المؤلفون: Salome Funes, Jonathan Jung, Del Hayden Gadd, Michelle Mosqueda, Jianjun Zhong, Shankaracharya, Matthew Unger, Karly Stallworth, Debra Cameron, Melissa S. Rotunno, Pepper Dawes, Megan Fowler-Magaw, Pamela J. Keagle, Justin A. McDonough, Sivakumar Boopathy, Miguel Sena-Esteves, Jeffrey A. Nickerson, Cathleen Lutz, William C. Skarnes, Elaine T. Lim, Dorothy P. Schafer, Francesca Massi, John E. Landers, Daryl A. Bosco

المصدر: Nature Communications, Vol 15, Iss 1, Pp 1-25 (2024)

مصطلحات موضوعية: Science

وصف الملف: electronic resource

Relation: https://doaj.org/toc/2041-1723

URL الوصول: https://doaj.org/article/2c85d3969eb543aa96ee0dead9833143

المؤلفون: Minggang Fang, Sara K. Deibler, Pranathi Meda Krishnamurthy, Feng Wang, Paola Rodriguez, Shahid Banday, Ching-Man Virbasius, Miguel Sena-Esteves, Jonathan K. Watts, Michael R. Green

المصدر: Frontiers in Neuroscience, Vol 18 (2024)

مصطلحات موضوعية: FXS, FMR1 silencing, FMR1 reactivation, EZH2 (enhancer of zeste homolog 2), ASO - antisense oligonucleotides, neuron differentiation, Neurosciences. Biological psychiatry. Neuropsychiatry, RC321-571

وصف الملف: electronic resource

Relation: https://www.frontiersin.org/articles/10.3389/fnins.2024.1348478/full; https://doaj.org/toc/1662-453X

URL الوصول: https://doaj.org/article/3a274fd2567945d28c750f6d92ecc94f

المؤلفون: Hector Ribeiro Benatti, Rachel D. Prestigiacomo, Toloo Taghian, Rachael Miller, Robert King, Matthew J. Gounis, Ugur Celik, Stephanie Bertrand, Susan Tuominen, Lindsey Bierfeldt, Elizabeth Parsley, Jillian Gallagher, Erin F. Hall, Abigail W. McElroy, Miguel Sena-Esteves, Anastasia Khvorova, Neil Aronin, Heather L. Gray-Edwards

المصدر: Molecular Therapy: Methods & Clinical Development, Vol 31, Iss , Pp 101122- (2023)

مصطلحات موضوعية: oligonucleotide safety assessment, large animal model, CNS drug administration, CSF drug delivery, i.c.v. injection, Ommaya reservoir, Genetics, QH426-470, Cytology, QH573-671

وصف الملف: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2329050123001614; https://doaj.org/toc/2329-0501

URL الوصول: https://doaj.org/article/47746a402a8d49c9b29336720b82daae

المؤلفون: Michaël Hocquemiller, Laura Giersch, Xin Mei, Amanda L. Gross, Ashley N. Randle, Heather L. Gray-Edwards, Judith A. Hudson, Sophia Todeasa, Lorelei Stoica, Douglas R. Martin, Miguel Sena-Esteves, Karen Aiach, Ralph Laufer

المصدر: Molecular Therapy: Methods & Clinical Development, Vol 27, Iss , Pp 281-292 (2022)

مصطلحات موضوعية: AAV, gangliosidosis, GM1, gene therapy, lysosomal storage disease, Genetics, QH426-470, Cytology, QH573-671

وصف الملف: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2329050122001462; https://doaj.org/toc/2329-0501

URL الوصول: https://doaj.org/article/157bca7427c44bc4a59c76e532caa74e

المؤلفون: Bernie S. Owusu-Yaw, Yongzhi Zhang, Lilyan Garrett, Alvin Yao, Kai Shing, Ana Rita Batista, Miguel Sena-Esteves, Jaymin Upadhyay, Kimberly Kegel-Gleason, Nick Todd

