First use of gene therapy to treat growth hormone resistant dwarfism in a mouse model
Gene Therapy, Published online: 01 February 2022; doi:10.1038/s41434-022-00313-wFirst use of gene therapy to treat growth hormone resistant dwarfism in a mouse model (Source: Gene Therapy)
Source: Gene Therapy - February 1, 2022 Category: Genetics & Stem Cells Authors: Kian Chuan Sia Shu Uin Gan Siti Humairah Mohd Rodhi Zhen Ying Fu John J. Kopchick Michael J. Waters Kok Onn Lee Source Type: research
Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy in cardiac and skeletal muscles in the DMDmdx rat model
Gene Therapy, Published online: 01 February 2022; doi:10.1038/s41434-022-00317-6Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy in cardiac and skeletal muscles in the DMDmdx rat model (Source: Gene Therapy)
Source: Gene Therapy - February 1, 2022 Category: Genetics & Stem Cells Authors: Audrey Bourdon Virginie Fran çois Liwen Zhang Aude Lafoux Bodvael Fraysse Gilles Toumaniantz Thibaut Larcher Tiphaine Girard Mireille Ledevin Cyrielle Lebreton Agn ès Hivonnait Anna Creismeas Marine Allais Basile Marie Justine Guguin V éronique Blouin Source Type: research
Targeting mAKAPβ expression as a therapeutic approach for ischemic cardiomyopathy
Gene Therapy, Published online: 01 February 2022; doi:10.1038/s41434-022-00321-wTargeting mAKAPβ expression as a therapeutic approach for ischemic cardiomyopathy (Source: Gene Therapy)
Source: Gene Therapy - February 1, 2022 Category: Genetics & Stem Cells Authors: Eliana C. Martinez Jinliang Li Jennifer Arthur Ataam Kristin Tokarski Hrishikesh Thakur Ioannis Karakikes Kimberly Dodge-Kafka Michael S. Kapiloff Source Type: research
Two high-fidelity variants: efSaCas9 and SaCas9-HF, which one is better?
Gene Therapy, Published online: 31 January 2022; doi:10.1038/s41434-022-00319-4Two high-fidelity variants: efSaCas9 and SaCas9-HF, which one is better? (Source: Gene Therapy)
Source: Gene Therapy - January 31, 2022 Category: Genetics & Stem Cells Authors: Jineng Lv Haitao Xi Xiujuan Lv Yue Zhou Jiahua Wang Haoran Chen Tong Yan Jiang Jin Junzhao Zhao Feng Gu Zongming Song Source Type: research
Antisense oligonucleotides as a potential treatment for brain deficits observed in myotonic dystrophy type 1
Gene Therapy, Published online: 25 January 2022; doi:10.1038/s41434-022-00316-7Antisense oligonucleotides as a potential treatment for brain deficits observed in myotonic dystrophy type 1 (Source: Gene Therapy)
Source: Gene Therapy - January 25, 2022 Category: Genetics & Stem Cells Authors: Siham Ait Benichou Dominic Jauvin Thi éry De Serres-Bérard Marion Pierre Karen K. Ling C. Frank Bennett Frank Rigo Genevieve Gourdon Mohamed Chahine Jack Puymirat Source Type: research
Rapid characterization of adeno-associated virus (AAV) gene therapy vectors by mass photometry
Gene Therapy, Published online: 20 January 2022; doi:10.1038/s41434-021-00311-4Rapid characterization of adeno-associated virus (AAV) gene therapy vectors by mass photometry (Source: Gene Therapy)
Source: Gene Therapy - January 20, 2022 Category: Genetics & Stem Cells Authors: Di Wu Philsang Hwang Tiansen Li Grzegorz Piszczek Source Type: research
Getting the upper hand in ALS
Gene Therapy, Published online: 20 January 2022; doi:10.1038/s41434-022-00314-9Getting the upper hand in ALS (Source: Gene Therapy)
Source: Gene Therapy - January 20, 2022 Category: Genetics & Stem Cells Authors: Clive N. Svendsen Source Type: research
Development of a cGMP-compliant process to manufacture donor-derived, CD45RA-depleted memory CD19-CAR T cells
Gene Therapy, Published online: 08 January 2022; doi:10.1038/s41434-021-00307-0Development of a cGMP-compliant process to manufacture donor-derived, CD45RA-depleted memory CD19-CAR T cells (Source: Gene Therapy)
Source: Gene Therapy - January 8, 2022 Category: Genetics & Stem Cells Authors: Young-In Kim-Hoehamer Janice M. Riberdy Fei Zheng Jeoungeun J. Park Na Shang Jean-Yves M étais Timothy Lockey Catherine Willis Salem Akel Jennifer Moore Michael M. Meagher M. Paulina Velasquez Brandon M. Triplett Aimee C. Talleur Stephen Gottschalk Sheng Source Type: research
