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Preclinical evaluation of KH631, a novel rAAV8 gene therapy product for neovascular age-related macular degeneration Mol. Ther. (IF 12.4) Pub Date : 2023-09-26 Xiao Ke, Hao Jiang, Qingwei Li, Shuang Luo, Yingfei Qin, Jing Li, Qing Xie, Qiang Zheng
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Di-valent siRNA-mediated silencing of MSH3 blocks somatic repeat expansion in mouse models of Huntington’s disease Mol. Ther. (IF 12.4) Pub Date : 2023-09-25 Daniel O’Reilly, Jillian Belgrad, Chantal Ferguson, Ashley Summers, Ellen Sapp, Cassandra McHugh, Ella Mathews, Adel Boudi, Julianna Buchwald, Socheata Ly, Dimas Moreno, Raymond Furgal, Eric Luu, Zachary Kennedy, Vignesh Hariharan, Kathryn Monopoli, X. William Yang, Jeffery Carroll, Marian DiFiglia, Neil Aronin, Anastasia Khvorova
Abstract not available
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METTL5-mediated 18S rRNA m6A modification promotes oncogenic mRNA translation and intrahepatic cholangiocarcinoma progression Mol. Ther. (IF 12.4) Pub Date : 2023-09-21 Zihao Dai, Wanjie Zhu, Yingdong Hou, Xinyue Zhang, Xuxin Ren, Kai Lei, Junbin Liao, Haining Liu, Zhihang Chen, Sui Peng, Shaoqiang Li, Shuibin Lin, Ming Kuang
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Comprehensive landscape and future perspective of long noncoding RNAs in non-small cell lung cancer: it takes a village Mol. Ther. (IF 12.4) Pub Date : 2023-09-21 Yong-Qiang Ao, Jian Gao, Jia-Hao Jiang, Hai-Kun Wang, Shuai Wang, Jian-Yong Ding
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Human macrophage migration inhibitory factor potentiates mesenchymal stromal cell efficacy in a clinically relevant model of allergic asthma Mol. Ther. (IF 12.4) Pub Date : 2023-09-20 Ian J. Hawthorne, Hazel Dunbar, Courteney Tunstead, Tamara Schorpp, Daniel J. Weiss, Sara Rolandsson Enes, Claudia C. dos Santos, Michelle E. Armstrong, Seamas C. Donnelly, Karen English
Current asthma therapies focus on reducing symptoms but fail to restore existing structural damage. Mesenchymal stromal cell (MSC) administration can ameliorate airway inflammation and reverse airway remodelling. However, differences in patient disease microenvironments seem to influence MSC therapeutic effects. Polymorphic CATT tetranucleotide repeat at position 794 of the human macrophage migration
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Expressing CXCR4 in CAR-T cells Suppresses MDSCs Recruitment via STAT3/NF-κB/SDF-1α axis to enhance Anti-tumor Efficacy against Pancreatic Cancer Mol. Ther. (IF 12.4) Pub Date : 2023-09-20 Sun Ruixin, Sun Yansha, Wu Chuanlong, Liu Yifan, Zhou Min, Dong Yiwei, Du Guoxiu, Luo Hong, Shi Bizhi, Jiang Hua, Li Zonghai
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Targeting PRMT1 prevents acute and chronic graft versus host disease Mol. Ther. (IF 12.4) Pub Date : 2023-09-20 Xiaoyan Zhao, Yan Sun, Ziwei Xu, Li Cai, Yu Hu, Huafang Wang
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A potential novel therapy addressing RNA oxidative damage in AKI Mol. Ther. (IF 12.4) Pub Date : 2023-09-20 Cheng Wan, Chun Zhang
Abstract not available
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Optimal delivery of RNA interference by viral vectors for cancer therapy Mol. Ther. (IF 12.4) Pub Date : 2023-09-20 Boaz Wong, Rayanna Birtch, Reza Rezaei, Taylor Jamieson, Mathieu Crupi, Jean-Simon Diallo, Carolina S. Ilkow
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Gene augmentation in FAM161A ciliopathy: Toward functional vision rescue Mol. Ther. (IF 12.4) Pub Date : 2023-09-19 José-Alain Sahel, Katia Marazova, Deniz Dalkara
Abstract not available
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Nanofitins and their applications in human health and lung diseases Mol. Ther. (IF 12.4) Pub Date : 2023-09-19 Federico Ávila-Moreno
Abstract not available
