Adeno-associated virus mediated expression of monoclonal antibody MR191 protects mice against Marburg virus and provides long-term expression in sheep
Gene Therapy, Published online: 01 September 2022; doi:10.1038/s41434-022-00361-2Adeno-associated virus mediated expression of monoclonal antibody MR191 protects mice against Marburg virus and provides long-term expression in sheep (Source: Gene Therapy)
Source: Gene Therapy - September 1, 2022 Category: Genetics & Stem Cells Authors: Amira D. Rghei Laura P. van Lieshout Wenguang Cao Shihua He Kevin Tierney Jordyn A. Lopes Nicole Zielinska Enzo M. Baracuhy Elena S. B. Campbell Jessica A. Minott Matthew M. Guilleman Pamela C. Hasson Brad Thompson Khalil Karimi Byram W. Bridle Leonardo S Source Type: research
Multiplex viral tropism assay in complex cell populations with single-cell resolution
Gene Therapy, Published online: 23 August 2022; doi:10.1038/s41434-022-00360-3Multiplex viral tropism assay in complex cell populations with single-cell resolution (Source: Gene Therapy)
Source: Gene Therapy - August 23, 2022 Category: Genetics & Stem Cells Authors: Choong Tat Keng Ke Guo Yu-Chi Liu Kimberle Yanyin Shen Daryl Shern Lim Matthew Lovatt Heng Pei Ang Jodhbir S. Mehta Wei Leong Chew Source Type: research
Preclinical assessment of antigen-specific chimeric antigen receptor regulatory T cells for use in solid organ transplantation
Gene Therapy, Published online: 05 August 2022; doi:10.1038/s41434-022-00358-xPreclinical assessment of antigen-specific chimeric antigen receptor regulatory T cells for use in solid organ transplantation (Source: Gene Therapy)
Source: Gene Therapy - August 5, 2022 Category: Genetics & Stem Cells Authors: Emma Proics Marion David Majid Mojibian Madeline Speck Nadia Lounnas-Mourey Adeline Govehovitch Wissam Baghdadi Justine Desnouveaux Herv é Bastian Laura Freschi Geoffrey Privat C édric Pouzet Mauro Grossi Pierre Heimendinger Tobias Abel David Fenard Meg Source Type: research
rAAV-mediated over-expression of acid ceramidase prevents retinopathy in a mouse model of Farber lipogranulomatosis
Gene Therapy, Published online: 28 July 2022; doi:10.1038/s41434-022-00359-wrAAV-mediated over-expression of acid ceramidase prevents retinopathy in a mouse model of Farber lipogranulomatosis (Source: Gene Therapy)
Source: Gene Therapy - July 28, 2022 Category: Genetics & Stem Cells Authors: Hanmeng Zhang Murtaza S. Nagree Haoyuan Liu Xiaoqing Pan Jeffrey A. Medin Daniel M. Lipinski Source Type: research
A novel preclinical model of mucopolysaccharidosis type II for developing human hematopoietic stem cell gene therapy
Gene Therapy, Published online: 14 July 2022; doi:10.1038/s41434-022-00357-yA novel preclinical model of mucopolysaccharidosis type II for developing human hematopoietic stem cell gene therapy (Source: Gene Therapy)
Source: Gene Therapy - July 14, 2022 Category: Genetics & Stem Cells Authors: Yohta Shimada Natsumi Ishii Takashi Higuchi Motohito Goto Toya Ohashi Hiroshi Kobayashi Source Type: research
Loss of RAGE prevents chronic intermittent hypoxia-induced nonalcoholic fatty liver disease via blockade of NF-кB pathway
Gene Therapy, Published online: 12 July 2022; doi:10.1038/s41434-022-00351-4Loss of RAGE prevents chronic intermittent hypoxia-induced nonalcoholic fatty liver disease via blockade of NF-кB pathway (Source: Gene Therapy)
Source: Gene Therapy - July 12, 2022 Category: Genetics & Stem Cells Authors: Haoqu Tan Jinfang Hu Wei Zuo Yun Huang Jian Cui Fei Gong Wei Bai Source Type: research
Selective attachment of a microtubule interacting peptide to plasmid DNA via a triplex forming oligonucleotide for transfection improvement
Gene Therapy, Published online: 07 July 2022; doi:10.1038/s41434-022-00354-1Selective attachment of a microtubule interacting peptide to plasmid DNA via a triplex forming oligonucleotide for transfection improvement (Source: Gene Therapy)
