المؤلفون: Kanter, Julie, Thompson, Alexis A, Pierciey, Francis J, Hsieh, Matthew, Uchida, Naoya, Leboulch, Philippe, Schmidt, Manfred, Bonner, Melissa, Guo, Ruiting, Miller, Alex, Ribeil, Jean‐Antoine, Davidson, David, Asmal, Mohammed, Walters, Mark C, Tisdale, John F

المصدر: American Journal of Hematology. 98(1)

مصطلحات موضوعية: Medical Biotechnology, Biomedical and Clinical Sciences, Genetics, Clinical Trials and Supportive Activities, Sickle Cell Disease, Stem Cell Research, Hematology, Rare Diseases, Clinical Research, Gene Therapy, Humans, Lentivirus, Hematopoietic Stem Cell Transplantation, Anemia, Sickle Cell, Genetic Therapy, Hemoglobins, Cardiorespiratory Medicine and Haematology, Immunology, Cardiovascular medicine and haematology

وصف الملف: application/pdf

URL الوصول: https://escholarship.org/uc/item/78x282hm

المؤلفون: Thompson, Alexis A, Walters, Mark C, Kwiatkowski, Janet, Rasko, John EJ, Ribeil, Jean-Antoine, Hongeng, Suradej, Magrin, Elisa, Schiller, Gary J, Payen, Emmanuel, Semeraro, Michaela, Moshous, Despina, Lefrere, Francois, Puy, Hervé, Bourget, Philippe, Magnani, Alessandra, Caccavelli, Laure, Diana, Jean-Sébastien, Suarez, Felipe, Monpoux, Fabrice, Brousse, Valentine, Poirot, Catherine, Brouzes, Chantal, Meritet, Jean-François, Pondarré, Corinne, Beuzard, Yves, Chrétien, Stany, Lefebvre, Thibaud, Teachey, David T, Anurathapan, Usanarat, Ho, P Joy, von Kalle, Christof, Kletzel, Morris, Vichinsky, Elliott, Soni, Sandeep, Veres, Gabor, Negre, Olivier, Ross, Robert W, Davidson, David, Petrusich, Alexandria, Sandler, Laura, Asmal, Mohammed, Hermine, Olivier, De Montalembert, Mariane, Hacein-Bey-Abina, Salima, Blanche, Stéphane, Leboulch, Philippe, Cavazzana, Marina

المصدر: New England Journal of Medicine. 378(16)

مصطلحات موضوعية: Medical Biotechnology, Biomedical and Clinical Sciences, Rare Diseases, Gene Therapy, Genetics, Biotechnology, Stem Cell Research, Cooley's Anemia, Transplantation, Hematology, Stem Cell Research - Nonembryonic - Human, Development of treatments and therapeutic interventions, 5.2 Cellular and gene therapies, Blood, Adolescent, Adult, Antigens, CD34, Child, Erythrocyte Transfusion, Female, Gene Transfer Techniques, Genetic Therapy, Genetic Vectors, Hemoglobins, Humans, Lentivirus, Male, Mutation, Transplantation, Autologous, Young Adult, beta-Globins, beta-Thalassemia, Medical and Health Sciences, General & Internal Medicine, Biomedical and clinical sciences, Health sciences

وصف الملف: application/pdf

URL الوصول: https://escholarship.org/uc/item/3xm1v7p4

المؤلفون: Pawliuk, R, Bachelot, T, Zurkiya, O, Eriksson, A, Cao, YH, Leboulch, P

المصدر: Molecular therapy : the journal of the American Society of Gene Therapy. 5(4):345-351

مصطلحات موضوعية: Medicin och hälsovetenskap

URL الوصول: http://kipublications.ki.se/Default.aspx?queryparsed=id:1929711

المؤلفون: Eriksson, A, Cao, RH, Pawliuk, R, Berg, SM, Tsang, M, Zhou, D, Fleet, C, Tritsaris, K, Dissing, S, Leboulch, P, Cao, YH

المصدر: Cancer cell. 1(1):99-108

مصطلحات موضوعية: Medicin och hälsovetenskap

URL الوصول: http://kipublications.ki.se/Default.aspx?queryparsed=id:1946707

المؤلفون: Nualkaew, T, Sii-Felice, K, Giorgi, M, McColl, B, Gouzil, J, Glaser, A, Voon, HPJ, Tee, HY, Grigoriadis, G, Svasti, S, Fucharoen, S, Hongeng, S, Leboulch, P, Payen, E, Vadolas, J

