-
mRNA-LNP vaccine-induced CD8+ T cells protect mice from lethal SARS-CoV-2 infection in the absence of specific antibodies Mol. Ther. (IF 12.4) Pub Date : 2024-04-11 Brian Montoya, Carolina R. Melo-Silva, Lingjuan Tang, Samita Kafle, Peter Lidskiy, Csaba Bajusz, Máté Vadovics, Hiromi Muramatsu, Edit Abraham, Zoltan Lipinszki, Debotri Chatterjee, Gabrielle Scher, Juliana Benitez, Molly M.H. Sung, Ying K. Tam, Nicholas J. Catanzaro, Alexandra Schäfer, Raul Andino, Ralph S. Baric, David R. Martinez, Norbert Pardi, Luis J. Sigal
The role of CD8 T cells in SARS-CoV-2 pathogenesis or mRNA-LNP vaccine-induced protection from lethal COVID-19 is unclear. Using mouse-adapted SARS-CoV-2 virus (MA30) in C57BL/6 mice, we show that CD8 T cells are unnecessary for the intrinsic resistance of female or the susceptibility of male mice to lethal SARS-CoV-2 infection. Also, mice immunized with a di-proline prefusion-stabilized full-length
-
Transient TCR-based T cell therapy in a patient with advanced treatment-resistant MSI-high colorectal cancer Mol. Ther. (IF 12.4) Pub Date : 2024-04-06 Solrun Melkorka Maggadottir, Svein Dueland, Nadia Mensali, Hanne Hamre, Per Arne Andresen, Marit Renée Myhre, Hedvig V. Juul, Iris Bigalke, Marianne Lundby, Turid Kirsti Hønnåshagen, Stein Sæbøe-Larssen, Dag Josefsen, Trond Hagtvedt, Sébastien Wälchli, Gunnar Kvalheim, Else Marit Inderberg
-
Identification of memory mechanism in tissue-resident stem cells via ANGPTL4 beyond immune cells upon viral antigen exposure Mol. Ther. (IF 12.4) Pub Date : 2024-04-06 Eun-kyung Min, Soo-Rim Kim, Choon-Mi Lee, Kun-Hee Na, Chan Hum Park, Byung-Chul Oh, YunJae Jung, In-Sun Hong
-
Engineering allorejection-resistant CAR-NKT cells from hematopoietic stem cells for off-the-shelf cancer immunotherapy Mol. Ther. (IF 12.4) Pub Date : 2024-04-06 Yan-Ruide Li, Yang Zhou, Jiaji Yu, Yichen Zhu, Derek Lee, Enbo Zhu, Zhe Li, Yu Jeong Kim, Kuangyi Zhou, Ying Fang, Zibai Lyu, Yuning Chen, Yanxin Tian, Jie Huang, Xinjian Cen, Tiffany Husman, Jae Min Cho, Tzung Hsiai, Jin J. Zhou, Pin Wang, Benjamin R. Puliafito, Sarah M. Larson, Lili Yang
-
Decoding and overcoming T cell exhaustion: Epigenetic and transcriptional dynamics in CAR-T cells against solid tumors Mol. Ther. (IF 12.4) Pub Date : 2024-04-06 Taeyoung Ahn, Eun-Ah Bae, Hyungseok Seo
-
Fast, accurate ranking of engineered proteins by target-binding propensity using structure modeling Mol. Ther. (IF 12.4) Pub Date : 2024-04-06 Xiaozhe Ding, Xinhong Chen, Erin E. Sullivan, Timothy F. Shay, Viviana Gradinaru
-
A p21-ATD mouse model for monitoring and eliminating senescent cells and its application in liver regeneration post injury Mol. Ther. (IF 12.4) Pub Date : 2024-04-06 Miaomiao Chen, Guoxiu Wu, Yanli Lu, Shiwen Sun, Zhao Yu, Xin Pan, Wenjian Chen, Hongyu Xu, Hua Qiu, Weizhi He, Xiuhua Li, Xicheng Wang, Yi Luo, Yuan Du, Jialing Wu, Ke Wei, Wencheng Zhang, Zhongmin Liu, Zhiying He
-
TALEN-mediated intron editing of HSPCs enables transgene expression restricted to the myeloid lineage Mol. Ther. (IF 12.4) Pub Date : 2024-04-06 Eduardo Seclen, Jessica C. Jang, Aminah O. Lawal, Sylvain Pulicani, Alex Boyne, Diane Tkach, Alexandre Juillerat, Philippe Duchateau, Julien Valton
-
Interaction of SMAC/Diablo with a survivin/BIRC5-derived peptide alters essential cancer hallmarks: Tumor growth, inflammation, and immunosuppression Mol. Ther. (IF 12.4) Pub Date : 2024-04-05 M. Santhanam, S. Kumar Pandey, A. Shteinfer-Kuzmine, A. Paul, N. Abusiam, R. Zalk, V. Shoshan-Barmatz
