Featured
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Brief Communication |
Lentiviral gene therapy and vitamin B3 treatment enable granulocytic differentiation of G6PC3-deficient induced pluripotent stem cells
- Dirk Hoffmann
- , Johannes Kuehle
- & Axel Schambach
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Article |
LncRNA OIP5-AS1 interacts with miR-363-3p to contribute to hepatocellular carcinoma progression through up-regulation of SOX4
- Jianchu Wang
- , Qianli Tang
- & Jian Pu
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Article |
Cancer gene therapy by NF-κB-activated cancer cell-specific expression of CRISPR/Cas9 targeting telomeres
- Wei Dai
- , Jian Wu
- & Jinke Wang
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Article |
Adeno-associated virus vector enables safe and efficient Cas9 activation in neonatal and adult Cas9 knockin murine cochleae
- Wen Kang
- , Xingle Zhao
- & Hao Wu
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Article |
miR-19a/19b improves the therapeutic potential of mesenchymal stem cells in a mouse model of myocardial infarction
- Chengbo Chen
- , Tianbao Chen
- & Youbang Xu
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Article |
Suppression of miR-203-3p inhibits lipopolysaccharide induced human intervertebral disc inflammation and degeneration through upregulating estrogen receptor α
- Zhongxu Cai
- , Kunpeng Li
- & Hui Xu
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Article |
A novel gene editing system to treat both Tay–Sachs and Sandhoff diseases
- Li Ou
- , Michael J. Przybilla
- & Chester B. Whitley
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Article |
Neonatal nonviral gene editing with the CRISPR/Cas9 system improves some cardiovascular, respiratory, and bone disease features of the mucopolysaccharidosis I phenotype in mice
- Roselena Silvestri Schuh
- , Esteban Alberto Gonzalez
- & Guilherme Baldo
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Article |
MicroRNA-204 silencing relieves pain of cervical spondylotic radiculopathy by targeting GDNF
- Wen-Sheng Shen
- , Cun-Feng Li
- & Lu-Ping Pan
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Article |
The deletion of mutant SOD1 via CRISPR/Cas9/sgRNA prolongs survival in an amyotrophic lateral sclerosis mouse model
- Weisong Duan
- , Moran Guo
- & Chunyan Li
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Comment |
AAV shedding after intracoronary delivery: just a safety concern?
- Kelly P. Yamada
- , Taro Kariya
- & Kiyotake Ishikawa
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Review Article |
A rare disease patient/caregiver perspective on fair pricing and access to gene-based therapies
- Wendy White
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Article
| Open AccessTransduction optimization of AAV vectors for human gene therapy of glaucoma and their reversed cell entry characteristics
- Laura Rodriguez-Estevez
- , Priyadarsini Asokan
- & Teresa Borrás
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Article |
MiR-21 derived from the exosomes of MSCs regulates the death and differentiation of neurons in patients with spinal cord injury
- Jian Kang
- , Zhenhuan Li
- & Guanghui Xu
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Article |
Estrogen administration reduces the risk of pulmonary arterial hypertension by modulating the miR-133a signaling pathways in rats
- Sijing Zhou
- , Ke Zhu
- & Ran Wang
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Article |
Human umbilical cord perivascular cells-derived extracellular vesicles mediate the transfer of IGF-I to the liver and ameliorate hepatic fibrogenesis in mice
- Esteban Fiore
- , Luciana M. Domínguez
- & Guillermo Mazzolini
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Article |
Inferring novel genes related to oral cancer with a network embedding method and one-class learning algorithms
- Lei Chen
- , Yu-Hang Zhang
- & Yu-Dong Cai
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Article
| Open AccessDownregulation of the human peripheral myelin protein 22 gene by miR-29a in cellular models of Charcot–Marie–Tooth disease
- Jacquelyn Serfecz
- , Hannah Bazick
- & Lucia Notterpek
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Article |
Inferring novel genes related to colorectal cancer via random walk with restart algorithm
- Sheng Lu
- , Zheng-Gang Zhu
- & Wen-Cong Lu
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Review Article |
Beyond cystic fibrosis transmembrane conductance regulator therapy: a perspective on gene therapy and small molecule treatment for cystic fibrosis
- Elena K. Schneider-Futschik
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Article |
Gene therapy for refractory angina and cell therapy for heart failure: experience of a Brazilian research group
- Roberto Tofani Sant’Anna
- , Bruna Eibel
- & Renato Abdala Karam Kalil
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Brief Communication |
Design and applications of gene therapy vectors for mucopolysaccharidosis in Colombia
- Carlos J. Alméciga-Diaz
- & Luis A. Barrera
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Review Article |
