Heat-shrinking DNA nanoparticles for in vivo gene delivery
Gene Therapy, Published online: 03 January 2020; doi:10.1038/s41434-019-0117-0Heat-shrinking DNA nanoparticles for in vivo gene delivery (Source: Gene Therapy)
Source: Gene Therapy - January 2, 2020 Category: Genetics & Stem Cells Authors: Basil Mathew Raghu Ramanathan Nathan A. Delvaux Jacob Poliskey Kevin G. Rice Source Type: research
Integrating gene delivery and gene-editing technologies by adenoviral vector transfer of optimized CRISPR-Cas9 components
Gene Therapy, Published online: 03 January 2020; doi:10.1038/s41434-019-0119-yIntegrating gene delivery and gene-editing technologies by adenoviral vector transfer of optimized CRISPR-Cas9 components (Source: Gene Therapy)
Source: Gene Therapy - January 2, 2020 Category: Genetics & Stem Cells Authors: Ignazio Maggio Hidde A. Zittersteijn Qian Wang Jin Liu Josephine M. Janssen Ivan Toral Ojeda Silv ère M. van der Maarel Arjan C. Lankester Rob C. Hoeben Manuel A. F. V. Gon çalves Source Type: research
A novel gene editing system to treat both Tay–Sachs and Sandhoff diseases
Gene Therapy, Published online: 02 January 2020; doi:10.1038/s41434-019-0120-5A novel gene editing system to treat both Tay–Sachs and Sandhoff diseases (Source: Gene Therapy)
Source: Gene Therapy - January 1, 2020 Category: Genetics & Stem Cells Authors: Li Ou Michael J. Przybilla Alexandru-Flaviu T ăbăran Paula Overn M. Gerard O ’Sullivan Xuntian Jiang Rohini Sidhu Pamela J. Kell Daniel S. Ory Chester B. Whitley Source Type: research
Suppression of miR-203-3p inhibits lipopolysaccharide induced human intervertebral disc inflammation and degeneration through upregulating estrogen receptor α
Gene Therapy, Published online: 02 January 2020; doi:10.1038/s41434-019-0118-zSuppression of miR-203-3p inhibits lipopolysaccharide induced human intervertebral disc inflammation and degeneration through upregulating estrogen receptor α (Source: Gene Therapy)
Source: Gene Therapy - January 1, 2020 Category: Genetics & Stem Cells Authors: Zhongxu Cai Kunpeng Li Keshi Yang Dawei Luo Hui Xu Source Type: research
Inhibition of miR-296-5p protects the heart from cardiac hypertrophy by targeting CACNG6
Gene Therapy, Published online: 16 December 2019; doi:10.1038/s41434-019-0109-0Inhibition of miR-296-5p protects the heart from cardiac hypertrophy by targeting CACNG6 (Source: Gene Therapy)
Source: Gene Therapy - December 15, 2019 Category: Genetics & Stem Cells Authors: Wei Wang Nian Liu Li Xin Yanfei Ruan Xin Du Rong Bai Jianzeng Dong ChangSheng Ma Source Type: research
Neonatal nonviral gene editing with the CRISPR/Cas9 system improves some cardiovascular, respiratory, and bone disease features of the mucopolysaccharidosis I phenotype in mice
Gene Therapy, Published online: 11 December 2019; doi:10.1038/s41434-019-0113-4Neonatal nonviral gene editing with the CRISPR/Cas9 system improves some cardiovascular, respiratory, and bone disease features of the mucopolysaccharidosis I phenotype in mice (Source: Gene Therapy)
Source: Gene Therapy - December 10, 2019 Category: Genetics & Stem Cells Authors: Roselena Silvestri Schuh Esteban Alberto Gonzalez Angela Maria Vicente Tavares Bruna Gazzi Seolin Lais de Souza Elias Luisa Natalia Pimentel Vera Francyne Kubaski Edina Poletto Roberto Giugliani Helder Ferreira Teixeira Ursula Matte Guilherme Baldo Source Type: research
MicroRNA-204 silencing relieves pain of cervical spondylotic radiculopathy by targeting GDNF
Gene Therapy, Published online: 09 December 2019; doi:10.1038/s41434-019-0114-3MicroRNA-204 silencing relieves pain of cervical spondylotic radiculopathy by targeting GDNF (Source: Gene Therapy)