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Prospects and challenges of in vivo hematopoietic stem cell genome editing for hemoglobinopathies Mol. Ther. (IF 12.4) Pub Date : 2023-09-19 André Lieber, Hans-Peter Kiem
Abstract not available
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Refining chimeric antigen receptors via barcoded protein domain combination pooled screening Mol. Ther. (IF 12.4) Pub Date : 2023-09-13 Xavier Rios, Osmay Pardias, Marc A. Morales, Pradyot Bhattacharya, Yibin X. Chen, Linjie Guo, Chunchao Zhang, Erica J. Di Pierro, Gengwen Tian, Gabriel A. Barragan, Pavel Sumazin, Leonid S. Metelitsa
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Myelodysplasia after clonal hematopoiesis with APOBEC3-mediated CYBB inactivation in retroviral gene therapy for X-CGD Mol. Ther. (IF 12.4) Pub Date : 2023-09-13 Toru Uchiyama, Toshinao Kawai, Kazuhiko Nakabayashi, Yumiko Nakazawa, Fumihiro Goto, Kohji Okamura, Toyoki Nishimura, Koji Kato, Nobuyuki Watanabe, Akane Miura, Toru Yasuda, Yukiko Ando, Tomoko Minegishi, Kaori Edasawa, Marika Shimura, Yumi Akiba, Aiko Sato-Otsubo, Tomoyuki Mizukami, Motohiro Kato, Koichi Akashi, Masafumi Onodera
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Asparagine endopeptidase protects podocytes in adriamycin-induced nephropathy by regulating actin dynamics through cleaving transgelin Mol. Ther. (IF 12.4) Pub Date : 2023-09-09 Yang Qiu, Chuntao Lei, Jieyu Zeng, Yaru Xie, Yilin Cao, Qian Yuan, Hua Su, Zhentao Zhang, Chun Zhang
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Development of AAV-delivered broadly neutralizing anti-human ACE2 antibodies against SARS-CoV-2 variants Mol. Ther. (IF 12.4) Pub Date : 2023-09-09 Cheng-Pu Sun, Chi-Wen Chiu, Ping-Yi Wu, Szu-I Tsung, I-Jung Lee, Chih-Wei Hu, Min-Feng Hsu, Tzu-Jiun Kuo, Yu-Hua Lan, Li-Yao Chen, Hui-Yee Ng, Meng-Jhe Chung, Hsin-Ni Liao, Sheng-Che Tseng, Chia-Hui Lo, Yung-Jiun Chen, Chun-Che Liao, Chih-Shin Chang, Jian-Jong Liang, Piotr Draczkowski, Mi-Hua Tao
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In Vivo CRISPR Gene Editing in Patients with Herpes Stromal Keratitis Mol. Ther. (IF 12.4) Pub Date : 2023-08-31 Anji Wei, Di Yin, Zimeng Zhai, Sikai Ling, Huangying Le, Lijia Tian, Jianjiang Xu, Soren R. Paludan, Yujia Cai, Jiaxu Hong
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Inhalable Nanofitin demonstrates high neutralization of SARS-CoV-2 virus via direct application in respiratory tract Mol. Ther. (IF 12.4) Pub Date : 2023-08-30 Sébastien Viollet, Elise Enouf, Justine Picot, Léna Noël, Simon Huet, Déborah Le Pennec, Thomas Sécher, Nathalie Heuzé-Vourc’h, Olivier Kitten, Mathieu Cinier
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Engineered arylsulfatase A with increased activity, stability and brain delivery for therapy of metachromatic leukodystrophy Mol. Ther. (IF 12.4) Pub Date : 2023-08-29 Claudia Yaghootfam, Marc Sylvester, Boris Turk, Volkmar Gieselmann, Ulrich Matzner
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MicroRNA137-loaded lipid nanoparticles regulate synaptic proteins in the prefrontal cortex Mol. Ther. (IF 12.4) Pub Date : 2023-08-28 Michelle C. Palumbo, Milan Gautam, Alex Sonneborn, Kilsun Kim, Phillip A. Wilmarth, Ashok P. Reddy, Xiao Shi, Daniel L. Marks, Gaurav Sahay, Atheir I. Abbas, Aaron Janowsky
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Atractylodinol prevents pulmonary fibrosis through inhibiting TGF-β receptor 1 recycling by stabilizing vimentin Mol. Ther. (IF 12.4) Pub Date : 2023-08-28 Mengjiao Hao, Zhuoji Guan, Zhikang Zhang, Haopeng Ai, Xing Peng, Huihao Zhou, Jun Xu, Qiong Gu
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Chemerin triggers migration of a CD8 T cell subset with natural killer cell functions Mol. Ther. (IF 12.4) Pub Date : 2023-08-28 Romain Ballet, Melissa LaJevic, Noelle Huskey-Mullin, Rachel Roach, Kevin Brulois, Ying Huang, Muhammad A. Saeed, Ha X. Dang, Russell K. Pachynski, Elizabeth Wilson, Eugene C. Butcher, Brian A. Zabel
