Stem Cell Research - Nonembryonic - Human, Pediatric, Regenerative Medicine, Hematology, Rare Diseases, Genetics, Stem Cell Research - Induced Pluripotent Stem Cell, Gene Therapy, 5.2 Cellular and gene therapies, 2.1 Biological and endogenous factors, Inflammatory and immune system, Animals, Antigens, CD34, Base Sequence, CD40 Ligand, CRISPR-Associated Protein 9, CRISPR-Cas Systems, Cell Differentiation, Cell Line, Colony-Forming Units Assay, DNA Repair, DNA, Complementary, Gene Editing, Genetic Diseases, X-Linked, Hematopoietic Stem Cells, Humans, Hyper-IgM Immunodeficiency Syndrome, Mice, T-Lymphocytes, Transcription Activator-Like Effector Nucleases, CD40 ligand, CRISPR/Cas9, TALEN, X-linked hyper-IgM syndrome, gene editing, gene therapy, hematopoietic stem cell, primary immunodeficiency, Biochemistry and Cell Biology, Medical Physiology, Biological sciences

وصف الملف: application/pdf

URL الوصول: https://escholarship.org/uc/item/2x34q4m2
https://escholarship.org/content/qt2x34q4m2/qt2x34q4m2.pdf

المؤلفون: 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

المصدر: Molecular Therapy. 24(9)

مصطلحات موضوعية: Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Rare Diseases, Genetics, Regenerative Medicine, Biotechnology, Stem Cell Research, Gene Therapy, Sickle Cell Disease, Hematology, Stem Cell Research - Nonembryonic - Human, 5.2 Cellular and gene therapies, 1.1 Normal biological development and functioning, Blood, Anemia, Sickle Cell, Base Sequence, CRISPR-Cas Systems, Cell Line, DNA Cleavage, Gene Editing, Gene Targeting, Genetic Loci, Hematopoietic Stem Cells, Humans, Mutation, Protein Binding, RNA, Guide, CRISPR-Cas Systems, Targeted Gene Repair, Transcription Activator-Like Effector Nucleases, beta-Globins, Biological Sciences, Technology, Medical and Health Sciences, Clinical sciences, Medical biotechnology

وصف الملف: application/pdf

URL الوصول: https://escholarship.org/uc/item/7bm349wp
https://escholarship.org/content/qt7bm349wp/qt7bm349wp.pdf

المؤلفون: Chin, Chee Jia, Cooper, Aaron R, Lill, Georgia R, Evseenko, Denis, Zhu, Yuhua, Bin He, Chong, Casero, David, Pellegrini, Matteo, Kohn, Donald B, Crooks, Gay M

المصدر: Stem Cells. 34(5)

مصطلحات موضوعية: Medical Biotechnology, Biomedical and Clinical Sciences, Genetics, Regenerative Medicine, Stem Cell Research - Nonembryonic - Non-Human, Stem Cell Research - Induced Pluripotent Stem Cell, Stem Cell Research - Embryonic - Human, Stem Cell Research, Stem Cell Research - Nonembryonic - Human, Stem Cell Research - Induced Pluripotent Stem Cell - Human, 1.1 Normal biological development and functioning, Animals, Antigens, CD, Cell Differentiation, Cell Line, Cell Lineage, Cell Separation, Clone Cells, Flow Cytometry, Genetic Techniques, Humans, Lentivirus, Mesoderm, Mice, Multipotent Stem Cells, Pluripotent stem cells, Vector integration site analysis, Lineage tracing, Clonal tracking, Lentiviral vectors, Hematopoiesis, Biological Sciences, Technology, Medical and Health Sciences, Immunology, Biological sciences, Biomedical and clinical sciences

وصف الملف: application/pdf

URL الوصول: https://escholarship.org/uc/item/8s5434wj
https://escholarship.org/content/qt8s5434wj/qt8s5434wj.pdf

المؤلفون: Bjurström, Carmen F, Mojadidi, Michelle, Phillips, John, Kuo, Caroline, Lai, Stephen, Lill, Georgia R, Cooper, Aaron, Kaufman, Michael, Urbinati, Fabrizia, Wang, Xiaoyan, Hollis, Roger P, Kohn, Donald B

