Molecular therapy : the journal of the American Society of Gene Therapy

Keyphrases

Publications

volume 32, number 8, 2024

Yixian Cun, Wenbing Guo, Biao Ma, Yasushi Okuno, Jinkai Wang
Decoding the specificity of m 6 A RNA methylation and its implication in cancer therapy. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Immacolata Porreca, Robert Blassberg, Jennifer Harbottle, Bronwyn Joubert, Olga Mielczarek, Jesse Stombaugh, Kevin Hemphill, Jonathan Sumner, Deividas Pazeraitis, Julia Liz Touza, Margherita Francescatto, Mike Firth, Tommaso Selmi, Juan Carlos Collantes, Zaklina Strezoska, Benjamin Taylor, Shengkan Jin, Ceri M Wiggins, Anja van Brabant Smith, John J Lambourne
An aptamer-mediated base editing platform for simultaneous knockin and multiple gene knockout for allogeneic CAR-T cells generation. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Yating Zhao, Yongpu Feng, Fengyuan Sun, Lei Li, Jiayu Chen, Yingxiao Song, Wenbo Zhu, Xiulin Hu, Zhaoshen Li, Fanyang Kong, Yiqi Du, Xiangyu Kong
Optimized rAAV8 targeting acinar KLF4 ameliorates fibrosis in chronic pancreatitis via exosomes-enriched let-7s suppressing pancreatic stellate cells activation. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Rocio Sartori-Maldonado, Hossam Montaser, Inkeri Soppa, Solja Eurola, Juhana Juutila, Melanie Balaz, Henri Puttonen, Timo Otonkoski, Jonna Saarimäki-Vire, Kirmo Wartiovaara
Thymidylate synthase disruption to limit cell proliferation in cell therapies. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Piyush Gondaliya, Adil Ali Sayyed, Irene K Yan, Julia Driscoll, Abbye Ziemer, Tushar Patel
Targeting PD-L1 in cholangiocarcinoma using nanovesicle-based immunotherapy. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Caiqi Zhao, Jie Chen, Zhihua Liu, Huabin Liang, Xiaoyan Chen, Lianping Cheng, Shitao Xie, Zhekai Lin, Renlan Wu, Qi Zhao, Yue Xue, Xiaoyun Lai, Xia Jin, Jin-Fu Xu, Xiao Su
Activation of nicotinic acetylcholine receptor α7 subunit limits Zika viral infection via promoting autophagy and ferroptosis. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Carlos Javier Alméciga-Díaz
The first medical education program on gene and gene-modified cell therapies for Latin America. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Saru Basnet, Mirte Van der Heijden, Dafne C A Quixabeira, Elise Jirovec, Susanna A M Grönberg-Vähä-Koskela, James H A Clubb, Anna Kanerva, Santeri Pakola, Lyna Haybout, Victor Arias, Otto Hemminki, Tatiana Kudling, Sadia Zafar, Victor Cervera-Carrascon, Joao M Santos, Akseli Hemminki
Overcoming effector T cell exhaustion in ovarian cancer ascites with a novel adenovirus encoding for a MUC1 bispecific antibody engager and IL-2 cytokine. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Liya Ma, Kaiwen Zhang, Jian Xu, Jian Wang, Ting Jiang, Xiaolong Du, Jiaxin Zhang, Jing Huang, Fengyi Ren, Dong Liu, Weiwei Xue, Dongxu Kan, Mengjiao Yao, Yutian Liang, Hongxing Jason-Sun
Building a novel TRUCK by harnessing the endogenous IFN-gamma promoter for cytokine expression. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Lan Zhao, Yumei Lai, Hongli Jiao, Jun Li, Ke Lu, Jian Huang
CRISPR-mediated Sox9 activation and RelA inhibition enhance cell therapy for osteoarthritis. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Yuang Zhang, Chengzhen Liang, Haibin Xu, Yi Li, Kaishun Xia, Liyin Wang, Xianpeng Huang, Jiangjie Chen, Jiawei Shu, Feng Cheng, Kesi Shi, Jingkai Wang, Yiqing Tao, Shaoke Wang, Yongxiang Zhang, Hao Li, Shoumin Feng, Fangcai Li, Xiaopeng Zhou, Qixin Chen
Dedifferentiation-like reprogramming of degenerative nucleus pulposus cells into notochordal-like cells by defined factors. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Christine Tkaczyk, Michael Newton, Mun Mun Patnaik, George Thom, Martin Strain, Adam Gamson, Olalekan Daramola, Andal Murthy, Julie Douthwaite, Oleg Stepanov, Elin Boger, Haitao Yang, Mark T Esser, Ashley Lidwell, Antonio DiGiandomenico, Luis Santos, Bret R Sellman
