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Molecular Therapy Methods and Clinical Development

IF4.7
论文数229
被引数6169
期刊论文 229
发表时间
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Genome Editing in Patient iPSCs Corrects the Most Prevalent USH2A Mutations and Reveals Intriguing Mutant mRNA Expression Profiles患者iPSCs中的基因组编辑纠正了最常见的USH2A突变，并揭示了有趣的突变mRNA表达谱。
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-11-24
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deleteCarla Sanjurjo-Soriano; Nejla Erkilic; David Baux; Daria Mamaeva; Christian P. Hamel; Isabelle Meunier; Anne-Françoise Roux; Vasiliki Kalatzis
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HBVZ10, An AAV8 Vector-Based New HBV Therapy Candidate for cccDNA EliminationHBVZ10，一种基于AAV8载体的新型HBV治疗候选药物，用于cccDNA消除
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-11-19
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deleteBai-Hua Zhang; Yuanping Zhou; Stephen Horrigan; Laura Luckenbaugh; Jianming Hu; Fabien Zoulim; Yong-Yuan Zhang
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Molecular Therapy Advances: Building the bridge between discovery and cure《分子治疗进展：构建发现与治愈之间的桥梁》
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-11-17
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deleteMohamed Abou-el-Enein
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Unveiling Genomic Rearrangements in Engineered iPSC Lines by Optical Genome Mapping通过光学基因组图谱揭示工程化iPSC系中的基因组重排
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-11-15
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deleteDarren Finlay; Pooja Hor; Benjamin H. Goldenson; Xiao-Hua Li; Rabi Murad; Dan S. Kaufman; Kristiina Vuori
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Inhibition of immune response reduces pathology in dorsal root ganglia and peripheral nerves in cynomolgus macaques following adeno-associated virus gene therapy免疫反应的抑制可减轻食蟹猴在腺相关病毒基因治疗后的脊神经节和周围神经的病理变化
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-11-14
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deleteBranka Grubor; Kate L. Henry; Su Jing Chan; Mark Sheehan; Anumeha Shah; Alex Pellerin; Judy Bai; Prasad Nadella; Santhosh Bommegowda; Patrick Cullen; Eric Tien; Vivian Chen; Nicholas P. van der Munnik; Stephanie White-Hunt; Edward D. Plowey; Stefan Hamann; Amanda J. Guise; Shanqin Xu; Melissa Kirkland; Jessica Doherty
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Modified AAV5 Capsid for Improved Brain Biodistribution Following Direct Injection in Preclinical Models经修饰的AAV5衣壳在预临床模型直接注射后改善脑内生物分布
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-11-14
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deleteSebastian N. Kieper; Elisabeth A. Spronck; Meriem Bourajjaj; Roberto D.V.S Morais; M. Leontien van der Bent; Seyda Acar Broekmans; Charlot van Rooijen; Rhodé van Westen; Giorgia Squeri; Giso Brasser; Morgane Wartel; Stijn J.H. Waaijer; C.Warner Hoornenborg; Tycho M. Hoogland; Richard Porter; Angga kusuma
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Leveraging the Active Conformation of LFA-1 as a Potential Target for Hematological Malignancies利用LFA-1的活性构象作为血液系统恶性肿瘤的潜在靶点
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-11-12
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deleteAbhishek Puthenveetil; Guy Biber; Batel Sabag; Meirav Kedmi; Avigdor Abraham; Noah Joseph; Mira Barda-Saad
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AAV-PHP.eB achieves superior neuronal transduction compared to AAV9 in pigtail macaques following intracerebroventricular administration.AAV-PHP.eB在恒河猴经脑室内注射后，其神经元转导效果优于AAV9。
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-11-12
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deleteMichal G. Fortuna; Lyle H. Nyberg; Naz Taskin; Avery Hunker; Natalie Weed; Kathryn Gudsnuk; Melissa Berg; Boaz Levi; Ed Lein; Jonathan T. Ting
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CRISPR/Methotrexate-integrated Strategy for TCR-T Cell Engineering with Reduced Chromosome 14 LossCRISPR/甲氨蝶呤整合策略：用于减少染色体14丢失的TCR-T细胞工程
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-11-12
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deleteJian Xu; Lianghua Shen; Ziyu Chen; Changmeng Zhang; Xiaodan Ding; Qiaomei He; Xiao Zhou; Peiyao Jiang; Jiayu Liu; Lu Li; Jinan Fang; Fanlin Li; Liping Wan; Xueying Ding; Yuqin Yang; Xiaorui Wang; Pengran Wang; Xianmin Song; Yan Zhang
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Elucidation of the Binding Interaction Interface Between Adeno-Associated Virus Serotype 11 Capsid Protein and Host Nuclear Import Proteins腺相关病毒血清型11衣壳蛋白与宿主核输入蛋白之间结合相互作用界面的阐明
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-11-10
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deleteMikayla Hoad; Sepehr Nematollahzadeh; Justin A. Roby; Gualtiero Alvisi; Jade K. Forwood
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The Amazing AAV Capsids: Into the Structure-Verse惊人的AAV衣壳：进入结构宇宙