المصدر: Pharmaceutics, Vol 16, Iss 6, p 710 (2024)

مصطلحات موضوعية: focused ultrasound, microbubbles, blood–brain barrier opening, gene therapy, Huntington’s disease, Pharmacy and materia medica, RS1-441

وصف الملف: electronic resource

Relation: https://www.mdpi.com/1999-4923/16/6/710; https://doaj.org/toc/1999-4923

URL الوصول: https://doaj.org/article/43d0c465e7be489286ab7330c5da1dca

المؤلفون: Toloo Taghian, Ana Rita Batista, Sarah Kamper, Michael Caldwell, Laura Lilley, Hao Li, Paola Rodriguez, Katerina Mesa, Shaokuan Zheng, Robert M. King, Matthew J. Gounis, Sophia Todeasa, Anne Maguire, Douglas R. Martin, Miguel Sena-Esteves, Thomas J. Meade, Heather L. Gray-Edwards

المصدر: Molecular Therapy: Methods & Clinical Development, Vol 23, Iss , Pp 128-134 (2021)

مصطلحات موضوعية: AAV gene therapy, magnetic resonance imaging, enzyme-activated contrast agent probes, GM1 gangliosidosis, MR tracking of enzyme expression, Genetics, QH426-470, Cytology, QH573-671

وصف الملف: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2329050121001297; https://doaj.org/toc/2329-0501

URL الوصول: https://doaj.org/article/c16bf88485644845ad32d3c283989ad8

المؤلفون: Oeystein R. Brekk, Joanna A. Korecka, Cecile C. Crapart, Mylene Huebecker, Zachary K. MacBain, Sara Ann Rosenthal, Miguel Sena-Esteves, David A. Priestman, Frances M. Platt, Ole Isacson, Penelope J. Hallett

المصدر: Acta Neuropathologica Communications, Vol 8, Iss 1, Pp 1-14 (2020)

مصطلحات موضوعية: α-Synuclein, β-Hexosaminidase, Sandhoff disease, Parkinson’s disease, Neuroprotection, Lipid binding, Neurology. Diseases of the nervous system, RC346-429

وصف الملف: electronic resource

Relation: http://link.springer.com/article/10.1186/s40478-020-01004-6; https://doaj.org/toc/2051-5960

URL الوصول: https://doaj.org/article/a6679db376524eaf95dfb742563c7bce

المؤلفون: Heather L. Gray-Edwards, Anne S. Maguire, Nouha Salibi, Lauren E. Ellis, Taylor L. Voss, Elise B. Diffie, Jey Koehler, Ashley N. Randle, Amanda R. Taylor, Brandon L. Brunson, Thomas S. Denney, Ronald J. Beyers, Atoska S. Gentry, Amanda L. Gross, Ana R. Batista, Miguel Sena-Esteves, Douglas R. Martin

المصدر: Molecular Therapy: Methods & Clinical Development, Vol 17, Iss , Pp 258-270 (2020)

مصطلحات موضوعية: gangliosidosis, GM1, lysosomal storage disease, adeno-associated virus, AAV, gene therapy, Genetics, QH426-470, Cytology, QH573-671

وصف الملف: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2329050119301536; https://doaj.org/toc/2329-0501

URL الوصول: https://doaj.org/article/e5a002b5c4934f989fe4e2733f002031

المؤلفون: Killian S. Hanlon, Jonah C. Meltzer, Tetyana Buzhdygan, Ming J. Cheng, Miguel Sena-Esteves, Rachel E. Bennett, Timothy P. Sullivan, Roshanak Razmpour, Yi Gong, Carrie Ng, Josette Nammour, Daniela Maiz, Simon Dujardin, Servio H. Ramirez, Eloise Hudry, Casey A. Maguire

المصدر: Molecular Therapy: Methods & Clinical Development, Vol 15, Iss , Pp 320-332 (2019)

مصطلحات موضوعية: Genetics, QH426-470, Cytology, QH573-671

وصف الملف: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2329050119301159; https://doaj.org/toc/2329-0501