Source: Gene Therapy - July 7, 2022 Category: Genetics & Stem Cells Authors: Caroline Girardin Delphine Maze Cristine Gon çalves Yann Thierry Le Guen Kevin Pluchon Chantal Pichon Tristan Montier Patrick Midoux Source Type: research
Identification of RPGR ORF15 mutation for X-linked retinitis pigmentosa in a large Chinese family and in vitro correction with prime editor
Gene Therapy, Published online: 06 July 2022; doi:10.1038/s41434-022-00352-3Identification of RPGR ORF15 mutation for X-linked retinitis pigmentosa in a large Chinese family and in vitro correction with prime editor (Source: Gene Therapy)
Source: Gene Therapy - July 6, 2022 Category: Genetics & Stem Cells Authors: Xiujuan Lv Zheng Zheng Xiao Zhi Yilin Zhou Jineng Lv Yue Zhou Binrong Wu Sixiu Liu Wei Shi Zongming Song Jinling Xu Jia Qu Dan Xu Feng Gu Source Type: research
Intracerebral lentiviral ABCD1 gene therapy in an early disease onset ALD mouse model
Gene Therapy, Published online: 06 July 2022; doi:10.1038/s41434-022-00355-0Intracerebral lentiviral ABCD1 gene therapy in an early disease onset ALD mouse model (Source: Gene Therapy)
Source: Gene Therapy - July 6, 2022 Category: Genetics & Stem Cells Authors: Jie Gong Yunyun Liu Tsai-Hua Chung Liu Xu Troy C. Lund Lung-Ji Chang Source Type: research
Cerebral organoids as an in vitro model to study autism spectrum disorders
Gene Therapy, Published online: 05 July 2022; doi:10.1038/s41434-022-00356-zCerebral organoids as an in vitro model to study autism spectrum disorders (Source: Gene Therapy)
Source: Gene Therapy - July 5, 2022 Category: Genetics & Stem Cells Authors: Alexa Rabeling Mubeen Goolam Source Type: research
Clinical enrollment assay to detect preexisting neutralizing antibodies to AAV6 with demonstrated transgene expression in gene therapy trials
Gene Therapy, Published online: 01 July 2022; doi:10.1038/s41434-022-00353-2Clinical enrollment assay to detect preexisting neutralizing antibodies to AAV6 with demonstrated transgene expression in gene therapy trials (Source: Gene Therapy)
Source: Gene Therapy - July 1, 2022 Category: Genetics & Stem Cells Authors: Liching Cao Annemarie Ledeboer Yonghua Pan Yanmei Lu Kathleen Meyer Source Type: research
Splicing mutations in the CFTR gene as therapeutic targets
Gene Therapy, Published online: 02 June 2022; doi:10.1038/s41434-022-00347-0Splicing mutations in the CFTR gene as therapeutic targets (Source: Gene Therapy)
Source: Gene Therapy - June 2, 2022 Category: Genetics & Stem Cells Authors: Karine Deletang Magali Taulan-Cadars Source Type: research
Assessment of AAV9 distribution and transduction in rats after administration through Intrastriatal, Intracisterna magna and Lumbar Intrathecal routes
Gene Therapy, Published online: 30 May 2022; doi:10.1038/s41434-022-00346-1Assessment of AAV9 distribution and transduction in rats after administration through Intrastriatal, Intracisterna magna and Lumbar Intrathecal routes (Source: Gene Therapy)
Source: Gene Therapy - May 30, 2022 Category: Genetics & Stem Cells Authors: Jayanth Chandran Ekram Ahmed Chowdhury Michael Perkinton Tanguy Jamier Daniel Sutton Shengjia Wu Claire Dobson Dhaval K. Shah Iain Chessell Guy M. L. Meno-Tetang Source Type: research
Forced activation of dystrophin transcription by CRISPR/dCas9 reduced arrhythmia susceptibility via restoring membrane Nav1.5 distribution
Gene Therapy, Published online: 30 May 2022; doi:10.1038/s41434-022-00348-zForced activation of dystrophin transcription by CRISPR/dCas9 reduced arrhythmia susceptibility via restoring membrane Nav1.5 distribution (Source: Gene Therapy)
Source: Gene Therapy - May 30, 2022 Category: Genetics & Stem Cells Authors: Ruixin Zhang Junwu Liu Genlong Xue Jiming Yang Desheng Li Tao Tian Xiaofang Zhang Kangyi Gao Zhenwei Pan Source Type: research
Curing SMA: Are we there yet?
Gene Therapy, Published online: 26 May 2022; doi:10.1038/s41434-022-00349-yCuring SMA: Are we there yet? (Source: Gene Therapy)
Source: Gene Therapy - May 26, 2022 Category: Genetics & Stem Cells Authors: Aoife Reilly Lucia Chehade Rashmi Kothary Source Type: research
