Highly efficient neuronal gene knockout in vivo by CRISPR-Cas9 via neonatal intracerebroventricular injection of AAV in mice
Gene Therapy, Published online: 08 February 2021; doi:10.1038/s41434-021-00224-2Highly efficient neuronal gene knockout in vivo by CRISPR-Cas9 via neonatal intracerebroventricular injection of AAV in mice (Source: Gene Therapy)
Source: Gene Therapy - February 8, 2021 Category: Genetics & Stem Cells Authors: Sam Hana Michael Peterson Helen McLaughlin Eric Marshall Attila J. Fabian Olivia McKissick Kathryn Koszka Galina Marsh Michael Craft Shanqin Xu Alexander Sorets Tess Torregrosa Chao Sun Chris E. Henderson Shih-Ching Lo Source Type: research
Comment on “Delivering cellular and gene therapies to patients: solutions for realizing the potential of the next generation of medicine”
Gene Therapy, Published online: 08 February 2021; doi:10.1038/s41434-021-00228-yComment on “Delivering cellular and gene therapies to patients: solutions for realizing the potential of the next generation of medicine” (Source: Gene Therapy)
Source: Gene Therapy - February 8, 2021 Category: Genetics & Stem Cells Authors: Liz Bell Source Type: research
Liver targeting with rAAV7: balancing tropism with immune profiles
Gene Therapy, Published online: 05 February 2021; doi:10.1038/s41434-021-00230-4Liver targeting with rAAV7: balancing tropism with immune profiles (Source: Gene Therapy)
Source: Gene Therapy - February 5, 2021 Category: Genetics & Stem Cells Authors: Terence R. Flotte Source Type: research
AAV1.NT-3 gene therapy for X-linked Charcot–Marie–Tooth neuropathy type 1
Gene Therapy, Published online: 04 February 2021; doi:10.1038/s41434-021-00231-3AAV1.NT-3 gene therapy for X-linked Charcot–Marie–Tooth neuropathy type 1 (Source: Gene Therapy)
Source: Gene Therapy - February 4, 2021 Category: Genetics & Stem Cells Authors: Burcak Ozes Morgan Myers Kyle Moss Jennifer Mckinney Alicia Ridgley Lei Chen Shasha Bai Charles K. Abrams Mona M. Freidin Jerry R. Mendell Zarife Sahenk Source Type: research
From macrophage biology to macrophage-based cellular immunotherapies
Gene Therapy, Published online: 04 February 2021; doi:10.1038/s41434-021-00221-5From macrophage biology to macrophage-based cellular immunotherapies (Source: Gene Therapy)
Source: Gene Therapy - February 4, 2021 Category: Genetics & Stem Cells Authors: Elvira Mass Nico Lachmann Source Type: research
Proteosomal degradation impairs transcytosis of AAV vectors from suprachoroidal space to retina
Gene Therapy, Published online: 04 February 2021; doi:10.1038/s41434-021-00233-1Proteosomal degradation impairs transcytosis of AAV vectors from suprachoroidal space to retina (Source: Gene Therapy)
Source: Gene Therapy - February 4, 2021 Category: Genetics & Stem Cells Authors: Kun Ding Jikui Shen Sean Hackett Mahmood Khan Peter A. Campochiaro Source Type: research
Correction: Targeted gene correction in the mdx mouse using short DNA fragments: towards application with bone marrow-derived cells for autologous remodeling of dystrophic muscle
Gene Therapy, Published online: 03 February 2021; doi:10.1038/s41434-020-00217-7Correction: Targeted gene correction in the mdx mouse using short DNA fragments: towards application with bone marrow-derived cells for autologous remodeling of dystrophic muscle (Source: Gene Therapy)
Source: Gene Therapy - February 3, 2021 Category: Genetics & Stem Cells Authors: R. M. Kapsa A. F. Quigley J. Vadolas K. Steeper P. A. Ioannou E. Byrne A. J. Kornberg Source Type: research
Human MiniPromoters for ocular-rAAV expression in ON bipolar, cone, corneal, endothelial, Müller glial, and PAX6 cells
Gene Therapy, Published online: 02 February 2021; doi:10.1038/s41434-021-00227-zHuman MiniPromoters for ocular-rAAV expression in ON bipolar, cone, corneal, endothelial, Müller glial, and PAX6 cells (Source: Gene Therapy)
Source: Gene Therapy - February 2, 2021 Category: Genetics & Stem Cells Authors: Andrea J. Korecki Jorge L. Cueva-Vargas Oriol Fornes Jessica Agostinone Rachelle A. Farkas Jack W. Hickmott Siu Ling Lam Anthony Mathelier Michelle Zhou Wyeth W. Wasserman Adriana Di Polo Elizabeth M. Simpson Source Type: research
Genome editing for Duchenne muscular dystrophy: a glimpse of the future?
