Gene augmentation for autosomal dominant retinitis pigmentosa using rhodopsin genomic loci nanoparticles in the P23H+/− knock-in murine model
Gene Therapy, Published online: 20 March 2023; doi:10.1038/s41434-023-00394-1Gene augmentation for autosomal dominant retinitis pigmentosa using rhodopsin genomic loci nanoparticles in the P23H+/− knock-in murine model (Source: Gene Therapy)
Source: Gene Therapy - March 20, 2023 Category: Genetics & Stem Cells Authors: Simna SP Rajendra N. Mitra Min Zheng Jared D. Chrispell Kai Wang Yong-Su Kwon Ellen R. Weiss Zongchao Han Source Type: research
Correction: Adjuvant properties of IFN-γ and GM-CSF in the scFv6.C4 DNA vaccine against CEA-expressing tumors
Gene Therapy, Published online: 20 March 2023; doi:10.1038/s41434-023-00396-zCorrection: Adjuvant properties of IFN-γ and GM-CSF in the scFv6.C4 DNA vaccine against CEA-expressing tumors (Source: Gene Therapy)
Source: Gene Therapy - March 20, 2023 Category: Genetics & Stem Cells Authors: Bianca Ferrarini Zanetti Camila Pontes Ferreira Jos é Ronnie Carvalho Vasconcelos Sang Won Han Source Type: research
Comment on: Premature delivery in the domestic sow in response to in utero delivery of AAV9 to fetal piglets
Gene Therapy, Published online: 14 March 2023; doi:10.1038/s41434-023-00395-0Comment on: Premature delivery in the domestic sow in response to in utero delivery of AAV9 to fetal piglets (Source: Gene Therapy)
Source: Gene Therapy - March 14, 2023 Category: Genetics & Stem Cells Authors: Apeksha Dave Cara L. Berkowitz Valerie L. Luks Brandon M. White Rohan Palanki Marco D. Carpenter John S. Riley Sourav K. Bose Haiying Li Li Li Pallavi V. Menon Shiva Teerdhala Mina Ebrahimi Philip W. Zoltick William H. Peranteau Source Type: research
Terminal hairpins improve protein expression in IRES-initiated mRNA in the absence of a cap and polyadenylated tail
Gene Therapy, Published online: 24 February 2023; doi:10.1038/s41434-023-00391-4Terminal hairpins improve protein expression in IRES-initiated mRNA in the absence of a cap and polyadenylated tail (Source: Gene Therapy)
Source: Gene Therapy - February 24, 2023 Category: Genetics & Stem Cells Authors: Victor Solodushko Brian Fouty Source Type: research
Novel rAAV vector mediated intrathecal HGF delivery has an impact on neuroimmune modulation in the ALS motor cortex with TDP-43 pathology
Gene Therapy, Published online: 24 February 2023; doi:10.1038/s41434-023-00383-4Novel rAAV vector mediated intrathecal HGF delivery has an impact on neuroimmune modulation in the ALS motor cortex with TDP-43 pathology (Source: Gene Therapy)
Source: Gene Therapy - February 24, 2023 Category: Genetics & Stem Cells Authors: Bar ış Genç Boram Nho Hana Seung Benjamin Helmold Huiwon Park Öge Gözütok Seunghyun Kim Jinil Park Sanghyun Ye Haneul Lee Nayeon Lee Seung-Shin Yu Sunyoung Kim Junghun Lee Hande Özdinler Source Type: research
Removal of the GAA repeat in the heart of a Friedreich’s ataxia mouse model using CjCas9
Gene Therapy, Published online: 14 February 2023; doi:10.1038/s41434-023-00387-0Removal of the GAA repeat in the heart of a Friedreich’s ataxia mouse model using CjCas9 (Source: Gene Therapy)
Source: Gene Therapy - February 14, 2023 Category: Genetics & Stem Cells Authors: Pouir é Yaméogo Catherine G érard Nathalie Majeau Jacques P. Tremblay Source Type: research
Sonoselective delivery using ultrasound and microbubbles combined with intravenous rAAV9 CLDN5-GFP does not increase endothelial gene expression
Gene Therapy, Published online: 13 February 2023; doi:10.1038/s41434-023-00389-ySonoselective delivery using ultrasound and microbubbles combined with intravenous rAAV9 CLDN5-GFP does not increase endothelial gene expression (Source: Gene Therapy)
Source: Gene Therapy - February 13, 2023 Category: Genetics & Stem Cells Authors: Rikke Hahn Kofoed Elizabeth M. Simpson Kullervo Hynynen Isabelle Aubert Source Type: research
Homology-directed repair of an MYBPC3 gene mutation in a rat model of hypertrophic cardiomyopathy