-
Advances and transgressions of nuclear transport checkpoint inhibitors Mol. Ther. (IF 12.4) Pub Date : 2024-04-04 Jacek Hawiger
-
An innate immune response to adeno-associated virus genomes decreases cortical dendritic complexity and disrupts synaptic transmission Mol. Ther. (IF 12.4) Pub Date : 2024-04-01 Christos M. Suriano, Neerav Kumar, Jessica L. Verpeut, Jie Ma, Caroline Jung, Connor E. Dunn, Brigett V. Carvajal, Ai Vy Nguyen, Lisa M. Boulanger
-
Transplantation of dorsal root ganglia overexpressing the NaChBac sodium channel improves locomotion after complete SCI Mol. Ther. (IF 12.4) Pub Date : 2024-03-30 Sonia Hingorani, Guillem Paniagua Soriano, Carlos Sánchez Huertas, Eva María Villalba Riquelme, Eric López Mocholi, Beatriz Martínez Rojas, Ana Alastrué Agudo, Sebastián Dupraz, Antonio Vicente Ferrer Montiel, Victoria Moreno Manzano
-
CRISPR-Cas9n-mediated ELANE promoter editing for gene therapy of severe congenital neutropenia Mol. Ther. (IF 12.4) Pub Date : 2024-03-30 Masoud Nasri, Malte Ritter, Perihan Mir, Benjamin Dannenmann, Masako M. Kaufmann, Patricia Arreba-Tutusaus, Yun Xu, Natalia Borbaran-Bravo, Maksim Klimiankou, Claudia Lengerke, Cornelia Zeidler, Toni Cathomen, Karl Welte, Julia Skokowa
-
Targeting the nuclear long noncoding transcript LSP1P5 abrogates extracellular matrix component deposition by trans-upregulating CEBPA in keloids Mol. Ther. (IF 12.4) Pub Date : 2024-03-28 Shuchen Gu, Xin Huang, Shenying Luo, Yunhan Liu, Yimin Khoong, Hsin Liang, Liying Tu, Ruoqing Xu, En Yang, Yixuan Zhao, Min Yao, Tao Zan
-
RNAi mediated silencing of STAT3/PD-L1 in tumor-associated immune cells induces robust anti-tumor effects in immunotherapy resistant tumors Mol. Ther. (IF 12.4) Pub Date : 2024-03-27 Shanthi Ganesh, Min Ju Kim, Jenny Lee, Xudong Feng, Krisjanis Ule, Amy Mahan, Harini Sivagurunatha Krishnan, Zhe Wang, Maryam Yahyaee Anzahaee, Garima Singhal, Ilia Korboukh, Jennifer A. Lockridge, Laura Sanftner, Rene Rijnbrand, Marc Abrams, Bob D. Brown
-
Evaluation of safety and early efficacy of AAV gene therapy in mouse models of vanishing white matter disease Mol. Ther. (IF 12.4) Pub Date : 2024-03-27 Jessica A. Herstine, Pi-Kai Chang, Sergiy Chornyy, Tamara J. Stevenson, Alex C. Sunshine, Ksenia Nokhrina, Jessica Rediger, Julia Wentz, Tatyana A. Vetter, Erika Scholl, Caleb Holaway, Nettie K. Pyne, Anna Bratasz, Stewart Yeoh, Kevin M. Flanigan, Joshua L. Bonkowsky, Allison M. Bradbury
-
GSNOR overexpression enhances CAR-T cell stemness and anti-tumor function by enforcing mitochondrial fitness Mol. Ther. (IF 12.4) Pub Date : 2024-03-27 Qing Niu, Haixiao Zhang, Fang Wang, Xing Xu, Yuechen Luo, Baolin He, Mingxia Shi, Erlie Jiang, Xiaoming Feng
-
Anti-CD45 PBD-based antibody-drug conjugates are effective targeted conditioning agents for gene therapy and stem cell transplant Mol. Ther. (IF 12.4) Pub Date : 2024-03-27 Jenny Yeung, Aiyin Liao, Matthew Shaw, Soraia Silva, Winston Vetharoy, Diego Leon Rico, Ian Kirby, Francesca Zammarchi, Karin Havenith, Lolke de Haan, Patrick H. van Berkel, Neil Sebire, Olumide K. Ogunbiyi, Claire Booth, H. Bobby Gaspar, Adrian J. Thrasher, Kerry A. Chester, Persis J. Amrolia
-
