Molecular therapy : the journal of the American Society of Gene Therapy最新文献_第3页

METTL1/WDR4-mediated m⁷G tRNA modifications and m⁷G codon usage promote mRNA translation and lung cancer progression. METTL1/ wdr4介导的m7G tRNA修饰和m7G密码子使用促进mRNA翻译和肺癌进展。

IF 12.4

Molecular therapy : the journal of the American Society of Gene Therapy Pub Date : 2021-12-01 Epub Date: 2021-08-08 DOI: 10.1016/j.ymthe.2021.08.005

Jieyi Ma, Hui Han, Ying Huang, Chunlong Yang, Siyi Zheng, Tiancai Cai, Jiong Bi, Xiaohui Huang, Ruiming Liu, Libin Huang, Yifeng Luo, Wen Li, Shuibin Lin

{"title":"METTL1/WDR4-mediated m7G tRNA modifications and m7G codon usage promote mRNA translation and lung cancer progression.","authors":"Jieyi Ma, Hui Han, Ying Huang, Chunlong Yang, Siyi Zheng, Tiancai Cai, Jiong Bi, Xiaohui Huang, Ruiming Liu, Libin Huang, Yifeng Luo, Wen Li, Shuibin Lin","doi":"10.1016/j.ymthe.2021.08.005","DOIUrl":"https://doi.org/10.1016/j.ymthe.2021.08.005","url":null,"abstract":"Mis-regulated epigenetic modifications in RNAs are associated with human cancers. The transfer RNAs (tRNAs) are the most heavily modified RNA species in cells; however, little is known about the functions of tRNA modifications in cancers. In this study, we uncovered that the expression levels of tRNA N7-methylguanosine (m7G) methyltransferase complex components methyltransferase-like 1 (METTL1) and WD repeat domain 4 (WDR4) are significantly elevated in human lung cancer samples and negatively associated with patient prognosis. Impaired m7G tRNA modification upon METTL1/WDR4 depletion resulted in decreased cell proliferation, colony formation, cell invasion, and impaired tumorigenic capacities of lung cancer cells in vitro and in vivo. Moreover, gain-of-function and mutagenesis experiments revealed that METTL1 promoted lung cancer growth and invasion through regulation of m7G tRNA modifications. Profiling of tRNA methylation and mRNA translation revealed that highly translated mRNAs have higher frequencies of m7G tRNA-decoded codons, and knockdown of METTL1 resulted in decreased translation of mRNAs with higher frequencies of m7G tRNA codons, suggesting that tRNA modifications and codon usage play an essential function in mRNA translation regulation. Our data uncovered novel insights on mRNA translation regulation through tRNA modifications and the corresponding mRNA codon compositions in lung cancer, providing a new molecular basis underlying lung cancer progression.","PeriodicalId":519519,"journal":{"name":"Molecular therapy : the journal of the American Society of Gene Therapy","volume":" ","pages":"3422-3435"},"PeriodicalIF":12.4,"publicationDate":"2021-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.ymthe.2021.08.005","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39294486","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 104

Eliminating Panglossian thinking in development of AAV therapeutics. 消除对AAV疗法发展的乐观看法。

IF 12.4

Molecular therapy : the journal of the American Society of Gene Therapy Pub Date : 2021-12-01 Epub Date: 2021-11-10 DOI: 10.1016/j.ymthe.2021.10.025

Radoslaw Kaczmarek, Glenn F Pierce, Declan Noone, Brian O'Mahony, David Page, Mark W Skinner

引用次数: 8

Cell type-selective targeted delivery of a recombinant lysosomal enzyme for enzyme therapies. 细胞类型选择性靶向递送重组溶酶体酶用于酶治疗。

IF 12.4

Molecular therapy : the journal of the American Society of Gene Therapy Pub Date : 2021-12-01 Epub Date: 2021-08-14 DOI: 10.1016/j.ymthe.2021.08.020

Andrew D Baik, Philip Calafati, Xiaoli Zhang, Nina A Aaron, Antonia Mehra, Sven Moller-Tank, Lawrence Miloscio, Maria Praggastis, Nicholas Giovannone, Cheryl Pan, Yajun Tang, Susannah Bridges, Alejo Mujica, Peter Barbounis, Jean Yanolatos, Nicholas Gale, Ning Li, Christos A Kyratsous, Christopher J Schoenherr, Andrew J Murphy, Aris N Economides, Katherine D Cygnar