In vivo mRNA expression of a multi-mechanistic mAb combination protects against Staphylococcus aureus infection. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Lan Zhao, Yumei Lai, Hongli Jiao, Jun Li, Ke Lu, Jian Huang
CRISPR-mediated Sox9 activation and RelA inhibition enhance cell therapy for osteoarthritis. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Samer Al Hadidi, Helen E Heslop, Malcolm K Brenner, Masataka Suzuki
Bispecific antibodies and autologous chimeric antigen receptor T cell therapies for treatment of hematological malignancies. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Piyush Gondaliya, Adil Ali Sayyed, Irene K Yan, Julia Driscoll, Abbye Ziemer, Tushar Patel
Targeting PD-L1 in cholangiocarcinoma using nanovesicle-based immunotherapy. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Aravindhan Veerapandiyan, Ruthwik Duvuru
Transforming care for spinal muscular atrophy: A critical look at treatment paradigms. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Sylwia Bobis-Wozowicz, Pawel E Ferdek
Harnessing extracellular vesicles for pancreatic fibrosis therapy. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Yiying Li, Junting Zhang, Hanliang Sun, Yujie Chen, Wendi Li, Xiufeng Yu, Xijuan Zhao, Lixin Zhang, Jianfeng Yang, Wei Xin, Yuan Jiang, Guilin Wang, Wenbin Shi, Daling Zhu
lnc-Rps4l-encoded peptide RPS4XL regulates RPS6 phosphorylation and inhibits the proliferation of PASMCs caused by hypoxia. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Sung Eun Wang, Yan Xiong, Mi-Ae Jang, Kwang-Su Park, Meaghan Donahue, Julia Velez, Jian Jin, Yong-Hui Jiang
Newly developed oral bioavailable EHMT2 inhibitor as a potential epigenetic therapy for Prader-Willi syndrome. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Yuang Zhang, Chengzhen Liang, Haibin Xu, Yi Li, Kaishun Xia, Liyin Wang, Xianpeng Huang, Jiangjie Chen, Jiawei Shu, Feng Cheng, Kesi Shi, Jingkai Wang, Yiqing Tao, Shaoke Wang, Yongxiang Zhang, Hao Li, Shoumin Feng, Fangcai Li, Xiaopeng Zhou, Qixin Chen
Dedifferentiation-like reprogramming of degenerative nucleus pulposus cells into notochordal-like cells by defined factors. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Tessy A H Hick, Corinne Geertsema, Wilson Nguyen, Cameron R Bishop, Linda van Oosten, Sandra R Abbo, Troy Dumenil, Frank J M van Kuppeveld, Martijn A Langereis, Daniel J Rawle, Bing Tang, Kexin Yan, Monique M van Oers, Andreas Suhrbier, Gorben P Pijlman
Safety concern of recombination between self-amplifying mRNA vaccines and viruses is mitigated in vivo. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Rocio Sartori-Maldonado, Hossam Montaser, Inkeri Soppa, Solja Eurola, Juhana Juutila, Melanie Balaz, Henri Puttonen, Timo Otonkoski, Jonna Saarimäki-Vire, Kirmo Wartiovaara
Thymidylate synthase disruption to limit cell proliferation in cell therapies. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Patricia Lam, Deborah A Zygmunt, Anna Ashbrook, Macey Bennett, Tatyana A Vetter, Paul T Martin
Dual FKRP/FST gene therapy normalizes ambulation, increases strength, decreases pathology, and amplifies gene expression in LGMDR9 mice. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Samer Al Hadidi, Helen E Heslop, Malcolm K Brenner, Masataka Suzuki
Bispecific antibodies and autologous chimeric antigen receptor T cell therapies for treatment of hematological malignancies. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Caiqi Zhao, Jie Chen, Zhihua Liu, Huabin Liang, Xiaoyan Chen, Lianping Cheng, Shitao Xie, Zhekai Lin, Renlan Wu, Qi Zhao, Yue Xue, Xiaoyun Lai, Xia Jin, Jin-Fu Xu, Xiao Su
Activation of nicotinic acetylcholine receptor α7 subunit limits Zika viral infection via promoting autophagy and ferroptosis. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Molly A McPheron, Marcia V Felker
Clinical perspectives: Treating spinal muscular atrophy. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Yixian Cun, Wenbing Guo, Biao Ma, Yasushi Okuno, Jinkai Wang