Need for standardization of cytokine profiling in CAR T cell therapy Mol. Ther. (IF 12.4) Pub Date : 2024-03-26 D. Nathan Biery, David P. Turicek, Caroline Diorio, Brett A. Schroeder, Nirali N. Shah
-
Restoring T and B cell generation in X-linked severe combined immunodeficiency mice through hematopoietic stem cells adenine base editing Mol. Ther. (IF 12.4) Pub Date : 2024-03-26 Lu Zhang, Kai Li, Zhiwei Liu, Lisha An, Haikun Wei, Shanshan Pang, Zongfu Cao, Xingxu Huang, Xiaohua Jin, Xu Ma
-
In vitro and in vivo validation of the antiviral effect of hCypA against SARS-CoV-2 via binding to the RBD of spike protein Mol. Ther. (IF 12.4) Pub Date : 2024-03-25 Woo-Ri Shin, Do-Young Kim, Sang Yong Kim, Gna Ahn, Dae-Young Park, Jiho Min, Ji-Young Ahn, Yang-Hoon Kim
-
Otoferlin gene therapy restores hearing in deaf children Mol. Ther. (IF 12.4) Pub Date : 2024-03-23 John V. Brigande
-
Immune tolerance induction by hepatic gene transfer: First-in-human evidence Mol. Ther. (IF 12.4) Pub Date : 2024-03-21 Radoslaw Kaczmarek, Benjamin J. Samelson-Jones, Roland W. Herzog
-
Chimeric oncolytic adenovirus to break away from neutralizing antibodies Mol. Ther. (IF 12.4) Pub Date : 2024-03-20 Judit Sanchez Gil, Hirotaka Fudaba, Hiroaki Wakimoto
-
APOE2 gene therapy reduces amyloid deposition and improves markers of neuroinflammation and neurodegeneration in a mouse model of Alzheimer disease Mol. Ther. (IF 12.4) Pub Date : 2024-03-19 Rosemary J. Jackson, Megan S. Keiser, Jonah C. Meltzer, Dustin P. Fykstra, Steven E. Dierksmeier, Soroush Hajizadeh, Johannes Kreuzer, Robert Morris, Alexandra Melloni, Tsuneo Nakajima, Luis Tecedor, Paul T. Ranum, Ellie Carrell, YongHong Chen, Maryam A. Nishtar, David M. Holtzman, Wilhelm Haas, Beverly L. Davidson, Bradley T. Hyman
-
High-multiplex single-cell imaging analysis reveals tumor immune contexture associated with clinical outcomes after CAR T cell therapy Mol. Ther. (IF 12.4) Pub Date : 2024-03-19 Jin Jin, Li Lin, Jiao Meng, Lijun Jiang, Man Zhang, Yuekun Fang, Wanying Liu, Xiangke Xin, Xiaolu Long, Dong Kuang, Xilai Ding, Miao Zheng, Yicheng Zhang, Yi Xiao, Liting Chen
-
-
Large-scale falsification of research papers risks public trust in biomedical sciences Mol. Ther. (IF 12.4) Pub Date : 2024-03-19 Rory Bricker-Anthony, Roland W. Herzog
-
FDA approval of the first cellular therapy for a solid (non-hematologic) cancer Mol. Ther. (IF 12.4) Pub Date : 2024-03-19 James N. Kochenderfer
-
In a quest for bivalent mRNA vaccine for respiratory viruses: An effective strategy to overcome antigenic competition Mol. Ther. (IF 12.4) Pub Date : 2024-03-19 Gokulnath Mahalingam, Srujan Marepally
-
Tiny CRISPR molecular switch tool opens a new world Mol. Ther. (IF 12.4) Pub Date : 2024-03-19 Yeounsun Oh, Seung Hwan Lee
-
Rescue of cone and rod photoreceptor function in a CDHR1-model of age-related retinal degeneration Mol. Ther. (IF 12.4) Pub Date : 2024-03-19 Imran H. Yusuf, Thomas Burgoyne, Ahmed Salman, Michelle E. McClements, Robert E. MacLaren, Peter Charbel Issa
-
SOCS3 regulates pathological retinal angiogenesis through modulating SPP1 expression in microglia and macrophages Mol. Ther. (IF 12.4) Pub Date : 2024-03-19 Tianxi Wang, Satoshi Kaneko, Emil Kriukov, David Alvarez, Enton Lam, Yidi Wang, Sara La Manna, Daniela Marasco, Angeles Fernandez-Gonzalez, S. Alex Mitsialis, Stella Kourembanas, Andreas Stahl, Mei Chen, Heping Xu, Petr Baranov, Guoshuai Cai, Ulrich H. von Andrian, Ye Sun
