Molecular Therapy - Methods & Clinical Development

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Global regulatory progress in delivering on the promise of gene therapies for unmet medical needs
Molecular Therapy - Methods & Clinical Development - Tập 21 - Trang 524-529 - 2021
Daniela Drago, Betsy Foss-Campbell, Keith Wonnacott, David Barrett, Adora Ndu
Engineering a highly durable adeno-associated virus receptor for analytical applications
Molecular Therapy - Methods & Clinical Development - Tập 31 - Trang 101157 - 2023
Kouhei Yoshida, Yuji Tsunekawa, Kento Kurihara, Kazuya Watanabe, Yuriko Makino-Manabe, Mikako Wada, Toru Tanaka, Teruhiko Ide, Takashi Okada
Long-Term Metabolic Correction of Phenylketonuria by AAV-Delivered Phenylalanine Amino Lyase
Molecular Therapy - Methods & Clinical Development - Tập 19 - Trang 507-517 - 2020
Rui Tao, Lin Xiao, Lifang Zhou, Zhaoyue Zheng, Jie Long, Lixing Zhou, Minghai Tang, Biao Dong, Shaohua Yao
Production of lentiviral vectors
Molecular Therapy - Methods & Clinical Development - Tập 3 - Trang 16017 - 2016
Otto-Wilhelm Merten, Matthias Hebben, Chiara Bovolenta
A Single “All-in-One” Helper-Dependent Adenovirus to Deliver Donor DNA and CRISPR/Cas9 for Efficient Homology-Directed Repair
Molecular Therapy - Methods & Clinical Development - Tập 17 - Trang 441-447 - 2020
Donna Palmer, Dustin L. Turner, Philip Ng
iMATCH: an integrated modular assembly system for therapeutic combination high-capacity adenovirus gene therapy
Molecular Therapy - Methods & Clinical Development - Tập 20 - Trang 572-586 - 2021
Dominik Brücher, Nicole Kirchhammer, Sheena N. Smith, Jatina Schumacher, Nina Schumacher, Jonas Kolibius, Patrick C. Freitag, Markus Schmid, Fabian Weiss, Corina Keller, Melanie Grove, Urs F. Greber, Alfred Zippelius, Andreas Plückthun
Expression mediated by three partial sequences of the human tyrosine hydroxylase promoter in vivo
Molecular Therapy - Methods & Clinical Development - Tập 3 - Trang 16062 - 2016
Anne-Sophie Rolland, Tatyana Kareva, Olga Yarygina, Nikolai Kholodilov, Robert E Burke
Generation and in vivo evaluation of IL10-treated dendritic cells in a nonhuman primate model of AAV-based gene transfer
Molecular Therapy - Methods & Clinical Development - Tập 1 - Trang 14028 - 2014
Aurélie Moreau, Céline Vandamme, Mercedes Segovia, Marie Devaux, Mickaël Guilbaud, Gaëlle Tilly, Nicolas Jaulin, Johanne Le Duff, Yan Cherel, Jack-Yves Deschamps, Ignacio Anegon, Philippe Moullier, Maria Cristina Cuturi, Oumeya Adjali
Efficient detection of human circulating tumor cells without significant production of false-positive cells by a novel conditionally replicating adenovirus
Molecular Therapy - Methods & Clinical Development - Tập 3 - Trang 16001 - 2016
Fuminori Sakurai, Nobuhiro Narii, Kyoko Tomita, Shinsaku Togo, Kazuhisa Takahashi, Mitsuhiro Machitani, Masashi Tachibana, Masaaki Ouchi, Nobuyoshi Katagiri, Yasuo Urata, Toshiyoshi Fujiwara, Hiroyuki Mizuguchi
Dosing and Re-Administration of Lentiviral Vector for In Vivo Gene Therapy in Rhesus Monkeys and ADA-Deficient Mice
Molecular Therapy - Methods & Clinical Development - Tập 16 - Trang 78-93 - 2020
Denise A. Carbonaro-Sarracino, Alice F. Tarantal, C. Chang I. Lee, Michael L. Kaufman, Stephen Wandro, Xiangyang Jin, Michele Martinez, Danielle N. Clark, Krista Chun, Colin Koziol, Cinnamon L. Hardee, Xiaoyan Wang, Donald B. Kohn
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