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-11-10
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deleteJane His-Bell; Mario Mietzsch; Robert McKenna
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Nonclinical Toxicity Study Duration in AAV Gene Therapy Development: Evidence from Industry Survey Supports Adequacy of Short-Term AssessmentsAAV基因治疗开发中的非临床毒理学研究持续时间：行业调查证据支持短期评估的充分性
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-11-10
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deleteBasel T. Assaf; David Edwards; Shawn Jay Berens; Brian Lauritzen; Christelle Pierrot; Francis Fonyuy Tukov; Cristina Bertinetti-Lapatki; Manuela Braun; Kai Schaefer; Jessica L. Lynch
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Retinal organoids mirror CRISPR/Cas9 gene editing efficiency observed in vivo视网膜类器官反映了在体观察到的CRISPR/Cas9基因编辑效率
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-11-10
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deleteJuliette Pulman; Hugo Malki; Paul Oudin; Ecem Aydin; Sophie Tran; Laura Visticot; Camille Robert; Anne De Cian; Marie As; Olivier Goureau; Jean-Paul Concordet; Deniz Dalkara
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Targeted Inhibition of ELANE Expression Using Adenine Base Editing to Treat Severe Congenital Neutropenia利用腺嘌呤碱基编辑靶向抑制ELANE表达以治疗重症先天性中性粒细胞减少症
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-11-05
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deleteBetül Findik; Benjamin Dannenmann; Franka Bernhard; Masako Monika Kaufmann; Sandra Ammann; Sergey Kandabarau; Maksim Klimiankou; Fabian Mauch; Patrick Münzer; Oliver Borst; Isabel Klefenz; Doris Steinemann; Claudia Lengerke; Cornelia Zeidler; Toni Cathomen; Karl Welte; Masoud Nasri; Julia Skokowa
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Safety and Efficacy of AAV8-aflibercept in Treating Choroidal Neovascularization via Single-Cell RNA SequencingAAV8-aflibercept治疗脉络膜新生血管的单细胞RNA测序安全性及有效性研究
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-11-05
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deleteJiemei Shi; Kaicheng Wu; Lihui Chen; Yinling Li; Chunhui Jiang
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End-to-end characterization of AAV manufacturing process using charge detection mass spectrometry使用电荷检测质谱法对AAV制造过程进行端到端表征
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-11-05
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deleteLohra M. Young; Benjamin E. Draper; Martin F. Jarrold; Clifford A. Froelich; Seyed Pouria Motevalian
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Characterization of Difficult-to-Remove Host Cell Proteins in Adeno-Associated Virus Downstream Processing难去除宿主细胞蛋白在腺相关病毒下游处理中的表征
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-10-30
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deleteThomas M. Leibiger; Lie Min; Kelvin H. Lee
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Gene therapy supports long-term reconstitution of patient hematopoietic stem cells in deficiency of adenosine deaminase 2基因疗法支持腺苷脱氨酶2缺乏症患者造血干细胞的长期重建
deleteMolecular Therapy Methods & Clinical Development
IF4.7
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deleteChiara Rigamonti; Dimitri Bulté; Federica Barzaghi; Cristina Mesa-Nuñez; Luca Basso-Ricci; Emanuela Pettinato; Camilla Visconti; Sara Degl’Innocenti; Francesco Gazzo; Fabrizio Benedicenti; Sergio Arévalo; Pamela Quaranta; Francesca Sanvito; Roberta Caorsi; Francesca Schena; Michela Lupia; Silvia Federici; Antonella Insalaco; Fabrizio De Benedetti; Serena Scala; Maurizio Miano; Francesca Conti; Maria Pia Cicalese; Eugenio Montini; Marco Gattorno; Carlo Dufour; Alessandro Aiuti; Alessandra Mortellaro
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Intrathalamic delivery of adeno-associated viral vector expressing progranulin as gene therapy for GRN-related frontotemporal dementia丘脑内递送表达progranulin的腺相关病毒载体作为GRN相关额颞叶痴呆的基因治疗
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-10-30
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deleteYoun Bok Lee; Do Young Lee; Zoe Walker; Joseph Allison; Aleksandra Kaliszewska; Alinda Fernandes; Katharine Y. Zhang; Yalem Bekele; Paniz Hosseini; Romain Joubert; Joana Furtado; McQuinn Gumus; Olivier Brock; Ali Bienemann; Steven Gill; Samantha Murray; Nadia Mitchell; Page Bouchard; Alex Bloom; John Isaac; Carlos J. Miranda; Christopher E. Shaw
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Nanobody-based gene therapy targeting complement component C3 reduces choroidal neovascularization in mice基于纳米抗体的基因疗法靶向补体成分C3可减少小鼠脉络膜新生血管形成
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-10-25
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deleteEmilie Grarup Jensen; Thomas Stax Jakobsen; Gloriane Schnabolk; Kyrie Wilson; Mathias Rask-Pedersen; Nanna Jensen; Gregers Rom Andersen; Steffen Thiel; Lars Aagaard; Bärbel Rohrer; Anne Louise Askou; Thomas J. Corydon
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