-
The role of the mitochondrial protein VDAC1 in inflammatory bowel disease: a potential therapeutic target Mol. Ther. (IF 12.4) Pub Date : 2024-03-13 Ankit Verma, Srinivas Pittala, Belal Alhozeel, Anna Shteinfer-Kuzmine, Ehud Ohana, Rajeev Gupta, Jay H. Chung, Varda Shoshan-Barmatz
-
Nuclear AGO2 promotes myocardial remodeling by activating ANKRD1 transcription in failing hearts Mol. Ther. (IF 12.4) Pub Date : 2024-03-11 Rong Xie, Shuai Yuan, Guo Hu, Jiabing Zhan, Kunying Jin, Yuyan Tang, Jiahui Fan, Yanru Zhao, Feng Wang, Chen Chen, Dao Wen Wang, Huaping Li
-
In vivo base editing of a pathogenic Eif2b5 variant improves vanishing white matter phenotypes in mice Mol. Ther. (IF 12.4) Pub Date : 2024-03-07 Desirée Böck, Ilma M. Revers, Anastasia S.J. Bomhof, Anne E.J. Hillen, Claire Boeijink, Lucas Kissling, Sabina Egli, Miguel A. Moreno-Mateos, Marjo S. van der Knaap, Niek P. van Til, Gerald Schwank
Vanishing white matter (VWM) is a fatal leukodystrophy caused by recessive mutations in subunits of the eukaryotic translation initiation factor 2B. Currently, there are no effective therapies for VWM. Here, we assessed the potential of adenine base editing to correct human pathogenic VWM variants in mouse models. Using adeno-associated viral vectors, we delivered intein-split adenine base editors
-
The extracellular cyclophilin A-integrin β2 complex as a therapeutic target of viral pneumonia Mol. Ther. (IF 12.4) Pub Date : 2024-03-07 Xiaoyuan Bai, Wenxian Yang, Yuna Zhao, Tongtong Cao, Runshan Lin, Pengtao Jiao, Heqiao Li, Huizi Li, Jie Min, Xiaoxiao Jia, He Zhang, Wenhui Fan, Xiaojuan Jia, Yuhai Bi, Wenjun Liu, Lei Sun
-
ANKRD22 promotes resolution of psoriasiform skin inflammation by antagonizing NIK-mediated IL-23 production Mol. Ther. (IF 12.4) Pub Date : 2024-03-07 Xichun Xia, Leqing Zhu, Miaomiao Xu, Zhiwei Lei, Hai Yu, Guangqiang Li, Xiao Wang, Hongling Jia, Zhinan Yin, Fang Huang, Yunfei Gao
-
On- and off-target effects of paired CRISPR-Cas nickase in primary human cells Mol. Ther. (IF 12.4) Pub Date : 2024-03-07 Julia Klermund, Manuel Rhiel, Thomas Kocher, Kay Ole Chmielewski, Johannes Bischof, Geoffroy Andrieux, Melina el Gaz, Stefan Hainzl, Melanie Boerries, Tatjana I. Cornu, Ulrich Koller, Toni Cathomen
Undesired on- and off-target effects of CRISPR-Cas nucleases remain a challenge in genome editing. While the use of Cas9 nickases has been shown to minimize off-target mutagenesis, their use in therapeutic genome editing has been hampered by a lack of efficacy. To overcome this limitation, we and others have developed double-nickase-based strategies to generate staggered DNA double-strand breaks to
-
Autologous gene therapy for hemoglobinopathies: From bench to patient’s bedside Mol. Ther. (IF 12.4) Pub Date : 2024-03-07 Franco Locatelli, Marina Cavazzana, Haydar Frangoul, Josu de la Fuente, Mattia Algeri, Roland Meisel
-
Targeting lipid nanoparticles to the blood-brain barrier to ameliorate acute ischemic stroke Mol. Ther. (IF 12.4) Pub Date : 2024-03-07 Jia Nong, Patrick M. Glassman, Vladimir V. Shuvaev, Sahily Reyes-Esteves, Helene C. Descamps, Raisa Y. Kiseleva, Tyler E. Papp, Mohamad-Gabriel Alameh, Ying K. Tam, Barbara L. Mui, Serena Omo-Lamai, Marco E. Zamora, Tea Shuvaeva, Evguenia Arguiri, Xijing Gong, Taylor V. Brysgel, Ai Wen Tan, Ashley G. Woolfork, Aalim Weljie, Christoph A. Thaiss, Jacob W. Myerson, Drew Weissman, Scott E. Kasner, Hamideh