Gene Therapy, Published online: 10 February 2023; doi:10.1038/s41434-023-00384-3Homology-directed repair of an MYBPC3 gene mutation in a rat model of hypertrophic cardiomyopathy (Source: Gene Therapy)
Source: Gene Therapy - February 10, 2023 Category: Genetics & Stem Cells Authors: Jiali Nie Yu Han Zhiyuan Jin Weijian Hang Hongyang Shu Zheng Wen Li Ni Dao Wen Wang Source Type: research
Reversible stabilization of DNA/PEI complexes by reducible click-linkage between DNA and polymer. A new polyplex concept for lowering polymer quantity
Gene Therapy, Published online: 09 February 2023; doi:10.1038/s41434-023-00386-1Reversible stabilization of DNA/PEI complexes by reducible click-linkage between DNA and polymer. A new polyplex concept for lowering polymer quantity (Source: Gene Therapy)
Source: Gene Therapy - February 9, 2023 Category: Genetics & Stem Cells Authors: Delphine Maze Chantal Pichon Patrick Midoux Source Type: research
Antibody-based protection against respiratory syncytial virus in mice and their offspring through vectored immunoprophylaxis
Gene Therapy, Published online: 02 February 2023; doi:10.1038/s41434-023-00385-2Antibody-based protection against respiratory syncytial virus in mice and their offspring through vectored immunoprophylaxis (Source: Gene Therapy)
Source: Gene Therapy - February 2, 2023 Category: Genetics & Stem Cells Authors: Amira D. Rghei Jacob G. E. Yates Jordyn A. Lopes Xuiaoyan Zhan Matthew M. Guilleman Yanlong Pei Laura P. van Lieshout Lisa A. Santry Byram W. Bridle Khalil Karimi Brad Thompson Leonardo Susta James E. Crowe Jr. Sarah K. Wootton Source Type: research
Ptbp1 knockdown failed to induce astrocytes to neurons in vivo
Gene Therapy, Published online: 01 February 2023; doi:10.1038/s41434-023-00382-5Ptbp1 knockdown failed to induce astrocytes to neurons in vivo (Source: Gene Therapy)
Source: Gene Therapy - February 1, 2023 Category: Genetics & Stem Cells Authors: Guixiang Yang Zixiang Yan Xiaoqing Wu Meng Zhang Chunlong Xu Linyu Shi Hui Yang Kailun Fang Source Type: research
Improving adeno-associated viral (AAV) vector-mediated transgene expression in retinal ganglion cells: comparison of five promoters
Gene Therapy, Published online: 13 January 2023; doi:10.1038/s41434-022-00380-zImproving adeno-associated viral (AAV) vector-mediated transgene expression in retinal ganglion cells: comparison of five promoters (Source: Gene Therapy)
Source: Gene Therapy - January 13, 2023 Category: Genetics & Stem Cells Authors: Bart Nieuwenhuis Elise Laperrousaz James R. Tribble Joost Verhaagen James W. Fawcett Keith R. Martin Pete A. Williams Andrew Osborne Source Type: research
Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease
Gene Therapy, Published online: 11 January 2023; doi:10.1038/s41434-022-00381-yPreclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease (Source: Gene Therapy)
Source: Gene Therapy - January 11, 2023 Category: Genetics & Stem Cells Authors: Jey M. Jeyakumar Azadeh Kia Lawrence C. S. Tam Jenny McIntosh Justyna Spiewak Kevin Mills Wendy Heywood Elisa Chisari Noemi Castaldo Dani ël Verhoef Paniz Hosseini Petya Kalcheva Clement Cocita Carlos J. Miranda Miriam Canavese Jaminder Khinder Cecilia R Source Type: research
Empowering gene delivery with protein engineering platforms
Gene Therapy, Published online: 19 December 2022; doi:10.1038/s41434-022-00379-6Empowering gene delivery with protein engineering platforms (Source: Gene Therapy)
Source: Gene Therapy - December 19, 2022 Category: Genetics & Stem Cells Authors: Monika Kizerwetter Kevin Pietz Luke M. Tomasovic Jamie B. Spangler Source Type: research
Glyco-binding domain chimeric antigen receptors as a new option for cancer immunotherapy
Gene Therapy, Published online: 19 December 2022; doi:10.1038/s41434-022-00374-xGlyco-binding domain chimeric antigen receptors as a new option for cancer immunotherapy (Source: Gene Therapy)
Source: Gene Therapy - December 19, 2022 Category: Genetics & Stem Cells Authors: Anna-Katharina Franke Charlotte Wessolowski Vanessa Thaden Ingo M üller Kerstin Cornils Source Type: research