URL الوصول: https://doaj.org/article/52e01ca87cb94b9eaa2bbaf0a5e3ec5e

المؤلفون: Shilpa Prabhakar, Pike See Cheah, Xuan Zhang, Max Zinter, Maria Gianatasio, Eloise Hudry, Roderick T. Bronson, David J. Kwiatkowski, Anat Stemmer-Rachamimov, Casey A. Maguire, Miguel Sena-Esteves, Bakhos A. Tannous, Xandra O. Breakefield

المصدر: Molecular Therapy: Methods & Clinical Development, Vol 15, Iss , Pp 18-26 (2019)

مصطلحات موضوعية: Genetics, QH426-470, Cytology, QH573-671

وصف الملف: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S232905011930083X; https://doaj.org/toc/2329-0501

URL الوصول: https://doaj.org/article/5011cb5ccfc6461d9959126df3595260

المؤلفون: Dan Wang, Li Zhong, Mengxin Li, Jia Li, Karen Tran, Lingzhi Ren, Ran He, Jun Xie, Richard P. Moser, Cara Fraser, Tim Kuchel, Miguel Sena-Esteves, Terence R. Flotte, Neil Aronin, Guangping Gao

المصدر: Molecular Therapy: Methods & Clinical Development, Vol 11, Iss , Pp 65-72 (2018)

مصطلحات موضوعية: Genetics, QH426-470, Cytology, QH573-671

وصف الملف: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2329050118300949; https://doaj.org/toc/2329-0501

URL الوصول: https://doaj.org/article/e3be954612544666b30cee85c60993ec

المؤلفون: Maria L. Allende, Emily K. Cook, Bridget C. Larman, Adrienne Nugent, Jacqueline M. Brady, Diane Golebiowski, Miguel Sena-Esteves, Cynthia J. Tifft, Richard L. Proia

المصدر: Journal of Lipid Research, Vol 59, Iss 3, Pp 550-563 (2018)

مصطلحات موضوعية: storage diseases, gangliosides, Tay-Sachs disease, sphingolipids, brain lipids, Clustered Regularly Interspaced Short Palindromic Repeats/Cas9, Biochemistry, QD415-436

وصف الملف: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S0022227520331424; https://doaj.org/toc/0022-2275

URL الوصول: https://doaj.org/article/92b131018d354437940c5128314b85f9

المؤلفون: Heather L. Gray-Edwards, Xuntian Jiang, Ashley N. Randle, Amanda R. Taylor, Taylor L. Voss, Aime K. Johnson, Victoria J. McCurdy, Miguel Sena-Esteves, Daniel S. Ory, Douglas R. Martin

المصدر: Molecular Therapy: Methods & Clinical Development, Vol 6, Iss C, Pp 135-142 (2017)

مصطلحات موضوعية: adeno-associated virus, AAV, biomarkers, feline, gene therapy, GM1 gangliosidosis, lipidomics, neurologic disease, Genetics, QH426-470, Cytology, QH573-671

وصف الملف: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2329050117300888; https://doaj.org/toc/2329-0501

URL الوصول: https://doaj.org/article/3e3a1ecbcf1a457a8a1a35bcff0c25d7

المؤلفون: Yvonne L. Latour, Robin Yoon, Sarah E. Thomas, Christina Grant, Cuiling Li, Miguel Sena-Esteves, Maria L. Allende, Richard L. Proia, Cynthia J. Tifft

المصدر: Molecular Genetics and Metabolism Reports, Vol 21, Iss , Pp - (2019)

مصطلحات موضوعية: Medicine (General), R5-920, Biology (General), QH301-705.5

وصف الملف: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2214426919301326; https://doaj.org/toc/2214-4269

URL الوصول: https://doaj.org/article/2b29594b637545ee87b35dd4170f78d5

المؤلفون: Shilpa Prabhakar, Xuan Zhang, June Goto, Sangyeul Han, Charles Lai, Roderick Bronson, Miguel Sena-Esteves, Vijaya Ramesh, Anat Stemmer-Rachamimov, David J. Kwiatkowski, Xandra O. Breakefield