-
In memoriam: Kenneth I. Berns, MD, PhD (1938–2024) Mol. Ther. (IF 12.4) Pub Date : 2024-03-06 Barry J. Byrne, Terence R. Flotte, Roland W. Herzog, Arun Srivastava
-
C5a-C5aR1 axis controls mitochondrial fission to promote podocyte injury in lupus nephritis Mol. Ther. (IF 12.4) Pub Date : 2024-03-06 Baokui Ye, Binfeng Chen, Chaohuan Guo, Ningjing Xiong, Yuefang Huang, Mengyuan Li, Yimei Lai, Jin Li, Mianjing Zhou, Shuang Wang, Shuyi Wang, Niansheng Yang, Hui Zhang
Podocytes are essential to maintaining the integrity of the glomerular filtration barrier, but they are frequently affected in lupus nephritis (LN). Here, we show that the significant upregulation of Drp1 phosphorylation in podocytes promotes mitochondrial fission, leading to mitochondrial dysfunction and podocyte injury in LN. Inhibition or knockdown of Drp1 promotes mitochondrial fusion and protects
-
Envelope protein-specific B cell receptors direct lentiviral vector tropism in vivo Mol. Ther. (IF 12.4) Pub Date : 2024-03-06 Kari-Ann Takano, Anita A.L. Wong, Rebecca Brown, Kathy Situ, Bernadette Anne Chua, Angel Elma Abu, Truc T. Pham, Glania Carel Reyes, Sangeetha Ramachandran, Masakazu Kamata, Melody M.H. Li, Ting-Ting Wu, Dinesh S. Rao, Vaithilingaraja Arumugaswami, Kenneth Dorshkind, Steve Cole, Kouki Morizono
-
Engineering small-molecule and protein drugs for targeting bone tumors Mol. Ther. (IF 12.4) Pub Date : 2024-03-06 Yixian Wang, Chenhang Wang, Meng Xia, Zeru Tian, Joseph Zhou, Julian Meyer Berger, Xiang H.-F. Zhang, Han Xiao
-
Nr4a1 enhances Wnt4 transcription to promote mesenchymal stem cell osteogenesis and alleviates inflammation-inhibited bone regeneration Mol. Ther. (IF 12.4) Pub Date : 2024-03-01 Yangshuai Gao, Yuming Zou, Dimitri Sokolowskei, Xin Xing, Robert J. Tower, Zejia Lai, Jiawei Shi, Liheng Zhu, Quan Zheng, Aaron W. James, Jiajia Xu, Zhongmin Zhang
Intense inflammatory response impairs bone marrow mesenchymal stem cell (BMSC)-mediated bone regeneration, with transforming growth factor (TGF)-β1 being the most highly expressed cytokine. However, how to find effective and safe means to improve bone formation impaired by excessive TGF-β1 remains unclear. In this study, we found that the expression of orphan nuclear receptor Nr4a1, an endogenous repressor
-
Gene therapy approaches for obesity-induced adipose neuropathy: Device-targeted AAV-mediated neurotrophic factor delivery to adipocytes in subcutaneous adipose Mol. Ther. (IF 12.4) Pub Date : 2024-03-01 Magdalena Blaszkiewicz, Tianyi Tao, Kofi Mensah-Arhin, Jake W. Willows, Rhiannon Bates, Wei Huang, Lei Cao, Rosemary L. Smith, Kristy L. Townsend
Maintaining functional adipose innervation is critical for metabolic health. We found that subcutaneous white adipose tissue (scWAT) undergoes peripheral neuropathy (PN) with obesity, diabetes, and aging (reduced small-fiber innervation and nerve/synaptic/growth-cone/vesicle markers, altered nerve activity). Unlike with nerve injuries, peripheral nerves do not regenerate with PN, and therefore new
-
ASOs are an effective treatment for disease-associated oligodendrocyte signatures in premanifest and symptomatic SCA3 mice Mol. Ther. (IF 12.4) Pub Date : 2024-02-29 Kristen H. Schuster, Annie J. Zalon, Danielle M. DiFranco, Alexandra F. Putka, Nicholas R. Stec, Sabrina I. Jarrah, Arsal Naeem, Zaid Haque, Hanrui Zhang, Yuanfang Guan, Hayley S. McLoughlin