Decoding the specificity of m 6 A RNA methylation and its implication in cancer therapy. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Tessy A H Hick, Corinne Geertsema, Wilson Nguyen, Cameron R Bishop, Linda van Oosten, Sandra R Abbo, Troy Dumenil, Frank J M van Kuppeveld, Martijn A Langereis, Daniel J Rawle, Bing Tang, Kexin Yan, Monique M van Oers, Andreas Suhrbier, Gorben P Pijlman
Safety concern of recombination between self-amplifying mRNA vaccines and viruses is mitigated in vivo. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Liya Ma, Kaiwen Zhang, Jian Xu, Jian Wang, Ting Jiang, Xiaolong Du, Jiaxin Zhang, Jing Huang, Fengyi Ren, Dong Liu, Weiwei Xue, Dongxu Kan, Mengjiao Yao, Yutian Liang, Hongxing Jason-Sun
Building a novel TRUCK by harnessing the endogenous IFN-gamma promoter for cytokine expression. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Marco Cortese, Erica Torchiaro, Alice D'Andrea, Consalvo Petti, Federica Invrea, Letizia Franco, Chiara Donini, Valeria Leuci, Simonetta Maria Leto, Valentina Vurchio, Francesca Cottino, Claudio Isella, Sabrina Arena, Elisa Vigna, Andrea Bertotti, Livio Trusolino, Dario Sangiolo, Enzo Medico
Preclinical efficacy of a HER2 synNotch/CEA-CAR combinatorial immunotherapy against colorectal cancer with HER2 amplification. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Christine Tkaczyk, Michael Newton, Mun Mun Patnaik, George Thom, Martin Strain, Adam Gamson, Olalekan Daramola, Andal Murthy, Julie Douthwaite, Oleg Stepanov, Elin Boger, Haitao Yang, Mark T Esser, Ashley Lidwell, Antonio DiGiandomenico, Luis Santos, Bret R Sellman
In vivo mRNA expression of a multi-mechanistic mAb combination protects against Staphylococcus aureus infection. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Killian S Hanlon, Ming Cheng, Roberto Montoro Ferrer, Jae Ryun Ryu, Boram Lee, Demitri De La Cruz, Nikita Patel, Paula Espinoza, Miguel C Santoscoy, Yi Gong, Carrie Ng, Diane M Nguyen, Josette Nammour, Sean W Clark, Vivi M Heine, Woong Sun, Karen Kozarsky, Casey A Maguire
In vivo selection in non-human primates identifies AAV capsids for on-target CSF delivery to spinal cord. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Chun-Liang Yang, Fa-Xi Wang, Jia-Hui Luo, Shan-Jie Rong, Wan-Ying Lu, Qi-Jie Chen, Jun Xiao, Ting Wang, Dan-Ni Song, Jing Liu, Qian Mo, Shuo Li, Yu Chen, Ya-Nan Wang, Yan-Jun Liu, Tong Yan, Wei-Kuan Gu, Shu Zhang, Fei Xiong, Qi-Lin Yu, Zi-Yun Zhang, Ping Yang, Shi-Wei Liu, Decio Eizirik, Ling-Li Dong, Fei Sun, Cong-Yi Wang
PDIA3 orchestrates effector T cell program by serving as a chaperone to facilitate the non-canonical nuclear import of STAT1 and PKM2. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Octavio A Trejo-Villegas, Irene H Heijink, Federico Ávila-Moreno
Preclinical evidence in the assembly of mammalian SWI/SNF complexes: Epigenetic insights and clinical perspectives in human lung disease therapy. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Roland W Herzog, Rory Bricker-Anthony
Molecular Therapy's growing influence. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Li Ding, Bin Deng, Gang Chen
RNA therapeutics targeting PD-L1 is a promising immune-activation strategy against difficult-to-treat cancers. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Irafasha C Casmil, Anna K Blakney
Can self-amplifying RNA vaccines and viruses exchange genetic material? Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Rob W J Collin, Bart P Leroy
In vivo genome editing for inherited retinal disease: Opportunities and challenges. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Tyler F Hill, Parnal Narvekar, Gregory D Asher, Jasmine N Edelstein, Nathan D Camp, Annaiz Grimm, Kerri R Thomas, Michael D Leiken, Katherine M Molloy, Peter J Cook, Sean P Arlauckas, Richard A Morgan, Sarah K Tasian, David J Rawlings, Richard G James