المصدر: Molecular Therapy - Nucleic Acids. 5(8)

مصطلحات موضوعية: Biochemistry and Cell Biology, Biological Sciences, Stem Cell Research - Nonembryonic - Human, Human Genome, Regenerative Medicine, Hematology, Stem Cell Research, Biotechnology, Genetics, Cancer, BCL11A, CRISPR/Cas9, TALENs, fetal hemoglobin, nonhomologous end joining, zinc finger nucleases, Clinical Sciences, Biochemistry and cell biology

وصف الملف: application/pdf

URL الوصول: https://escholarship.org/uc/item/533142xv
https://escholarship.org/content/qt533142xv/qt533142xv.pdf

المؤلفون: Cooper, Aaron R, Lill, Georgia R, Gschweng, Eric H, Kohn, Donald B

المصدر: Nucleic Acids Research. 43(1)

مصطلحات موضوعية: Biological Sciences, Environmental Sciences, Chemical Sciences, Genetics, Biotechnology, Gene Therapy, Prevention, Enhancer Elements, Genetic, Exons, Gene Expression, Genetic Vectors, HEK293 Cells, Humans, Introns, K562 Cells, Lentivirus, Peptide Elongation Factor 1, Promoter Regions, Genetic, RNA Splicing, Ubiquitin C, Information and Computing Sciences, Developmental Biology, Biological sciences, Chemical sciences, Environmental sciences

وصف الملف: application/pdf

URL الوصول: https://escholarship.org/uc/item/11j9p3rz
https://escholarship.org/content/qt11j9p3rz/qt11j9p3rz.pdf

المؤلفون: Cooper, Aaron R., Shaw, Kit L., Lill, Georgia R., Sokolic, Robert, Candotti, Fabio, Kohn, Donald B.

المصدر: Molecular Therapy ; volume 23, page S94 ; ISSN 1525-0016

الاتاحة: http://dx.doi.org/10.1016/s1525-0016(16)33845-x
https://api.elsevier.com/content/article/PII:S152500161633845X?httpAccept=text/xml
https://api.elsevier.com/content/article/PII:S152500161633845X?httpAccept=text/plain

المؤلفون: Hoban, Megan D., 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., Cost, Gregory J.

المصدر: Molecular Therapy ; volume 23, page S48 ; ISSN 1525-0016

الاتاحة: http://dx.doi.org/10.1016/s1525-0016(16)33720-0
https://api.elsevier.com/content/article/PII:S1525001616337200?httpAccept=text/xml
https://api.elsevier.com/content/article/PII:S1525001616337200?httpAccept=text/plain

المؤلفون: Cooper, Aaron R.^1,2, Lill, Georgia R.¹, Shaw, Kit¹, Carbonaro-Sarracino, Denise A.¹, Davila, Alejandra¹, Sokolic, Robert³, Candotti, Fabio³, Pellegrini, Matteo⁴, Kohn, Donald B.^1,5,6 dkohn1@mednet.ucla.edu

المصدر: Blood. 5/11/2017, Vol. 129 Issue 19, p2624-2635. 12p.

مصطلحات موضوعية: *SEVERE combined immunodeficiency, *ADENOSINE deaminase deficiency, *GENE therapy, *RETROVIRUSES, *CANCER chemotherapy, *LEUKEMIA risk factors, *T cell receptors, *GENETIC toxicology, *THERAPEUTICS

المؤلفون: 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.

المصدر: Blood; April 2015, Vol. 125 Issue: 17 p2597-2604, 8p

المؤلفون: Bjurström, Carmen Flores, Mojadidi, Michelle, Lomova, Anastasia, Lai, Stephen, Fitz-Gibbon, Sorel, Pellegrini, Matteo, Cooper, Aaron R, Lill, Georgia R, Kaufman, Michael, Cost, Gregory J, Holmes, Michael C., Hollis, Roger P, Kohn, Donald B

المصدر: Blood; December 2016, Vol. 128 Issue: 22 p3636-3636, 1p