المؤلفون: Hsu, Ying, Bhattarai, Sajag, Thompson, Jacob M., Mahoney, Angela, Thomas, Jacintha, Mayer, Sara K., Datta, Poppy, Garrison, Janelle, Searby, Charles C., Vandenberghe, Luk H., Seo, Seongjin, Sheffield, Val C., Drack, Arlene V.

المصدر: Molecular Therapy - Nucleic Acids ; volume 31, page 164-181 ; ISSN 2162-2531

مصطلحات موضوعية: Drug Discovery, Molecular Medicine

الإتاحة: https://doi.org/10.1016/j.omtn.2022.12.007Test
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https://api.elsevier.com/content/article/PII:S2162253122003286?httpAccept=text/plainTest

المؤلفون: Kumar, Prashant, Wang, Michael, Kumru, Ozan S., Hickey, John M., Sanmiguel, Julio, Zabaleta, Nerea, Vandenberghe, Luk H., Joshi, Sangeeta B., Volkin, David B.

المساهمون: Bill and Melinda Gates Foundation

المصدر: Molecular Therapy - Methods & Clinical Development ; volume 30, page 103-121 ; ISSN 2329-0501

مصطلحات موضوعية: Genetics, Molecular Biology, Molecular Medicine

الإتاحة: https://doi.org/10.1016/j.omtm.2023.06.004Test
https://api.elsevier.com/content/article/PII:S2329050123000943?httpAccept=text/xmlTest
https://api.elsevier.com/content/article/PII:S2329050123000943?httpAccept=text/plainTest

المؤلفون: Florea, Michael, Nicolaou, Fotini, Pacouret, Simon, Zinn, Eric M., Sanmiguel, Julio, Andres-Mateos, Eva, Unzu, Carmen, Wagers, Amy J., Vandenberghe, Luk H.

المصدر: Molecular Therapy - Methods & Clinical Development ; volume 28, page 146-159 ; ISSN 2329-0501

مصطلحات موضوعية: Genetics, Molecular Biology, Molecular Medicine

الإتاحة: https://doi.org/10.1016/j.omtm.2022.12.009Test
https://api.elsevier.com/content/article/PII:S2329050122001826?httpAccept=text/xmlTest
https://api.elsevier.com/content/article/PII:S2329050122001826?httpAccept=text/plainTest

المؤلفون: Zabaleta, Nerea, Bhatt, Urja, Hérate, Cécile, Maisonnasse, Pauline, Sanmiguel, Julio, Diop, Cheikh, Castore, Sofia, Estelien, Reynette, Li, Dan, Dereuddre-Bosquet, Nathalie, Cavarelli, Mariangela, Gallouët, Anne-Sophie, Pascal, Quentin, Naninck, Thibaut, Kahlaoui, Nidhal, Lemaitre, Julien, Relouzat, Francis, Ronzitti, Giuseppe, Thibaut, Hendrik, Jan, Montomoli, Emanuele, Wilson, James, M, Le Grand, Roger, Vandenberghe, Luk, H

المساهمون: Infectious Diseases Models for Innovative Therapies (IDMIT), Université Paris-Saclay-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)-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), ANR-11-INBS-0008,IDMIT,Infrastructure nationale pour la modélisation des maladies infectieuses humaines(2011)

المصدر: ISSN: 1525-0016.

مصطلحات موضوعية: [SDV]Life Sciences [q-bio]

العلاقة: hal-04510127; https://hal.science/hal-04510127Test; https://hal.science/hal-04510127/documentTest; https://hal.science/hal-04510127/file/2022%20Zabaleta%20MolTher.pdfTest

الإتاحة: https://doi.org/10.1016/j.ymthe.2022.05.007Test
https://hal.science/hal-04510127Test
https://hal.science/hal-04510127/documentTest
https://hal.science/hal-04510127/file/2022%20Zabaleta%20MolTher.pdfTest

المؤلفون: Grotz, Sophia, Schäfer, Jessica, Wunderlich, Kirsten A, Ellederova, Zdenka, Auch, Hannah, Bähr, Andrea, Runa‐Vochozkova, Petra, Fadl, Janet, Arnold, Vanessa, Ardan, Taras, Veith, Miroslav, Santamaria, Gianluca, Dhom, Georg, Hitzl, Wolfgang, Kessler, Barbara, Eckardt, Christian, Klein, Joshua, Brymova, Anna, Linnert, Joshua, Kurome, Mayuko, Zakharchenko, Valeri, Fischer, Andrea, Blutke, Andreas, Döring, Anna, Suchankova, Stepanka, Popelar, Jiri, Rodríguez‐Bocanegra, Eduardo, Dlugaiczyk, Julia, Straka, Hans, May‐Simera, Helen, Wang, Weiwei, Laugwitz, Karl‐Ludwig, Vandenberghe, Luk H, Wolf, Eckhard, Nagel‐Wolfrum, Kerstin, Peters, Tobias, Motlik, Jan, Fischer, M Dominik, Wolfrum, Uwe, Klymiuk, Nikolai

المساهمون: Deutsche Forschungsgemeinschaft, Foundation Fighting Blindness

المصدر: EMBO Molecular Medicine ; volume 14, issue 4 ; ISSN 1757-4676 1757-4684

مصطلحات موضوعية: Molecular Medicine

الإتاحة: https://doi.org/10.15252/emmm.202114817Test

المؤلفون: Andres-Mateos, Eva, Landegger, Lukas D., Unzu, Carmen, Phillips, Jean, Lin, Brian M., Dewyer, Nicholas A., Sanmiguel, Julio, Nicolaou, Fotini, Valero, Michelle D., Bourdeu, Kathrin I., Sewell, William F., Beiler, Rudolph J., McKenna, Michael J., Stankovic, Konstantina M., Vandenberghe, Luk H.