المصدر: Neurobiology of Disease, Vol 82, Iss , Pp 22-31 (2015)

مصطلحات موضوعية: Tuberous sclerosis complex, TSC, TSC1, TSC2, Gene therapy, AAV, Neurosciences. Biological psychiatry. Neuropsychiatry, RC321-571

وصف الملف: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S0969996115001758; https://doaj.org/toc/1095-953X

URL الوصول: https://doaj.org/article/8eae6760b48943a698ff72cfc816c4b9

المؤلفون: Lorelei Stoica, Miguel Sena-Esteves

المصدر: Frontiers in Molecular Neuroscience, Vol 9 (2016)

مصطلحات موضوعية: Gene Therapy, Superoxide Dismutase, RNAi, ALS, AAV vectors, Neurosciences. Biological psychiatry. Neuropsychiatry, RC321-571

وصف الملف: electronic resource

Relation: http://journal.frontiersin.org/Journal/10.3389/fnmol.2016.00056/full; https://doaj.org/toc/1662-5099

URL الوصول: https://doaj.org/article/faf9f57afba746eebbc277773eac7138

المؤلفون: Sandra Almeida, Zhijun Zhang, Giovanni Coppola, Wenjie Mao, Kensuke Futai, Anna Karydas, Michael D. Geschwind, M. Carmela Tartaglia, Fuying Gao, Davide Gianni, Miguel Sena-Esteves, Daniel H. Geschwind, Bruce L. Miller, Robert V. Farese, Jr., Fen-Biao Gao

المصدر: Cell Reports, Vol 2, Iss 4, Pp 789-798 (2012)

مصطلحات موضوعية: Biology (General), QH301-705.5

وصف الملف: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2211124712002884; https://doaj.org/toc/2211-1247

URL الوصول: https://doaj.org/article/2adf8552b17e40d2a9317f84afa9d11e

المؤلفون: Clelia Fusco, Joana Saraiva, Joao Castelhano, Jose Sereno, Luis Pereira De Almeida, Magda Santana, Miguel Castelo-Branco, Miguel Sena-Esteves, Rui Jorge Nobre, Susana Paixao

المصدر: Proceedings for Annual Meeting of The Japanese Pharmacological Society. 2018, :OR24-4

المؤلفون: Hannah E. Rockwell, Victoria J. McCurdy, Samuel C. Eaton, Diane U. Wilson, Aime K. Johnson, Ashley N. Randle, Allison M. Bradbury, Heather L. Gray-Edwards, Henry J. Baker, Judith A. Hudson, Nancy R. Cox, Miguel Sena-Esteves, Thomas N. Seyfried, Douglas R. Martin

المصدر: ASN Neuro, Vol 7 (2015)

مصطلحات موضوعية: Neurosciences. Biological psychiatry. Neuropsychiatry, RC321-571

وصف الملف: electronic resource

Relation: https://doaj.org/toc/1759-0914

URL الوصول: https://doaj.org/article/ca9dfbf3374b4466a2748535a40d9de2

المؤلفون: Andreas Jacobs, Michael Dubrovin, Jeff Hewett, Miguel Sena-Esteves, Cui-Wen Tan, Mark Slack, Michele Sadelain, Xandra O. Breakefield, Juri G. Tjuvajev

المصدر: Neoplasia: An International Journal for Oncology Research, Vol 1, Iss 2, Pp 154-161 (1999)

مصطلحات موضوعية: thymidine kinase, ganciclovir, FIAU, cancer gene therapy, fusion genes, imaging, Neoplasms. Tumors. Oncology. Including cancer and carcinogens, RC254-282

وصف الملف: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S1476558699800566; https://doaj.org/toc/1476-5586; https://doaj.org/toc/1522-8002

URL الوصول: https://doaj.org/article/7c5692980c014d1a9e70091d6d02bd80