Source: Gene Therapy - December 8, 2019 Category: Genetics & Stem Cells Authors: Wen-Sheng Shen Cun-Feng Li Zhi-Shui Zhou Nan-Nan Zhai Lu-Ping Pan Source Type: research
An engineered serum albumin-binding AAV9 capsid achieves improved liver transduction after intravenous delivery in mice
Gene Therapy, Published online: 09 December 2019; doi:10.1038/s41434-019-0107-2An engineered serum albumin-binding AAV9 capsid achieves improved liver transduction after intravenous delivery in mice (Source: Gene Therapy)
Source: Gene Therapy - December 8, 2019 Category: Genetics & Stem Cells Authors: Quan Jin Chunping Qiao Jianbin Li Juan Li Xiao Xiao Source Type: research
The deletion of mutant SOD1 via CRISPR/Cas9/sgRNA prolongs survival in an amyotrophic lateral sclerosis mouse model
Gene Therapy, Published online: 09 December 2019; doi:10.1038/s41434-019-0116-1The deletion of mutant SOD1 via CRISPR/Cas9/sgRNA prolongs survival in an amyotrophic lateral sclerosis mouse model (Source: Gene Therapy)
Source: Gene Therapy - December 8, 2019 Category: Genetics & Stem Cells Authors: Weisong Duan Moran Guo Le Yi Yakun Liu Zhongyao Li Yanqin Ma Guisen Zhang Yaling Liu Hui Bu Xueqin Song Chunyan Li Source Type: research
AAV shedding after intracoronary delivery: just a safety concern?
Gene Therapy, Published online: 03 December 2019; doi:10.1038/s41434-019-0115-2AAV shedding after intracoronary delivery: just a safety concern? (Source: Gene Therapy)
Source: Gene Therapy - December 2, 2019 Category: Genetics & Stem Cells Authors: Kelly P. Yamada Taro Kariya Kiyotake Ishikawa Source Type: research
Hsa_circ_0000285 functions as a competitive endogenous RNA to promote osteosarcoma progression by sponging hsa-miRNA-599
Gene Therapy, Published online: 29 November 2019; doi:10.1038/s41434-019-0112-5Hsa_circ_0000285 functions as a competitive endogenous RNA to promote osteosarcoma progression by sponging hsa-miRNA-599 (Source: Gene Therapy)
Source: Gene Therapy - November 28, 2019 Category: Genetics & Stem Cells Authors: Zhicai Zhang Feifei Pu Baichuan Wang Qiang Wu Jianxiang Liu Zengwu Shao Source Type: research
Treating genes and patients
Gene Therapy, Published online: 27 November 2019; doi:10.1038/s41434-019-0111-6Treating genes and patients (Source: Gene Therapy)
Source: Gene Therapy - November 26, 2019 Category: Genetics & Stem Cells Authors: John Wilson Source Type: research
A rare disease patient/caregiver perspective on fair pricing and access to gene-based therapies
Gene Therapy, Published online: 13 November 2019; doi:10.1038/s41434-019-0110-7A rare disease patient/caregiver perspective on fair pricing and access to gene-based therapies (Source: Gene Therapy)
Source: Gene Therapy - November 12, 2019 Category: Genetics & Stem Cells Authors: Wendy White Source Type: research
Long noncoding RNA RBMS3-AS3 acts as a microRNA-4534 sponge to inhibit the progression of prostate cancer by upregulating VASH1
Gene Therapy, Published online: 11 November 2019; doi:10.1038/s41434-019-0108-1Long noncoding RNA RBMS3-AS3 acts as a microRNA-4534 sponge to inhibit the progression of prostate cancer by upregulating VASH1 (Source: Gene Therapy)
Source: Gene Therapy - November 10, 2019 Category: Genetics & Stem Cells Authors: Zhenming Jiang Yuxi Zhang Xi Chen Pingeng Wu Dong Chen Source Type: research
Distinct transduction of muscle tissue in mice after systemic delivery of AAVpo1 vectors
Gene Therapy, Published online: 17 October 2019; doi:10.1038/s41434-019-0106-3Distinct transduction of muscle tissue in mice after systemic delivery of AAVpo1 vectors (Source: Gene Therapy)
Source: Gene Therapy - October 16, 2019 Category: Genetics & Stem Cells Authors: Warut Tulalamba Jonas Weinmann Quang Hong Pham Jihad El Andari Thierry VandenDriessche Marinee K. Chuah Dirk Grimm Source Type: research