Spinocerebellar ataxia type 3 (SCA3) is the most common dominantly inherited ataxia. Currently, no preventive or disease-modifying treatments exist for this progressive neurodegenerative disorder, although efforts using gene silencing approaches are under clinical trial investigation. The disease is caused by a CAG repeat expansion in the mutant gene, , producing an enlarged polyglutamine tract in
-
Lentiviral expression of wild-type LAMA3A restores cell adhesion in airway basal cells from children with epidermolysis bullosa Mol. Ther. (IF 12.4) Pub Date : 2024-02-29 Chun Hang Lau, Maral J. Rouhani, Elizabeth F. Maughan, Jessica C. Orr, Krishna K. Kolluri, David R. Pearce, Elizabeth K. Haughey, Liam Sutton, Sam Flatau, Pablo Lopez Balboa, Maria Laura Bageta, Christopher O’Callaghan, Claire M. Smith, Sam M. Janes, Richard Hewitt, Gabriela Petrof, Anna E. Martinez, John A. McGrath, Colin R. Butler, Robert E. Hynds
-
Activation of the YAP/KLF5 transcriptional cascade in renal tubular cells aggravates kidney injury Mol. Ther. (IF 12.4) Pub Date : 2024-02-27 Yang Liu, Yu Wang, Chunhua Xu, Yu Zhang, Yang Wang, Jinzhong Qin, Hui-Yao Lan, Li Wang, Yu Huang, Kingston Kinglun Mak, Zhihua Zheng, Yin Xia
The Hippo/YAP pathway plays a critical role in tissue homeostasis. Our previous work demonstrated that renal tubular YAP activation induced by double knockout (dKO) of the upstream Hippo kinases Mst1 and Mst2 promotes tubular injury and renal inflammation under basal conditions. However, the importance of tubular YAP activation remains to be established in injured kidneys in which many other injurious
-
CD20 CAR T cells safely and reversibly ablate B cell follicles in a non-human primate model of HIV persistence Mol. Ther. (IF 12.4) Pub Date : 2024-02-27 John K. Bui, Carly E. Starke, Nikhita H. Poole, Blake J. Rust, Keith R. Jerome, Hans-Peter Kiem, Christopher W. Peterson
Chimeric antigen receptor (CAR) T cell therapies have demonstrated immense clinical success for B cell and plasma cell malignancies. We tested their impact on the viral reservoir in a macaque model of HIV persistence, comparing the functions of CD20 CAR T cells between animals infected with simian/human immunodeficiency virus (SHIV) and uninfected controls. We focused on the potential of this approach
-
Inhibition of Gpx4-mediated ferroptosis alleviates cisplatin-induced hearing loss in C57BL/6 mice Mol. Ther. (IF 12.4) Pub Date : 2024-02-27 Ziyi Liu, Hanbing Zhang, Guodong Hong, Xiuli Bi, Jun Hu, Tiancheng Zhang, Yachun An, Na Guo, Fengyue Dong, Yu Xiao, Wen Li, Xiaoxu Zhao, Bo Chu, Siwei Guo, Xiaohan Zhang, Renjie Chai, Xiaolong Fu
Cisplatin-induced hearing loss is a common side effect of cancer chemotherapy in clinics; however, the mechanism of cisplatin-induced ototoxicity is still not completely clarified. Cisplatin-induced ototoxicity is mainly associated with the production of reactive oxygen species, activation of apoptosis, and accumulation of intracellular lipid peroxidation, which also is involved in ferroptosis induction
-