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FAP-retargeted Ad5 enables in vivo gene delivery to stromal cells in the tumor microenvironment Mol. Ther. (IF 12.4) Pub Date : 2023-08-28 K.Patricia Hartmann, Merel van Gogh, Patrick C. Freitag, Florian Kast, Gabriela Nagy-Davidescu, Lubor Borsig, Andreas Plückthun
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AAV9-mediated SH3TC2 gene replacement therapy targeted to Schwann cells for the treatment of CMT4C Mol. Ther. (IF 12.4) Pub Date : 2023-08-28 Elena Georgiou, Alexia Kagiava, Irene Sargiannidou, Natasa Schiza, Marina Stavrou, Jan Richter, Christina Tryfonos, Amanda Heslegrave, Henrik Zetterberg, Christina Christodoulou, Kleopas A. Kleopa
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CANT1 lncRNA Triggers Efficient Therapeutic Efficacy by Correcting Aberrant lncing Cascade in Malignant Uveal Melanoma Mol. Ther. (IF 12.4) Pub Date : 2023-08-26 Yue Xing, Xuyang Wen, Xia Ding, Jiayan Fan, Peiwei Chai, Renbing Jia, Shengfang Ge, Guanxiang Qian, He Zhang, Xianqun Fan
Abstract not available
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AMPK activation coupling SENP1-Sirt3 axis protects against acute kidney injury Mol. Ther. (IF 12.4) Pub Date : 2023-08-21 Minyan Zhu, Jianli He, Yao Xu, Yong Zuo, Wenyan Zhou, Zhiying Yue, Xinghua Shao, Jinke Cheng, Tianshi Wang, Shan Mou
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Advancing in vivo genome editing: B cell engineering via adenoviral delivery systems Mol. Ther. (IF 12.4) Pub Date : 2023-08-23 Amaia Cadinanos-Garai, Mohamed Abou-el-Enein
Abstract not available
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CRISPR-Cas9 as a Trojan horse Mol. Ther. (IF 12.4) Pub Date : 2023-08-21 Daniel F.M. Monte
Abstract not available
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LIGHTing CAR T in the tumor microenvironment Mol. Ther. (IF 12.4) Pub Date : 2023-08-21 Yong Liang, Yang-Xin Fu
Abstract not available
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The Molecular Therapy family of journals continues to lead the field of gene and cell therapy Mol. Ther. (IF 12.4) Pub Date : 2023-08-21 Courtney Bricker-Anthony, Roland W. Herzog
Abstract not available
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Epithelioid Neoplasm of the Spinal Cord in a Child with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Mol. Ther. (IF 12.4) Pub Date : 2023-08-19 Laura Retson, Nishant Tiwari, Jennifer Vaughn, Saunder Bernes, P. David Adelson, Keith Mansfield, Silvana Libertini, Brent Kuzmiski, Iulian Alecu, Richard Gabriel, Ross Mangum
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Challenges in accelerated approvals for gene therapies Mol. Ther. (IF 12.4) Pub Date : 2023-08-19 Rafael Escandon, John Lantos
Abstract not available
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Implementation of a gene therapy education initiative by the ASGCT and Muhimbili University of Health and Allied Sciences Mol. Ther. (IF 12.4) Pub Date : 2023-08-17 Kenneth Cornetta, Samantha Kay, Florence Urio, Irene Kida Minja, Erasto Mbugi, Josephine Mgaya, Teddy Mselle, Siana Nkya, Mohamed Zahir Alimohamed, Kinuma Ndaki, Martín Bonamino, Richard C. Koya, Lesha D. Shah, Johnny Mahlangu, Daniela Drago, Savita Rangarajan, Giridhara Rao Jayandharan
There has been rapid growth in gene therapy development with an expanding list of approved clinical products. Several therapies are particularly relevant to patients in low- and middle-income countries. Moreover, investing in research and manufacturing presents an opportunity for economic development. To increase awareness of gene therapy, the American Society of Gene and Cell Therapy partnered with