Gene Therapy, Published online: 02 February 2021; doi:10.1038/s41434-021-00222-4Genome editing for Duchenne muscular dystrophy: a glimpse of the future? (Source: Gene Therapy)
Source: Gene Therapy - February 2, 2021 Category: Genetics & Stem Cells Authors: Christian Kupatt Alina Windisch Alessandra Moretti Eckhard Wolf Wolfgang Wurst Maggie C. Walter Source Type: research
Targeted multi-epitope switching enables straightforward positive/negative selection of CAR T cells
Gene Therapy, Published online: 01 February 2021; doi:10.1038/s41434-021-00220-6Targeted multi-epitope switching enables straightforward positive/negative selection of CAR T cells (Source: Gene Therapy)
Source: Gene Therapy - February 1, 2021 Category: Genetics & Stem Cells Authors: Laura Mosti Lukas M. Langner Kay O. Chmielewski Patrick Arbuthnot Jamal Alzubi Toni Cathomen Source Type: research
Single-cell mapping of focused ultrasound-transfected brain
Gene Therapy, Published online: 01 February 2021; doi:10.1038/s41434-021-00226-0Single-cell mapping of focused ultrasound-transfected brain (Source: Gene Therapy)
Source: Gene Therapy - February 1, 2021 Category: Genetics & Stem Cells Authors: A. S. Mathew C. M. Gorick R. J. Price Source Type: research
Development of a dual hybrid AAV vector for endothelial-targeted expression of von Willebrand factor
Gene Therapy, Published online: 17 January 2021; doi:10.1038/s41434-020-00218-6Development of a dual hybrid AAV vector for endothelial-targeted expression of von Willebrand factor (Source: Gene Therapy)
Source: Gene Therapy - January 17, 2021 Category: Genetics & Stem Cells Authors: Elena Barbon Charlotte Kawecki Solenne Marmier Aboud Sakkal Fanny Collaud Severine Charles Giuseppe Ronzitti Caterina Casari Olivier D. Christophe C écile V. Denis Peter J. Lenting Federico Mingozzi Source Type: research
Double viral vector technology for selective manipulation of neural pathways with higher level of efficiency and safety
Gene Therapy, Published online: 11 January 2021; doi:10.1038/s41434-020-00212-yDouble viral vector technology for selective manipulation of neural pathways with higher level of efficiency and safety (Source: Gene Therapy)
Source: Gene Therapy - January 11, 2021 Category: Genetics & Stem Cells Authors: Yoshinori Koshimizu Kaoru Isa Kenta Kobayashi Tadashi Isa Source Type: research
Validation of an IFN-gamma ELISpot assay to measure cellular immune responses against viral antigens in non-human primates
Gene Therapy, Published online: 11 January 2021; doi:10.1038/s41434-020-00214-wValidation of an IFN-gamma ELISpot assay to measure cellular immune responses against viral antigens in non-human primates (Source: Gene Therapy)
Source: Gene Therapy - January 11, 2021 Category: Genetics & Stem Cells Authors: Fan Yang Kathryn Patton Theresa Kasprzyk Brian Long Soumi Gupta Stephen J. Zoog Kristin Tracy Christian Vettermann Source Type: research
Restored microRNA-519a enhances the radiosensitivity of non-small cell lung cancer via suppressing EphA2
Gene Therapy, Published online: 07 January 2021; doi:10.1038/s41434-020-00213-xRestored microRNA-519a enhances the radiosensitivity of non-small cell lung cancer via suppressing EphA2 (Source: Gene Therapy)
Source: Gene Therapy - January 7, 2021 Category: Genetics & Stem Cells Authors: Shulei Gong Yu Li Ling Lv Wanfu Men Source Type: research