{"title":"Cell type-selective targeted delivery of a recombinant lysosomal enzyme for enzyme therapies.","authors":"Andrew D Baik, Philip Calafati, Xiaoli Zhang, Nina A Aaron, Antonia Mehra, Sven Moller-Tank, Lawrence Miloscio, Maria Praggastis, Nicholas Giovannone, Cheryl Pan, Yajun Tang, Susannah Bridges, Alejo Mujica, Peter Barbounis, Jean Yanolatos, Nicholas Gale, Ning Li, Christos A Kyratsous, Christopher J Schoenherr, Andrew J Murphy, Aris N Economides, Katherine D Cygnar","doi":"10.1016/j.ymthe.2021.08.020","DOIUrl":"https://doi.org/10.1016/j.ymthe.2021.08.020","url":null,"abstract":"Lysosomal diseases are a class of genetic disorders predominantly caused by loss of lysosomal hydrolases, leading to lysosomal and cellular dysfunction. Enzyme replacement therapy (ERT), where recombinant enzyme is given intravenously, internalized by cells, and trafficked to the lysosome, has been applied to treat several lysosomal diseases. However, current ERT regimens do not correct disease phenotypes in all affected organs because the biodistribution of enzyme uptake does not match that of the affected cells that require the enzyme. We present here targeted ERT, an approach that utilizes antibody-enzyme fusion proteins to target the enzyme to specific cell types. The antibody moiety recognizes transmembrane proteins involved in lysosomal trafficking and that are also preferentially expressed in those cells most affected in disease. Using Pompe disease (PD) as an example, we show that targeted ERT is superior to ERT in treating the skeletal muscle phenotypes of PD mice both as a protein replacement therapeutic and as a gene therapy.","PeriodicalId":519519,"journal":{"name":"Molecular therapy : the journal of the American Society of Gene Therapy","volume":" ","pages":"3512-3524"},"PeriodicalIF":12.4,"publicationDate":"2021-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.ymthe.2021.08.020","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39317573","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 8

Topics in AAV integration come front and center at ASGCT AAV Integration Roundtable. AAV集成的主题是ASGCT AAV集成圆桌会议的前沿和中心。

IF 12.4

Molecular therapy : the journal of the American Society of Gene Therapy Pub Date : 2021-12-01 Epub Date: 2021-11-10 DOI: 10.1016/j.ymthe.2021.10.024

Denise E Sabatino, Douglas M McCarty

引用次数: 2

Exosomal miR-1246 from glioma patient body fluids drives the differentiation and activation of myeloid-derived suppressor cells. 来自胶质瘤患者体液的外泌体miR-1246驱动髓源性抑制细胞的分化和激活。

IF 12.4

Molecular therapy : the journal of the American Society of Gene Therapy Pub Date : 2021-12-01 Epub Date: 2021-07-02 DOI: 10.1016/j.ymthe.2021.06.023

Wei Qiu, Xiaofan Guo, Boyan Li, Jian Wang, Yanhua Qi, Zihang Chen, Rongrong Zhao, Lin Deng, Mingyu Qian, Shaobo Wang, Zongpu Zhang, Qindong Guo, Shouji Zhang, Ziwen Pan, Shulin Zhao, Hao Xue, Gang Li