Relation: pii: S1525-0016(21)00251-3; Nualkaew, T., Sii-Felice, K., Giorgi, M., McColl, B., Gouzil, J., Glaser, A., Voon, H. P. J., Tee, H. Y., Grigoriadis, G., Svasti, S., Fucharoen, S., Hongeng, S., Leboulch, P., Payen, E. & Vadolas, J. (2021). Coordinated β-globin expression and α2-globin reduction in a multiplex lentiviral gene therapy vector for β-thalassemia. Mol Ther, 29 (9), pp.2841-2853. https://doi.org/10.1016/j.ymthe.2021.04.037.; http://hdl.handle.net/11343/301840

الاتاحة: http://hdl.handle.net/11343/301840

المؤلفون: Von Kalle, C., Schmidt, M., McCormack, M. P., Wulffraat, N., Leboulch, P., Lim, A., Osborne, C. S., Pawliuk, R., Morillon, E., Sorensen, R., Forster, A., Fraser, P., Cohen, J. I., Alexander, I., Wintergerst, U., Frebourg, T., Aurias, A., Stoppa-Lyonnet, D., Romana, S., Radford-Weiss, I., Gross, F., Valensi, F., Delabesse, E., Macintyre, E., Sigaux, F., Soulier, J., Leiva, L. E., Wissler, M., Prinz, C., Rabbitts, T. H., Le Deist, F., Fischer, A., Cavazzana-Calvo, M.

المصدر: Science, 2003 Oct . 302(5644), 415-419.

URL الوصول: https://www.jstor.org/stable/3835321

المؤلفون: Walters, M.C., Kwiatkowski, J.L., Rasko, J.E.J., Hongeng, S., Schiller, G.J., Anurathapan, U., Cavazzana, M., Ho, P.J., Schmidt, M., Kletzel, M., Leboulch, P., Vichinsky, E., Deary, B., Chen, Y., Petrusich, A., Thompson, A.A.

المصدر: HemaSphere ; volume 3, issue S1, page 22 ; ISSN 2572-9241 2572-9241

الاتاحة: http://dx.doi.org/10.1097/01.hs9.0000558784.71127.f8
https://onlinelibrary.wiley.com/doi/pdf/10.1097/01.HS9.0000558784.71127.f8

المؤلفون: Goupille, O, Penglong, T, Kadri, Z, Granger-Locatelli, M, Denis, R, Luquet, S, Badoual, C, Fucharoen, S, Maouche-Chretien, L, Leboulch, P, Chretien, S

المساهمون: Institut de Biologie François JACOB (JACOB), Direction de Recherche Fondamentale (CEA) (DRF (CEA)), Commissariat à l'énergie atomique et aux énergies alternatives (CEA)-Commissariat à l'énergie atomique et aux énergies alternatives (CEA), Mahidol University Bangkok, Unité de Biologie Fonctionnelle et Adaptative (BFA (UMR_8251 / U1133)), Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS)-Université Paris Cité (UPCité), Hôpital Européen Georges Pompidou APHP (HEGP), Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Hôpitaux Universitaires Paris Ouest - Hôpitaux Universitaires Île de France Ouest (HUPO), Université Paris Descartes - Paris 5 (UPD5), Brigham and Women's Hospital Boston, Harvard Medical School Boston (HMS)

المصدر: ISSN: 2211-1247.

مصطلحات موضوعية: GATA TRANSCRIPTION FACTORS, ZINC-FINGER PROTEINS, DIET-INDUCED OBESITY, ENERGY-EXPENDITURE, ADIPOSE-TISSUE, BROWN FAT, LACTATE, DIFFERENTIATION, WHITE, EXPRESSION, [SDV]Life Sciences [q-bio]

Relation: info:eu-repo/semantics/altIdentifier/pmid/29262331; hal-03108274; https://cnrs.hal.science/hal-03108274; https://cnrs.hal.science/hal-03108274/document; https://cnrs.hal.science/hal-03108274/file/PIIS2211124717317801.pdf; PUBMED: 29262331; WOS: 000418481600019

الاتاحة: https://cnrs.hal.science/hal-03108274
https://cnrs.hal.science/hal-03108274/document
https://cnrs.hal.science/hal-03108274/file/PIIS2211124717317801.pdf
https://doi.org/10.1016/j.celrep.2017.11.098

المؤلفون: Cao, RH, Brakenhielm, E, Pawliuk, R, Wariaro, D, Post, MJ, Wahlberg, E, Leboulch, P, Cao, YH

المصدر: Nature medicine. 9(5):604-613

مصطلحات موضوعية: Medicin och hälsovetenskap

URL الوصول: http://kipublications.ki.se/Default.aspx?queryparsed=id:1960196

المؤلفون: Gouze-Decaris, E, Pawliuk, R, Pilapil, C, Leboulch, P, Evans, CH, Ghivizzani, SC