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Response to: DNA transposon mechanisms and pathways of genotoxicity Mol. Ther. (IF 12.4) Pub Date : 2023-08-14 Blair B. Madison, Devon J. Shedlock
Abstract not available
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Gene Augmentation Therapy Attenuates Retinal Degeneration in a Knock-Out Mouse Model of Fam161a Retinitis Pigmentosa Mol. Ther. (IF 12.4) Pub Date : 2023-08-14 Chen Matsevich, Prakadeeswari Gopalakrishnan, Ning Chang, Alexey Obolensky, Avigail Beryozkin, Manar Salameh, Corinne Kostic, Dror Sharon, Yvan Arsenijevic, Eyal Banin
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Human cochlear diffusion from the cerebrospinal fluid space with gadolinium contrast Mol. Ther. (IF 12.4) Pub Date : 2023-08-15 Douglas J. Totten, Kevin T.A. Booth, Kristine M. Mosier, Evan C. Cumpston, Cody Whitted, Vanessa Okechuku, Nicholas A. Koontz, Rick F. Nelson
Abstract not available
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Adenovirus-mediated combined cytotoxic and immune-stimulatory gene therapy using dual-vector Ad-hCMV-TK and Ad-hCMV-Flt3L: translational explorations from rodents to glioma patients Mol. Ther. (IF 12.4) Pub Date : 2023-08-12 Syed M. Faisal, Maria G. Castro, Pedro R. Lowenstein
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CD90-targeted lentiviral vectors for HSC gene therapy Mol. Ther. (IF 12.4) Pub Date : 2023-08-07 Kurt Berckmueller, Justin Thomas, Eman A. Taha, Seunga Choo, Ravishankar Madhu, Greta Kanestrom, Peter B. Rupert, Roland Strong, Hans-Peter Kiem, Stefan Radtke
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Darwin’s gemmules and extracellular vesicles: A striking resemblance Mol. Ther. (IF 12.4) Pub Date : 2023-08-03 Songlin He, Yongsheng Liu
Abstract not available
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Response to a letter to the editor on Darwin’s gemmules and extracellular vesicles: A striking resemblance Mol. Ther. (IF 12.4) Pub Date : 2023-08-03 Owen G. Davies
Abstract not available
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LION: Taming RNA vaccine inflammation Mol. Ther. (IF 12.4) Pub Date : 2023-08-03 John S. Tregoning
Abstract not available
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Single-cell dissection of cellular and molecular features underlying mesenchymal stem cell therapy in ischemic acute kidney injury Mol. Ther. (IF 12.4) Pub Date : 2023-08-02 Wenjuan Wang, Min Zhang, Xuejing Ren, Yanqi Song, Yue Xu, Kaiting Zhuang, Tuo Xiao, Xinru Guo, Siyang Wang, Quan Hong, Zhe Feng, Xiangmei Chen, Guangyan Cai
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Stowaways in the cargo: Contaminating nucleic acids in rAAV preparations for gene therapy. Mol. Ther. (IF 12.4) Pub Date : 2023-08-02 Mark A. Brimble, Stephen M. Winston, Andrew M. Davidoff
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STING/ACSL4 axis-dependent ferroptosis and inflammation promote hypertension-associated chronic kidney disease Mol. Ther. (IF 12.4) Pub Date : 2023-08-02 Li Gao, Junsheng Zhang, Tingting Yang, Ling Jiang, Xueqi Liu, Sheng Wang, Xian Wang, Yuebo Huang, Huaying Wang, Mengya Zhang, Tingting Gong, Lijuan Ma, Chao Li, Chaoyong He, Xiao-ming Meng, Yonggui Wu
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Retraction Notice to: Neurotrophin 3 Transduction Augments Remyelinating and Immunomodulatory Capacity of Neural Stem Cells Mol. Ther. (IF 12.4) Pub Date : 2023-08-02 Jingxian Yang, Yaping Yan, Yang Xia, Tingguo Kang, Xing Li, Bogoljub Ciric, Hui Xu, Abdolmohamad Rostami, Guang-Xian Zhang
Abstract not available
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Development of an mRNA-lipid nanoparticle vaccine against Lyme disease Mol. Ther. (IF 12.4) Pub Date : 2023-08-02 Matthew Pine, Gunjan Arora, Thomas M. Hart, Emily Bettini, Brian T. Gaudette, Hiromi Muramatsu, István Tombácz, Taku Kambayashi, Ying K. Tam, Dustin Brisson, David Allman, Michela Locci, Drew Weissman, Erol Fikrig, Norbert Pardi
