Inhibition of MicroRNA-9-5p Protects Against Cardiac Remodeling Following Myocardial Infarction in Mice
Xiao, Yimin, Zhang, Yanxia, Chen, Yueqiu, Li, Jingjing, Zhang, Zihan, Sun, Yimin, Shen, Han, Zhao, Zhenao, Huang, Zan, Zhang, Wencheng, Chen, Weiqian, Shen, Zhenya
Published in Human gene therapy (01.03.2019)
Published in Human gene therapy (01.03.2019)
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Long-term evaluation of a phase 1 study of AADC gene therapy for Parkinson's disease
Mittermeyer, Gabriele, Christine, Chadwick W, Rosenbluth, Kathryn H, Baker, Suzanne L, Starr, Philip, Larson, Paul, Kaplan, Paul L, Forsayeth, John, Aminoff, Michael J, Bankiewicz, Krystof S
Published in Human gene therapy (01.04.2012)
Published in Human gene therapy (01.04.2012)
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Journal Article
Adeno-Associated Virus Gene Therapy in a Sheep Model of Tay-Sachs Disease
Gray-Edwards, Heather L, Randle, Ashley N, Maitland, Stacy A, Benatti, Hector R, Hubbard, Spencer M, Canning, Peter F, Vogel, Matthew B, Brunson, Brandon L, Hwang, Misako, Ellis, Lauren E, Bradbury, Allison M, Gentry, Atoska S, Taylor, Amanda R, Wooldridge, Anne A, Wilhite, Dewey R, Winter, Randolph L, Whitlock, Brian K, Johnson, Jacob A, Holland, Merilee, Salibi, Nouha, Beyers, Ronald J, Sartin, James L, Denney, Thomas S, Cox, Nancy R, Sena-Esteves, Miguel, Martin, Douglas R
Published in Human gene therapy (01.03.2018)
Published in Human gene therapy (01.03.2018)
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Journal Article
Development of Anti-Human Mesothelin-Targeted Chimeric Antigen Receptor Messenger RNA-Transfected Peripheral Blood Lymphocytes for Ovarian Cancer Therapy
Hung, Chien-Fu, Xu, Xuequn, Li, Linhong, Ma, Ying, Jin, Qiu, Viley, Angelia, Allen, Cornell, Natarajan, Pachai, Shivakumar, Rama, Peshwa, Madhusudan V, Emens, Leisha A
Published in Human gene therapy (01.05.2018)
Published in Human gene therapy (01.05.2018)
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Journal Article
Delivery of Adeno-Associated Virus Vectors in Adult Mammalian Inner-Ear Cell Subtypes Without Auditory Dysfunction
Tao, Yong, Huang, Mingqian, Shu, Yilai, Ruprecht, Adam, Wang, Hongyang, Tang, Yong, Vandenberghe, Luk H, Wang, Qiuju, Gao, Guangping, Kong, Wei-Jia, Chen, Zheng-Yi
Published in Human gene therapy (01.04.2018)
Published in Human gene therapy (01.04.2018)
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Journal Article
Single-Use Capture Purification of Adeno-Associated Viral Gene Transfer Vectors by Membrane-Based Steric Exclusion Chromatography
Marichal-Gallardo, Pavel, Börner, Kathleen, Pieler, Michael M, Sonntag-Buck, Vera, Obr, Martin, Bejarano, David, Wolff, Michael W, Kräusslich, Hans-Georg, Reichl, Udo, Grimm, Dirk
Published in Human gene therapy (01.09.2021)
Published in Human gene therapy (01.09.2021)
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Journal Article
Adenosine Deaminase 1 Overexpression Enhances the Antitumor Efficacy of Chimeric Antigen Receptor-Engineered T Cells
Qu, Yun, Dunn, Zachary S, Chen, Xianhui, MacMullan, Melanie, Cinay, Gunce, Wang, Hsuan-Yao, Liu, Jiangyue, Hu, Fangheng, Wang, Pin
Published in Human gene therapy (01.03.2022)
Published in Human gene therapy (01.03.2022)
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Journal Article
MALAT1-Driven Inhibition of Wnt Signal Impedes Proliferation and Inflammation in Fibroblast-Like Synoviocytes Through CTNNB1 Promoter Methylation in Rheumatoid Arthritis
Li, Guo-Qing, Fang, Yu-Xuan, Liu, Ying, Meng, Fan-Ru, Wu, Xia, Zhang, Chun-Wang, Zhang, Yu, Liu, Dan, Gao, Bo
Published in Human gene therapy (01.08.2019)
Published in Human gene therapy (01.08.2019)
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Journal Article