Human plasma cells engineered to secrete bispecifics drive effective in vivo leukemia killing. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
John S Tregoning
The tricky second album: Licensure of an mRNA vaccine for respiratory syncytial virus. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Ying Gong, Wilfred T V Germeraad, Xulin Zhang, Nisha Wu, Bo Li, Lynn Janssen, Zongzhong He, Marion J J Gijbels, Bodeng Wu, Birgit L M G Gijsbers, Timo I Olieslagers, Gerard M J Bos, Lei Zheng, Roel G J Klein Wolterink
NKG2A genetic deletion promotes human primary NK cell anti-tumor responses better than an anti-NKG2A monoclonal antibody. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Patricia Lam, Deborah A Zygmunt, Anna Ashbrook, Macey Bennett, Tatyana A Vetter, Paul T Martin
Dual FKRP/FST gene therapy normalizes ambulation, increases strength, decreases pathology, and amplifies gene expression in LGMDR9 mice. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Molly A McPheron, Marcia V Felker
Clinical perspectives: Treating spinal muscular atrophy. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Marco Cortese, Erica Torchiaro, Alice D'Andrea, Consalvo Petti, Federica Invrea, Letizia Franco, Chiara Donini, Valeria Leuci, Simonetta Maria Leto, Valentina Vurchio, Francesca Cottino, Claudio Isella, Sabrina Arena, Elisa Vigna, Andrea Bertotti, Livio Trusolino, Dario Sangiolo, Enzo Medico
Preclinical efficacy of a HER2 synNotch/CEA-CAR combinatorial immunotherapy against colorectal cancer with HER2 amplification. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Ying Gong, Wilfred T V Germeraad, Xulin Zhang, Nisha Wu, Bo Li, Lynn Janssen, Zongzhong He, Marion J J Gijbels, Bodeng Wu, Birgit L M G Gijsbers, Timo I Olieslagers, Gerard M J Bos, Lei Zheng, Roel G J Klein Wolterink
NKG2A genetic deletion promotes human primary NK cell anti-tumor responses better than an anti-NKG2A monoclonal antibody. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Immacolata Porreca, Robert Blassberg, Jennifer Harbottle, Bronwyn Joubert, Olga Mielczarek, Jesse Stombaugh, Kevin Hemphill, Jonathan Sumner, Deividas Pazeraitis, Julia Liz Touza, Margherita Francescatto, Mike Firth, Tommaso Selmi, Juan Carlos Collantes, Zaklina Strezoska, Benjamin Taylor, Shengkan Jin, Ceri M Wiggins, Anja van Brabant Smith, John J Lambourne
An aptamer-mediated base editing platform for simultaneous knockin and multiple gene knockout for allogeneic CAR-T cells generation. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Octavio A Trejo-Villegas, Irene H Heijink, Federico Ávila-Moreno
Preclinical evidence in the assembly of mammalian SWI/SNF complexes: Epigenetic insights and clinical perspectives in human lung disease therapy. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Killian S Hanlon, Ming Cheng, Roberto Montoro Ferrer, Jae Ryun Ryu, Boram Lee, Demitri De La Cruz, Nikita Patel, Paula Espinoza, Miguel C Santoscoy, Yi Gong, Carrie Ng, Diane M Nguyen, Josette Nammour, Sean W Clark, Vivi M Heine, Woong Sun, Karen Kozarsky, Casey A Maguire
In vivo selection in non-human primates identifies AAV capsids for on-target CSF delivery to spinal cord. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Chun-Liang Yang, Fa-Xi Wang, Jia-Hui Luo, Shan-Jie Rong, Wan-Ying Lu, Qi-Jie Chen, Jun Xiao, Ting Wang, Dan-Ni Song, Jing Liu, Qian Mo, Shuo Li, Yu Chen, Ya-Nan Wang, Yan-Jun Liu, Tong Yan, Wei-Kuan Gu, Shu Zhang, Fei Xiong, Qi-Lin Yu, Zi-Yun Zhang, Ping Yang, Shi-Wei Liu, Decio Eizirik, Ling-Li Dong, Fei Sun, Cong-Yi Wang
PDIA3 orchestrates effector T cell program by serving as a chaperone to facilitate the non-canonical nuclear import of STAT1 and PKM2. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)
Sung Eun Wang, Yan Xiong, Mi-Ae Jang, Kwang-Su Park, Meaghan Donahue, Julia Velez, Jian Jin, Yong-Hui Jiang
Newly developed oral bioavailable EHMT2 inhibitor as a potential epigenetic therapy for Prader-Willi syndrome. Molecular therapy : the journal of the American Society of Gene Therapy 32 (8) (2024)