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Targeting leucine-rich repeat serine/threonine-protein kinase 2 sensitizes anti-PD-L1 immunotherapy of pancreatic ductal adenocarcinoma Mol. Ther. (IF 12.4) Pub Date : 2023-07-28 Kang Sun, Xiaozhen Zhang, Mengyi Lao, Lihong He, Sicheng Wang, Hanshen Yang, Jian Xu, Jianghui Tang, Zhengtao Hong, Jinyuan Song, Chengxiang Guo, Muchun Li, Xinyuan Liu, Yan Chen, Hanjia Zhang, Jingxing Zhou, Jieru Lin, Sirui Zhang, Yifan Hong, Jinyan Huang, Xueli Bai
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Liver Injury in Cynomolgus Monkeys Following Intravenous and Intrathecal scAAV9 Gene Therapy Delivery Mol. Ther. (IF 12.4) Pub Date : 2023-07-28 Eloise Hudry, Fumiaki Aihara, Emily Meseck, Keith Mansfield, Cameron McElroy, Deepa Chand, Francis Fonyuy Tukov, Kelley Penraat
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Innovative stem cell-based strategies for corneal wound healing: A step forward Mol. Ther. (IF 12.4) Pub Date : 2023-07-26 Sharmila Fagoonee, Gabriele Saccu, Benedetta Bussolati
Abstract not available
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Dual-locus, dual-HDR editing permits efficient generation of antigen-specific, regulatory T cells with robust suppressive activity Mol. Ther. (IF 12.4) Pub Date : 2023-07-22 Martina S. Hunt, Soo Jung Yang, Emma Mortensen, Ahmad Boukhris, Jane Buckner, Peter J. Cook, David J. Rawlings
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PARP1 modulates METTL3 promoter chromatin accessibility and associated LPAR5 RNA m6A methylation to control cancer cell radiosensitivity Mol. Ther. (IF 12.4) Pub Date : 2023-07-23 Xiaoya Sun, Chenjun Bai, Haozheng Li, Dafei Xie, Shi Chen, Yang Han, Jinhua Luo, Yang Li, Yumeng Ye, Jin Jia, Xin Huang, Hua Guan, Dingxin Long, Ruixue Huang, Shanshan Gao, Ping-Kun Zhou
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Combined AAV-mediated gene replacement therapy improves auditory function in a mouse model of human DFNB42 deafness Mol. Ther. (IF 12.4) Pub Date : 2023-07-22 Kevin Isgrig, Alexander X. Cartagena-Rivera, Hong Jun Wang, Mhamed Grati, Katharine A. Fernandez, Thomas B. Friedman, Inna A. Belyantseva, Wade Chien
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Novel MECP2 gene therapy is effective in a multicenter study using two mouse models of Rett syndrome and is safe in non-human primates Mol. Ther. (IF 12.4) Pub Date : 2023-07-22 Samantha Powers, Shibi Likhite, Kamal K. Gadalla, Carlos J. Miranda, Amy J. Huffenberger, Cassandra Dennys, Kevin D. Foust, Pablo Morales, Christopher R. Pierson, Federica Rinaldi, Stephanie Perry, Brad Bolon, Nicolas Wein, Stuart Cobb, Brian K. Kaspar, Kathrin C. Meyer
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Inhibition of complement C3 signaling ameliorates locomotor and visual dysfunction in autoimmune inflammatory diseases Mol. Ther. (IF 12.4) Pub Date : 2023-07-22 Li Xu, Huiming Xu, Siqi Chen, Wei Jiang, Shabbir Khan Afridi, Yuge Wang, Xin Ren, Yipeng Zhao, Shuiqing Lai, Xiusheng Qiu, Yu-Wen Alvin Huang, Yaxiong Cui, Hui Yang, Wei Qiu, Changyong Tang
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Bypassing the cytokine sink: IL-2-independent IL-2 receptor signaling Mol. Ther. (IF 12.4) Pub Date : 2023-07-22 Tim Luetkens
Abstract not available
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Efficient T cell adoptive transfer in lymphoreplete hosts mediated by transient activation of Stat5 signaling Mol. Ther. (IF 12.4) Pub Date : 2023-07-21 Megan D. Tennant, Christina New, Leonardo M.R. Ferreira, Richard T. O’Neil
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Tailoring MSCs with modified mRNA for the eye Mol. Ther. (IF 12.4) Pub Date : 2023-07-19 Joo Youn Oh
Abstract not available
更新日期：2023-09-23
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