Complement Activation by Adeno-Associated Virus-Neutralizing Antibody Complexes
West, Cara, Federspiel, Joel D, Rogers, Kara, Khatri, Arpana, Rao-Dayton, Sheila, Ocana, Mireia Fernandez, Lim, Sean, D'Antona, Aaron Michael, Casinghino, Sandra, Somanathan, Suryanarayan
Published in Human gene therapy (01.06.2023)
Published in Human gene therapy (01.06.2023)
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Journal Article
Assessment of AAV Vector Tropisms for Mouse and Human Pluripotent Stem Cell-Derived RPE and Photoreceptor Cells
Gonzalez-Cordero, Anai, Goh, Debbie, Kruczek, Kamil, Naeem, Arifa, Fernando, Milan, Kleine Holthaus, Sophia-Martha, Takaaki, Matsuki, Blackford, Samuel J I, Kloc, Magdalena, Agundez, Leticia, Sampson, Robert D, Borooah, Shyamanga, Ovando-Roche, Patrick, Mehat, Manjit S, West, Emma L, Smith, Alexander J, Pearson, Rachael A, Ali, Robin R
Published in Human gene therapy (01.10.2018)
Published in Human gene therapy (01.10.2018)
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In Vivo Genome Editing Partially Restores Alpha1-Antitrypsin in a Murine Model of AAT Deficiency
Song, Chun-Qing, Wang, Dan, Jiang, Tingting, O'Connor, Kevin, Tang, Qiushi, Cai, Lingling, Li, Xiangrui, Weng, Zhiping, Yin, Hao, Gao, Guangping, Mueller, Christian, Flotte, Terence R, Xue, Wen
Published in Human gene therapy (01.08.2018)
Published in Human gene therapy (01.08.2018)
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Journal Article
Age-Related Seroprevalence of Antibodies Against AAV-LK03 in a UK Population Cohort
Perocheau, Dany P, Cunningham, Sharon, Lee, Juhee, Antinao Diaz, Juan, Waddington, Simon N, Gilmour, Kimberly, Eaglestone, Simon, Lisowski, Leszek, Thrasher, Adrian J, Alexander, Ian E, Gissen, Paul, Baruteau, Julien
Published in Human gene therapy (01.01.2019)
Published in Human gene therapy (01.01.2019)
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Journal Article
AAVrh10 Gene Therapy Ameliorates Central and Peripheral Nervous System Disease in Canine Globoid Cell Leukodystrophy (Krabbe Disease)
Bradbury, Allison M, Rafi, Mohammed A, Bagel, Jessica H, Brisson, Becky K, Marshall, Michael S, Pesayco Salvador, Jill, Jiang, Xuntain, Swain, Gary P, Prociuk, Maria L, ODonnell, Patricia A, Fitzgerald, Caitlin, Ory, Daniel S, Bongarzone, Ernesto R, Shelton, G Diane, Wenger, David A, Vite, Charles H
Published in Human gene therapy (01.07.2018)
Published in Human gene therapy (01.07.2018)
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Journal Article
Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce DMD Exon 2 Skipping
Gushchina, Liubov V, Frair, Emma C, Rohan, Natalie, Bradley, Adrienne J, Simmons, Tabatha R, Chavan, Hemantkumar D, Chou, Hsin-Jung, Eggers, Michelle, Waldrop, Megan A, Wein, Nicolas, Flanigan, Kevin M
Published in Human gene therapy (01.09.2021)
Published in Human gene therapy (01.09.2021)
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Journal Article
Results at 5 Years After Gene Therapy for RPE65-Deficient Retinal Dystrophy
Pennesi, Mark E, Weleber, Richard G, Yang, Paul, Whitebirch, Chris, Thean, Beverly, Flotte, Terence R, Humphries, Margaret, Chegarnov, Elvira, Beasley, Kathleen N, Stout, J Timothy, Chulay, Jeffrey D
Published in Human gene therapy (01.12.2018)
Published in Human gene therapy (01.12.2018)
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Journal Article
In Vivo Ovarian Cancer Gene Therapy Using CRISPR-Cas9
He, Zhi-Yao, Zhang, Ya-Guang, Yang, Yu-Han, Ma, Cui-Cui, Wang, Ping, Du, Wei, Li, Ling, Xiang, Rong, Song, Xiang-Rong, Zhao, Xia, Yao, Shao-Hua, Wei, Yu-Quan
Published in Human gene therapy (01.02.2018)
Published in Human gene therapy (01.02.2018)
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