AAV9 Vector: a Novel modality in gene therapy for spinal muscular atrophy
- Rithu Pattali
- , Yongchao Mou
- & Xue-Jun Li
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Brief Communication |
Safe and neuroprotective vectors for long-term traumatic brain injury gene therapy
- Daniela Blanco-Ocampo
- , Fabio Andrés Cawen
- & Hugo Peluffo
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Article |
Combination of cytokine-enhanced vaccine and chemo-gene therapy as surgery adjuvant treatments for spontaneous canine melanoma
- Liliana M. E. Finocchiaro
- , Lucrecia Agnetti
- & Gerardo C. Glikin
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Review Article |
Gene and cell therapy on the acquisition and relapse-like binge drinking in a model of alcoholism: translational options
- Yedy Israel
- , María Elena Quintanilla
- & Mario Herrera-Marschitz
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Article |
Safety and efficacy evaluations of an adeno-associated virus variant for preparing IL10-secreting human neural stem cell-based therapeutics
- Mira Cho
- , Kwangsoo Jung
- & Jae-Hyung Jang
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Article |
Dual-targeted NIS polyplexes—a theranostic strategy toward tumors with heterogeneous receptor expression
- Sarah Urnauer
- , Kathrin A. Schmohl
- & Christine Spitzweg
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Brief Communication
| Open AccessGDNF-mediated rescue of the nigrostriatal system depends on the degree of degeneration
- Luis Quintino
- , Martino Avallone
- & Cecilia Lundberg
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Article
| Open AccessImmunosuppression overcomes insulin- and vector-specific immune responses that limit efficacy of AAV2/8-mediated insulin gene therapy in NOD mice
- Asha Recino
- , Shu Uin Gan
- & Maja Wallberg
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Article |
Unilateral ex vivo gene therapy by GDNF in epileptic rats
- Avtandil Nanobashvili
- , Esbjörn Melin
- & Merab Kokaia
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Article |
Osteoinduction within BMP-2 transduced muscle tissue fragments with and without a fascia layer: implications for bone tissue engineering
- Bin Ren
- , Volker M. Betz
- & Oliver B. Betz
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Article |
Long noncoding RNA Meg3 regulates cardiomyocyte apoptosis in myocardial infarction
- Hongchun Wu
- , Zhen-Ao Zhao
- & Shijun Hu
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Article |
Anti-angiogenic treatment of endometriosis via anti-VEGFA siRNA delivery by means of peptide-based carrier in a rat subcutaneous model
- Anna Egorova
- , Mariya Petrosyan
- & Anton Kiselev
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Article |
Ectopic BAT mUCP-1 overexpression in SKM by delivering a BMP7/PRDM16/PGC-1a gene cocktail or single PRMD16 using non-viral UTMD gene therapy
- Shuyuan Chen
- , Raul A. Bastarrachea
- & Paul A. Grayburn
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Brief Communication |
Nanosphere-mediated co-delivery of VEGF-A and PDGF-B genes for accelerating diabetic foot ulcers healing in rats
- Rongfeng Shi
- , Weishuai Lian
- & Maoquan Li
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Brief Communication |
Additive effects of the combined expression of soluble forms of GAS1 and PTEN inhibiting glioblastoma growth
- Laura Sánchez-Hernández
- , Justino Hernández-Soto
- & José Segovia
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Brief Communication |
Electroporation-mediated delivery of FER gene enhances innate immune response and improves survival in a murine model of pneumonia
- Vladislav Dolgachev
- , Sreehari Panicker
- & David Machado-Aranda
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Brief Communication |
Effect of PAK1 gene silencing on proliferation and apoptosis in hepatocellular carcinoma cell lines MHCC97-H and HepG2 and cells in xenograft tumor
- Zhi-Lei Zhang
- , Guang-chao Liu
- & Lei Mao
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Comment
| Open AccessFludarabine and neurotoxicity in engineered T-cell therapy
- Kate L. Lowe
- , Crystal L. Mackall
- & Gwendolyn Binder
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Comment |
Understanding the molecular biology of intervertebral disc degeneration and potential gene therapy strategies for regeneration: a review
- Prasanthi Sampara
- , Rajkiran Reddy Banala
- & Subbaiah GPV
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Brief Communication |
MR-guided delivery of AAV2-BDNF into the entorhinal cortex of non-human primates
- Alan H. Nagahara
- , Bayard R. Wilson
- & Mark H. Tuszynski
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Brief Communication |
HIV-based lentivirus-mediated vasoactive intestinal peptide gene delivery protects against DIO animal model of Type 2 diabetes
- Hale M. Tasyurek
- , Yunus E. Eksi
- & Salih Sanlioglu
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Original Article |
CCAT1 stimulation of the symmetric division of NSCLC stem cells through activation of the Wnt signalling cascade
- C Xu
- , G Xiao
- & X Sun
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