SMART-lipid nanoparticles enabled mRNA vaccine elicits cross-reactive humoral responses against the omicron sub-variants Mol. Ther. (IF 12.4) Pub Date : 2024-02-27 Gokulnath Mahalingam, Hari Krishnareddy Rachamalla, Porkizhi Arjunan, Karthik V. Karuppusamy, Yogapriya Periyasami, Aruna Mohan, Kanimozhi Subramaniyam, Salma M, Vigneshwar Rajendran, Mahesh Moorthy, George M. Varghese, Kumarasamypet M. Mohankumar, Saravanabhavan Thangavel, Alok Srivastava, Srujan Marepally
The continual emergence of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) variants has necessitated the development of broad cross-reactive vaccines. Recent findings suggest that enhanced antigen presentation could lead to cross-reactive humoral responses against the emerging variants. Toward enhancing the antigen presentation to dendritic cells (DCs), we developed a novel shikimoylated
-
Hnrnpk protects against osteoarthritis through targeting WWC1 mRNA and inhibiting Hippo signaling pathway Mol. Ther. (IF 12.4) Pub Date : 2024-02-27 Shun Zhang, Baolin Zhang, Zhiheng Liao, Yuyu Chen, Weimin Guo, Jinna Wu, Hengyu Liu, Ricong Weng, Deying Su, Gengjia Chen, Zhenzhen Zhang, Chuan Li, Jiahui Long, Ya Xiao, Yuan Ma, Taifeng Zhou, Caixia Xu, Peiqiang Su
Osteoarthritis (OA) is an age-related or post-traumatic degenerative whole joint disease characterized by the rupture of articular cartilage homeostasis, the regulatory mechanisms of which remain elusive. This study identifies the essential role of heterogeneous nuclear ribonucleoprotein K (hnRNPK) in maintaining articular cartilage homeostasis. Hnrnpk expression is markedly downregulated in human
-
Stem cell-derived CAR T cells show greater persistence, trafficking, and viral control compared to ex vivo transduced CAR T cells Mol. Ther. (IF 12.4) Pub Date : 2024-02-27 Mayra A. Carrillo, Anjie Zhen, Wenli Mu, Valerie Rezek, Heather Martin, Christopher W. Peterson, Hans-Peter Kiem, Scott G. Kitchen
-
Osr1-mediated mesothelial transition of liver mesenchymal cells exacerbates fibrotic liver damage Mol. Ther. (IF 12.4) Pub Date : 2024-02-27 Xinxin Nian, Pengyan Lin, Yunfei Bai, Donglin Yu, Xinyan Yang, Bin Zhou, Jie Gao, Yang Zhao
In chronic liver diseases, hepatic stellate cells (HSCs) are induced to form the myofibroblasts responsible for scar formation, leading to liver fibrosis and cirrhosis. Here, single-cell RNA sequencing with lineage tracing in nonalcoholic steatohepatitis (NASH) model mice reveals a subpopulation of HSCs transitioning back to a state resembling their developmental precursors, mesothelial cells (MCs)
-
Therapeutic potential of RNA-enriched extracellular vesicles: The next generation in RNA delivery via biogenic nanoparticles Mol. Ther. (IF 12.4) Pub Date : 2024-02-27 Muskan Muskan, Pevindu Abeysinghe, Riccardo Cecchin, Heather Branscome, Kevin V. Morris, Fatah Kashanchi
Exosomes are extracellular vesicles (EVs) (∼50–150 nm) that have emerged as promising vehicles for therapeutic applications and drug delivery. These membrane-bound particles, released by all actively dividing cells, have the ability to transfer effector molecules, including proteins, RNA, and even DNA, from donor cells to recipient cells, thereby modulating cellular responses. RNA-based therapeutics
-
Analysis of pre-treatment tumors reveals gatekeepers of response to CAR T cells Mol. Ther. (IF 12.4) Pub Date : 2024-02-22 Nathan Singh
-
Inhibitory CARs fail to protect from immediate T cell cytotoxicity Mol. Ther. (IF 12.4) Pub Date : 2024-02-22 Maximilian A. Funk, Gerwin Heller, Petra Waidhofer-Söllner, Judith Leitner, Peter Steinberger
-