{"title":"Exosomal miR-1246 from glioma patient body fluids drives the differentiation and activation of myeloid-derived suppressor cells.","authors":"Wei Qiu, Xiaofan Guo, Boyan Li, Jian Wang, Yanhua Qi, Zihang Chen, Rongrong Zhao, Lin Deng, Mingyu Qian, Shaobo Wang, Zongpu Zhang, Qindong Guo, Shouji Zhang, Ziwen Pan, Shulin Zhao, Hao Xue, Gang Li","doi":"10.1016/j.ymthe.2021.06.023","DOIUrl":"https://doi.org/10.1016/j.ymthe.2021.06.023","url":null,"abstract":"Glioma is a heterogeneous cellular environment in which immune cells play critical roles in tumor progression. Myeloid-derived suppressor cells (MDSCs) contribute to the formation of the immunosuppressive microenvironment of glioma; however, how glioma cells interact with MDSCs and how this interaction affects the function of other immune cells are unclear. Glioma cells can systemically communicate with immune cells via the secretion of exosomes, which contain microRNAs (miRNAs). Leveraging miRNA sequencing of exosomes, we identified enrichment of miR-1246 in glioma-derived exosomes and exosomes isolated from the cerebrospinal fluid (CSF) of glioma patients. We demonstrated that miR-1246 drives the differentiation and activation of MDSCs in a dual specificity phosphatase 3 (DUSP3)/extracellular signal‑regulated kinase (ERK)-dependent manner. In addition, postoperative CSF exosomal miR-1246 expression was found to be associated with the glioma recurrence rate. Hypoxia, a well-recognized feature of the glioblastoma microenvironment, increased miR-1246 levels in glioma-derived exosomes by enhancing miR-1246 transcription and selective packaging via upregulation of POU class 5 homeobox 1 (POU5F1) and heterogeneous nuclear ribonucleoprotein A1 (hnRNPA1). Importantly, we identified a mechanism of 2-methoxyestradiol, a microtubule inhibitor currently undergoing clinical trials for glioblastoma. 2-Methoxyestradiol suppresses MDSC activation by inhibiting hypoxia-driven exosomal miR-1246 expression in glioma cells and PD-L1 expression in MDSCs.","PeriodicalId":519519,"journal":{"name":"Molecular therapy : the journal of the American Society of Gene Therapy","volume":" ","pages":"3449-3464"},"PeriodicalIF":12.4,"publicationDate":"2021-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.ymthe.2021.06.023","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39157373","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 38

m⁶A modification regulates lung fibroblast-to-myofibroblast transition through modulating KCNH6 mRNA translation. m6A修饰通过调节KCNH6 mRNA的翻译调控肺成纤维细胞向肌成纤维细胞的转化。

IF 12.4

Molecular therapy : the journal of the American Society of Gene Therapy Pub Date : 2021-12-01 Epub Date: 2021-06-08 DOI: 10.1016/j.ymthe.2021.06.008

Jia-Xiang Zhang, Pei-Jie Huang, Da-Peng Wang, Wen-Yu Yang, Jian Lu, Yong Zhu, Xiao-Xiao Meng, Xin Wu, Qiu-Hai Lin, Hui Lv, Hui Xie, Rui-Lan Wang

{"title":"m6A modification regulates lung fibroblast-to-myofibroblast transition through modulating KCNH6 mRNA translation.","authors":"Jia-Xiang Zhang, Pei-Jie Huang, Da-Peng Wang, Wen-Yu Yang, Jian Lu, Yong Zhu, Xiao-Xiao Meng, Xin Wu, Qiu-Hai Lin, Hui Lv, Hui Xie, Rui-Lan Wang","doi":"10.1016/j.ymthe.2021.06.008","DOIUrl":"https://doi.org/10.1016/j.ymthe.2021.06.008","url":null,"abstract":"Idiopathic pulmonary fibrosis (IPF) is a chronic, fatal lung disease characterized by progressive and non-reversible abnormal matrix deposition in lung parenchyma. Myofibroblasts originating mainly from resident fibroblasts via fibroblast-to-myofibroblast transition (FMT) are the dominant collagen-producing cells in pulmonary fibrosis. N6-methyladenosine (m6A) modification has been implicated in various biological processes. However, the role of m6A modification in pulmonary fibrosis remains elusive. In this study, we reveal that m6A modification is upregulated in a bleomycin (BLM)-induced pulmonary fibrosis mouse model, FMT-derived myofibroblasts, and IPF patient lung samples. Lowering m6A levels through silencing methyltransferase-like 3 (METTL3) inhibits the FMT process in vitro and in vivo. Mechanistically, KCNH6 is involved in the m6A-regulated FMT process. m6A modification regulates the expression of KCNH6 by modulating its translation in a YTH-domain family 1 (YTHDF1)-dependent manner. Together, our study highlights the critical role of m6A modification in pulmonary fibrosis. Manipulation of m6A modification through targeting METTL3 may become a promising strategy for the treatment of pulmonary fibrosis.","PeriodicalId":519519,"journal":{"name":"Molecular therapy : the journal of the American Society of Gene Therapy","volume":" ","pages":"3436-3448"},"PeriodicalIF":12.4,"publicationDate":"2021-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.ymthe.2021.06.008","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39081114","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 21

Next-generation strategies for gene-targeted therapies of central nervous system disorders: A workshop summary. 中枢神经系统疾病基因靶向治疗的新一代策略:研讨会总结。