المساهمون: U.S. Department of Health & Human Services | NIH | National Institute on Deafness and Other Communication Disorders, Nancy Sayles Day Foundation, Lauer Tinnitus Research Center, Massachusetts Eye and Ear

المصدر: Nature Communications ; volume 13, issue 1 ; ISSN 2041-1723

مصطلحات موضوعية: General Physics and Astronomy, General Biochemistry, Genetics and Molecular Biology, General Chemistry, Multidisciplinary

الإتاحة: https://doi.org/10.1038/s41467-022-28969-3Test
https://www.nature.com/articles/s41467-022-28969-3.pdfTest
https://www.nature.com/articles/s41467-022-28969-3Test

المؤلفون: Naninck, Thibaut, Kahlaoui, Nidhal, Lemaitre, Julien, Maisonnasse, Pauline, De Mori, Antoine, Pascal, Quentin, Contreras, Vanessa, Marlin, Romain, Relouzat, Francis, Delache, Benoît, Hérate, Cécile, Aldon, Yoann, van Gils, Marit, Zabaleta, Nerea, Ho Tsong Fang, Raphaël, Bosquet, Nathalie, Sanders, Rogier W., Vandenberghe, Luk H., Chapon, Catherine, Le Grand, Roger

المصدر: iScience ; volume 25, issue 4, page 104101 ; ISSN 2589-0042

مصطلحات موضوعية: Multidisciplinary

الإتاحة: https://doi.org/10.1016/j.isci.2022.104101Test
https://api.elsevier.com/content/article/PII:S2589004222003716?httpAccept=text/xmlTest
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المؤلفون: Katz, Michael G., Hadas, Yoav, Bailey, Rasheed A., Fazal, Shahood, Vincek, Adam, Madjarova, Sophia J., Shtraizent, Nataly, Vandenberghe, Luk H., Eliyahu, Efrat

المساهمون: NIH, National Heart, Lung, and Blood Institute, Icahn School of Medicine at Mount Sinai

المصدر: The Journal of Thoracic and Cardiovascular Surgery ; volume 164, issue 6, page e429-e443 ; ISSN 0022-5223

مصطلحات موضوعية: Cardiology and Cardiovascular Medicine, Pulmonary and Respiratory Medicine, Surgery

الإتاحة: https://doi.org/10.1016/j.jtcvs.2021.05.050Test
https://api.elsevier.com/content/article/PII:S0022522321009156?httpAccept=text/xmlTest
https://api.elsevier.com/content/article/PII:S0022522321009156?httpAccept=text/plainTest

المؤلفون: Zinn, Eric, Unzu, Carmen, Schmit, Pauline F., Turunen, Heikki T., Zabaleta, Nerea, Sanmiguel, Julio, Fieldsend, Allegra, Bhatt, Urja, Diop, Cheikh, Merkel, Erin, Gurrala, Rakesh, Peacker, Bryan, Rios, Christopher, Messemer, Kathleen, Santos, Jennifer, Estelien, Reynette, Andres-Mateos, Eva, Wagers, Amy J., Tipper, Christopher, Vandenberghe, Luk H.

المصدر: Cell Reports Medicine ; volume 3, issue 11, page 100803 ; ISSN 2666-3791

مصطلحات موضوعية: General Biochemistry, Genetics and Molecular Biology

الإتاحة: https://doi.org/10.1016/j.xcrm.2022.100803Test
https://api.elsevier.com/content/article/PII:S2666379122003627?httpAccept=text/xmlTest
https://api.elsevier.com/content/article/PII:S2666379122003627?httpAccept=text/plainTest

المؤلفون: Cheever, Thomas R, Berkley, Dale, Braun, Serge, Brown, Robert H, Byrne, Barry J, Chamberlain, Jeffrey S, Cwik, Valerie, Duan, Dongsheng, Federoff, Howard J, High, Katherine A, Kaspar, Brian K, Klinger, Katherine W, Larkindale, Jane, Lincecum, John, Mavilio, Fulvio, McDonald, Cheryl L, McLaughlin, James, McLeod, Bonnie Weiss, Mendell, Jerry R, Nuckolls, Glen, Stedman, Hansell H, Tagle, Danilo A, Vandenberghe, Luk H, Wang, Hao, Wernett, Pamela J, Wilson, James M, Porter, John D, Gubitz, Amelie K

المصدر: Human Gene Therapy. 26(3)

مصطلحات موضوعية: Muscular Dystrophy, Intellectual and Developmental Disabilities (IDD), Rare Diseases, Brain Disorders, Neurosciences, Mental Health, Orphan Drug, Gene Therapy, Pediatric, Genetics, Clinical Trials as Topic, Genetic Therapy, Government Regulation, Humans, Intellectual Property, Neuromuscular Diseases

وصف الملف: application/pdf

الوصول الحر: https://escholarship.org/uc/item/8z40m0q8Test