Relation: http://arthritis-research.com/content/3/S1/P34

الاتاحة: http://arthritis-research.com/content/3/S1/P34

المؤلفون: Leboulch, P

المصدر: HÄMATOLOGIE HEUTE 2019 ; Kinder- und Jugendmedizin ; ISSN 2567-577X

الاتاحة: http://dx.doi.org/10.1055/s-0039-1684059
http://www.thieme-connect.de/products/ejournals/html/10.1055/s-0039-1684059

المؤلفون: Maurisse, R, Feugeas, J-P, Biet, E, Kuzniak, I, Leboulch, P, Dutreix, M, Sun, J-S

المصدر: Gene Therapy ; volume 9, issue 11, page 703-707 ; ISSN 0969-7128 1476-5462

الاتاحة: http://dx.doi.org/10.1038/sj.gt.3301736
https://www.nature.com/articles/3301736.pdf
https://www.nature.com/articles/3301736

المؤلفون: Watanabe, T., Kobayashi, N., Noguchi, H., Totsugawa, T., Matsumura, T., Fujiwara, T., Tanaka, N., Hikida, M., Ohmori, A., Westerman, K. A., Leboulch, P.

المصدر: ASAIO Journal ; volume 47, issue 2, page 166 ; ISSN 1058-2916

الاتاحة: http://dx.doi.org/10.1097/00002480-200103000-00260
http://journals.lww.com/00002480-200103000-00260

المؤلفون: Faulkes, S., Leboulch, P., Helgason, C., Humphries, R.K.

المصدر: Experimental Hematology ; volume 28, issue 7, page 83-84 ; ISSN 0301-472X

الاتاحة: http://dx.doi.org/10.1016/s0301-472x(00)00348-9
https://api.elsevier.com/content/article/PII:S0301472X00003489?httpAccept=text/xml
https://api.elsevier.com/content/article/PII:S0301472X00003489?httpAccept=text/plain

المؤلفون: Leboulch, P., Huang, G.M., Humphries, R.K., Oh, Y.H., Eaves, C.J., Tuan, D.Y., London, I.M.

المصدر: The EMBO Journal ; volume 13, issue 13, page 3065-3076 ; ISSN 0261-4189

الاتاحة: http://dx.doi.org/10.1002/j.1460-2075.1994.tb06605.x
https://api.wiley.com/onlinelibrary/tdm/v1/articles/10.1002%2Fj.1460-2075.1994.tb06605.x
http://onlinelibrary.wiley.com/wol1/doi/10.1002/j.1460-2075.1994.tb06605.x/fullpdf

المؤلفون: Thompson, A. A., Walters, M. C., Kwiatkowski, J., Rasko, J. E. J., Ribeil, J. -A., Hongeng, S., Magrin, E., Schiller, G. J., Payen, E., Semeraro, M., Moshous, D., Lefrere, F., Puy, H., Bourget, P., Magnani, A., Caccavelli, L., Diana, J. -S., Suarez, F., Monpoux, F., Brousse, V., Poirot, C., Brouzes, C., Meritet, J. -F., Pondarre, C., Beuzard, Y., Chretien, S., Lefebvre, T., Teachey, D. T., Anurathapan, U., Ho, P. J., von Kalle, C., Kletzel, M., Vichinsky, E., Soni, S., Veres, G., Negre, O., Ross, R. W., Davidson, D., Petrusich, A., Sandler, L., Asmal, M., Hermine, O., de Montalembert, M., Hacein-Bey-Abina, S., Blanche, S., Leboulch, P., Cavazzana, M.

المساهمون: Unité de Technologies Chimiques et Biologiques pour la Santé (UTCBS - UM 4 (UMR 8258 / U1022)), Ecole Nationale Supérieure de Chimie de Paris - Chimie ParisTech-PSL (ENSCP), Université Paris Sciences et Lettres (PSL)-Université Paris Sciences et Lettres (PSL)-Université Paris Descartes - Paris 5 (UPD5)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Institut de Chimie - CNRS Chimie (INC-CNRS)-Centre National de la Recherche Scientifique (CNRS)

المصدر: ISSN: 0028-4793.