IF 12.4

Molecular therapy : the journal of the American Society of Gene Therapy Pub Date : 2021-12-01 Epub Date: 2021-09-20 DOI: 10.1016/j.ymthe.2021.09.010

Jill A Morris, Chris H Boshoff, Nina F Schor, Ling M Wong, Guangping Gao, Beverly L Davidson

{"title":"Next-generation strategies for gene-targeted therapies of central nervous system disorders: A workshop summary.","authors":"Jill A Morris, Chris H Boshoff, Nina F Schor, Ling M Wong, Guangping Gao, Beverly L Davidson","doi":"10.1016/j.ymthe.2021.09.010","DOIUrl":"https://doi.org/10.1016/j.ymthe.2021.09.010","url":null,"abstract":"The National Institute of Neurological Disorders and Stroke (NINDS) held a workshop titled \"Next generation strategies for gene-targeted therapies of central nervous system (CNS) disorders\" in September 2019 in Bethesda, MD, USA. The meeting brought together a multi-disciplinary group of experts in the field of CNS-directed gene-targeted therapy delivery from academia, industry, advocacy, and the government. The group was charged with identifying the key challenges and gaps in this evolving field, as well as suggesting potential solutions. The workshop was divided into four sessions: (1) control of level and location, (2) improving delivery and distribution, (3) enhancing models and manufacturing, and (4) impacting patients. Prior to the workshop, NINDS established working groups of key opinion leaders (KOLs) for each session. In pre-meeting teleconferences, KOLs were tasked with identifying the research gaps and key obstacles that delay and/or prevent gene-targeted therapies to move into the clinic. This approach allowed for the workshop to begin with problem-solving discussions and strategy development, as the key issues had been established. The overall purpose of the workshop was to consider knowledge gaps and potential strategies to inform the community around CNS gene-targeted therapies, including but not limited to researchers and funders.","PeriodicalId":519519,"journal":{"name":"Molecular therapy : the journal of the American Society of Gene Therapy","volume":" ","pages":"3332-3344"},"PeriodicalIF":12.4,"publicationDate":"2021-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/68/3f/main.PMC8636163.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39438567","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 11

Prior nasal delivery of antagomiR-122 prevents radiation-induced brain injury. 先前鼻给药安塔哥米-122可预防辐射引起的脑损伤。

IF 12.4

Molecular therapy : the journal of the American Society of Gene Therapy Pub Date : 2021-12-01 Epub Date: 2021-06-24 DOI: 10.1016/j.ymthe.2021.06.019

Haihong Zhou, Furong Sun, Mingqian Ou, Yu Zhang, Meijun Lin, Liqin Song, Yangsheng Yu, Haojie Liao, Weihao Fan, Huaijie Xing, Minhua Li, Kui Zhao, Xiaolian Wu, Yuanhong Sun, Chunmei Liang, Yujie Cai, Lili Cui

{"title":"Prior nasal delivery of antagomiR-122 prevents radiation-induced brain injury.","authors":"Haihong Zhou, Furong Sun, Mingqian Ou, Yu Zhang, Meijun Lin, Liqin Song, Yangsheng Yu, Haojie Liao, Weihao Fan, Huaijie Xing, Minhua Li, Kui Zhao, Xiaolian Wu, Yuanhong Sun, Chunmei Liang, Yujie Cai, Lili Cui","doi":"10.1016/j.ymthe.2021.06.019","DOIUrl":"https://doi.org/10.1016/j.ymthe.2021.06.019","url":null,"abstract":"Radiation-induced brain injury is a major adverse event in head and neck tumor treatment, influencing the quality of life for the more than 50% of patients who undergo radiation therapy and experience long-term survival. However, no effective treatments are available for these patients, and preventative drugs and effective drug-delivery methods must be developed. Based on our results, miR-122-5p was upregulated in the mouse radiation-induced brain injury (RBI) model and patients with nasopharyngeal carcinoma (NPC) who received radiation therapy. Intranasal administration of a single antagomiR-122-5p dose before irradiation effectively alleviated radiation-induced cognitive impairment, neuronal injury, and neuroinflammation in the mouse RBI model. Results further indicated that miR-122-5p inhibition in microglia reduced the levels of proinflammatory cytokines and enhanced the phagocytic function to protect against radiation-induced neuronal injury in cell models. Further, we profiled transcriptome data and verified that Tensin 1 (TNS1) may be the target of miR-122-5p in RBI. In summary, our results reveal a distinct role for miR-122-5p in regulating neuroinflammation in RBI, indicating that a non-invasive strategy for intranasal miR-122-5p administration may be an attractive therapeutic target in RBI, providing new insights for clinical trials. Further systematic safety assessment, optimization of drug administration, and clarity of mechanism will accelerate the process into clinical practice.","PeriodicalId":519519,"journal":{"name":"Molecular therapy : the journal of the American Society of Gene Therapy","volume":" ","pages":"3465-3483"},"PeriodicalIF":12.4,"publicationDate":"2021-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8636282/pdf/main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39109441","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 6