Equitable access to cell and gene therapies in South Africa: opportunities and hurdles
Gene Therapy, Published online: 08 January 2022; doi:10.1038/s41434-021-00309-yEquitable access to cell and gene therapies in South Africa: opportunities and hurdles (Source: Gene Therapy)
Source: Gene Therapy - January 8, 2022 Category: Genetics & Stem Cells Authors: Candice Laverne Hendricks Marco Alessandrini Michael Sean Pepper Source Type: research
Biodistribution and immunity of adenovirus 5/35 and modified vaccinia Ankara vector vaccines against human immunodeficiency virus 1 clade C
Gene Therapy, Published online: 06 January 2022; doi:10.1038/s41434-021-00308-zBiodistribution and immunity of adenovirus 5/35 and modified vaccinia Ankara vector vaccines against human immunodeficiency virus 1 clade C (Source: Gene Therapy)
Source: Gene Therapy - January 6, 2022 Category: Genetics & Stem Cells Authors: Masaru Shimada Haibin Wang Motohide Ichino Takehiro Ura Nobuhisa Mizuki Kenji Okuda Source Type: research
Outer retinal transduction by AAV2-7m8 following intravitreal injection in a sheep model of CNGA3 achromatopsia
Gene Therapy, Published online: 02 December 2021; doi:10.1038/s41434-021-00306-1Outer retinal transduction by AAV2-7m8 following intravitreal injection in a sheep model of CNGA3 achromatopsia (Source: Gene Therapy)
Source: Gene Therapy - December 2, 2021 Category: Genetics & Stem Cells Authors: M. Ross A. Obolensky E. Averbukh M. Desrosiers R. Ezra-Elia H. Honig E. Yamin A. Rosov H. Dvir E. Gootwine E. Banin D. Dalkara R. Ofri Source Type: research
Upper motor neurons are a target for gene therapy and UCHL1 is necessary and sufficient to improve cellular integrity of diseased upper motor neurons
Gene Therapy, Published online: 02 December 2021; doi:10.1038/s41434-021-00303-4Upper motor neurons are a target for gene therapy and UCHL1 is necessary and sufficient to improve cellular integrity of diseased upper motor neurons (Source: Gene Therapy)
Source: Gene Therapy - December 2, 2021 Category: Genetics & Stem Cells Authors: Bar ış Genç Javier H. Jara Santana S. Sanchez Amiko K. B. Lagrimas Öge Gözütok Nuran Ko çak Yongling Zhu P. Hande Özdinler Source Type: research
Premature delivery in the domestic sow in response to in utero delivery of AAV9 to fetal piglets
Gene Therapy, Published online: 22 November 2021; doi:10.1038/s41434-021-00305-2Premature delivery in the domestic sow in response to in utero delivery of AAV9 to fetal piglets (Source: Gene Therapy)
Source: Gene Therapy - November 22, 2021 Category: Genetics & Stem Cells Authors: Kelly A. Rich Christopher G. Wier Jessica Russo Lingling Kong Patrick L. Heilman Anthony Reynolds Amy Knapp Megan G. Pino Elizabeth Keckley Lori Mattox Raphael A. Malbrue Charlotte J. Sumner Catalin Buhimschi Stephen J. Kolb Source Type: research
A short hepatitis C virus NS5A peptide expression by AAV vector modulates human T cell activation and reduces vector immunogenicity
Gene Therapy, Published online: 11 November 2021; doi:10.1038/s41434-021-00302-5A short hepatitis C virus NS5A peptide expression by AAV vector modulates human T cell activation and reduces vector immunogenicity (Source: Gene Therapy)
Source: Gene Therapy - November 11, 2021 Category: Genetics & Stem Cells Authors: Winston Colon-Moran Alan Baer Gauri Lamture Jack T. Stapleton Joseph W. Fischer Nirjal Bhattarai Source Type: research
Progress on siRNA-based gene therapy targeting secondary injury after intracerebral hemorrhage
Gene Therapy, Published online: 10 November 2021; doi:10.1038/s41434-021-00304-3Progress on siRNA-based gene therapy targeting secondary injury after intracerebral hemorrhage (Source: Gene Therapy)
Source: Gene Therapy - November 10, 2021 Category: Genetics & Stem Cells Authors: Daniyah A. Almarghalani Zahoor A. Shah Source Type: research