مصطلحات موضوعية: STEM-CELL TRANSPLANTATION, BONE-MARROW-TRANSPLANTATION, GLOBIN GENE, CONDITIONING REGIMEN, HEMOGLOBINOPATHIES, ERYTHROPOIESIS, EFFICACY, DISEASE, BURDEN, VECTOR, [SDV.BIO]Life Sciences [q-bio]/Biotechnology

Relation: hal-03295931; https://cnrs.hal.science/hal-03295931; WOS: 000430312500004

الاتاحة: https://cnrs.hal.science/hal-03295931
https://doi.org/10.1056/NEJMoa1705342

المؤلفون: Magrin, Elisa, Semeraro, Michaela, Hebert, Nicolas, Joseph, Laure, Magnani, Alessandra, Chalumeau, Anne, Gabrion, Aurélie, Roudaut, Cécile, Marouene, Jouda, Lefrere, Francois, Diana, Jean-Sebastien, Denis, Adeline, Neven, Bénédicte, Funck-Brentano, Isabelle, Negre, Olivier, Renolleau, Sylvain, Brousse, Valentine, Kiger, Laurent, Touzot, Fabien, Poirot, Catherine, Bourget, Philippe, El Nemer, Wassim, Blanche, Stéphane, Tréluyer, Jean-Marc, Asmal, Mohammed, Walls, Courtney, Beuzard, Yves, Schmidt, Manfred, Hacein-Bey-Abina, Salima, Asnafi, Vahid, Guichard, Isabelle, Poirée, Maryline, Monpoux, Fabrice, Touraine, Philippe, Brouzes, Chantal, de Montalembert, Mariane, Payen, Emmanuel, Six, Emmanuelle, Ribeil, Jean-Antoine, Miccio, Annarita, Bartolucci, Pablo, Leboulch, Philippe, Cavazzana, Marina

المصدر: Nature Medicine; 20220101, Issue: Preprints p1-8, 8p

المؤلفون: Thompson AA, Walters MC, Kwiatkowski J, Rasko JEJ, Ribeil JA, Hongeng S, Magrin E, Schiller GJ, Payen E, Semeraro M, Moshous D, Lefrere F, Puy H, Bourget P, Alessandra Magnani, Caccavelli L, Diana JS, Suarez F, Monpoux F, Brousse V, Poirot C, Brouzes C, Meritet JF, Pondarré C, Beuzard Y, Chrétien S, Lefebvre T, Teachey DT, Anurathapan U, Ho PJ, von Kalle C, Kletzel M, Vichinsky E, Soni S, Veres G, Negre O, Ross RW, Davidson D, Petrusich A, Sandler L, Asmal M, Hermine O, De Montalembert M, Hacein-Bey-Abina S, Blanche S, Leboulch P, Cavazzana M

المصدر: NEW ENGLAND JOURNAL OF MEDICINE
r-FSJD. Repositorio Institucional de Producción Científica de la Fundació Sant Joan de Déu
Universidad de las Islas Baleares

URL الوصول: https://explore.openaire.eu/search/publication?articleId=RECOLECTA___::7922b9dd3a69e3d6a2c01ff4b54c8f63
http://fundanet.fsjd.org/Publicaciones/ProdCientif/PublicacionFrw.aspx?id=21069

المؤلفون: Rasko, J., Thompson, A., Kwiatkowski, J., Hongeng, S., Schiller, G.J., Anurathapan, U., Cavazzana-Calvo, M., Ho, P., von Kalle, C., Kletzel, M., Leboulch, P., Vichinsky, E., Petrusich, A., Asmal, M., Walters, M.

المصدر: Cytotherapy ; volume 19, issue 5, page S19 ; ISSN 1465-3249

الاتاحة: http://dx.doi.org/10.1016/j.jcyt.2017.02.033
https://api.elsevier.com/content/article/PII:S1465324917300919?httpAccept=text/xml
https://api.elsevier.com/content/article/PII:S1465324917300919?httpAccept=text/plain

المؤلفون: Hacein-Bey-Abina, S, Von Kalle, C, Schmidt, M, McCormack, M, Wulffraat, N, Leboulch, P, Lim, A, Osborne, C, Pawliuk, R, Morillon, E, Sorensen, R, Forster, A, Fraser, P, Cohen, J, de Saint Basile, G, Alexander, I, Wintergerst, U, Frebourg, T, Aurias, A, Stoppa-Lyonnet, D, Romana, S, Radford-Weiss, I, Gross, F, Valensi, F, Delabesse, E

Relation: https://ora.ox.ac.uk/objects/uuid:e0d55765-8770-48b8-a63d-c1031b150f2c; https://doi.org/10.1126/science.1088547

الاتاحة: https://doi.org/10.1126/science.1088547
https://ora.ox.ac.uk/objects/uuid:e0d55765-8770-48b8-a63d-c1031b150f2c