Cardiolipin remodeling by ALCAT1 links hypoxia to coronary artery disease by promoting mitochondrial dysfunction. ALCAT1介导的心磷脂重塑通过促进线粒体功能障碍将缺氧与冠状动脉疾病联系起来。

IF 12.4

Molecular therapy : the journal of the American Society of Gene Therapy Pub Date : 2021-12-01 Epub Date: 2021-06-08 DOI: 10.1016/j.ymthe.2021.06.007

Dandan Jia, Jun Zhang, Jia Nie, John-Paul Andersen, Samantha Rendon, Yue Zheng, Xueling Liu, Zhenjun Tian, Yuguang Shi

{"title":"Cardiolipin remodeling by ALCAT1 links hypoxia to coronary artery disease by promoting mitochondrial dysfunction.","authors":"Dandan Jia, Jun Zhang, Jia Nie, John-Paul Andersen, Samantha Rendon, Yue Zheng, Xueling Liu, Zhenjun Tian, Yuguang Shi","doi":"10.1016/j.ymthe.2021.06.007","DOIUrl":"https://doi.org/10.1016/j.ymthe.2021.06.007","url":null,"abstract":"Cardiolipin is a mitochondrial signature phospholipid that plays a pivotal role in maintaining cardiac health. A loss of tetralinoleoyl cardiolipin (TLCL), the predominant cardiolipin species in the healthy mammalian heart, is implicated in the pathogenesis of coronary heart disease (CHD) through poorly defined mechanisms. Here, we identified acyl-coenzyme A:lysocardiolipin acyltransferase-1 (ALCAT1) as the missing link between hypoxia and CHD in an animal model of myocardial infarction (MI). ALCAT1 is an acyltransferase that promotes mitochondrial dysfunction in aging-related diseases by catalyzing pathological remodeling of cardiolipin. In support of a causative role of ALCAT1 in CHD, we showed that ALCAT1 expression was potently upregulated by MI, linking myocardial hypoxia to oxidative stress, TLCL depletion, and mitochondrial dysfunction. Accordingly, ablation of the ALCAT1 gene or pharmacological inhibition of the ALCAT1 enzyme by Dafaglitapin (Dafa), a potent and highly specific ALCAT1 inhibitor, not only restored TLCL levels but also mitochondrial respiration by attenuating signal transduction pathways mediated by hypoxia-inducible factor 1α (HIF-1α). Consequently, ablation or pharmacological inhibition of ALCAT1 by Dafa effectively mitigated CHD and its underlying pathogenesis, including dilated cardiomyopathy, left ventricle dysfunction, myocardial inflammation, fibrosis, and apoptosis. Together, the findings have provided the first proof-of-concept studies for targeting ALCAT1 as an effective treatment for CHD.","PeriodicalId":519519,"journal":{"name":"Molecular therapy : the journal of the American Society of Gene Therapy","volume":" ","pages":"3498-3511"},"PeriodicalIF":12.4,"publicationDate":"2021-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.ymthe.2021.06.007","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39081115","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 15

Gene therapy reforms photoreceptor structure and restores vision in NPHP5-associated Leber congenital amaurosis. 基因治疗改变nphp5相关Leber先天性黑朦患者的感光体结构并恢复视力。

IF 12.4

Molecular therapy : the journal of the American Society of Gene Therapy Pub Date : 2021-12-01 Epub Date: 2021-10-29 DOI: 10.1016/j.ymthe.2021.10.021

Gustavo D Aguirre, Artur V Cideciyan, Valérie L Dufour, Ana Ripolles-García, Raghavi Sudharsan, Malgorzata Swider, Roman Nikonov, Simone Iwabe, Sanford L Boye, William W Hauswirth, Samuel G Jacobson, William A Beltran

引用次数: 1