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| Open AccessFunctional expression of complement factor I following AAV-mediated gene delivery in the retina of mice and human cells
- Anna K. Dreismann
- , Michelle E. McClements
- & Robert E. MacLaren
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Article |
Mesenchymal stem cells overexpressing BMP-9 by CRISPR-Cas9 present high in vitro osteogenic potential and enhance in vivo bone formation
- Gileade P. Freitas
- , Helena B. Lopes
- & Adalberto L. Rosa
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Article |
Ectopic expression of BBS1 rescues male infertility, but not retinal degeneration, in a BBS1 mouse model
- Matthew R. Cring
- , Kacie J. Meyer
- & Val C. Sheffield
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Review Article |
Genetic insights, disease mechanisms, and biological therapeutics for Waardenburg syndrome
- Sida Huang
- , Jian Song
- & Yong Feng
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Review Article |
Insights and future directions of potential genetic therapy for Apert syndrome: A systematic review
- Nisreen Mohammed Al-Namnam
- , Soher Nagi Jayash
- & Mohammed Abdullah Alshawsh
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Review Article
| Open AccessImplications of hematopoietic stem cells heterogeneity for gene therapies
- Jeremy Epah
- & Richard Schäfer
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Review Article
| Open AccessGene delivery to the hypoglossal motor system: preclinical studies and translational potential
- Brendan M. Doyle
- , Michele L. Singer
- & David D. Fuller
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Review Article
| Open AccessCas9-directed immune tolerance in humans—a model to evaluate regulatory T cells in gene therapy?
- Dimitrios Laurin Wagner
- , Lena Peter
- & Michael Schmueck-Henneresse
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Article |
Proteosomal degradation impairs transcytosis of AAV vectors from suprachoroidal space to retina
- Kun Ding
- , Jikui Shen
- & Peter A. Campochiaro
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Article
| Open AccessAAV1.NT-3 gene therapy for X-linked Charcot–Marie–Tooth neuropathy type 1
- Burcak Ozes
- , Morgan Myers
- & Zarife Sahenk
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Review Article
| Open AccessGenome editing for Duchenne muscular dystrophy: a glimpse of the future?
- Christian Kupatt
- , Alina Windisch
- & Maggie C. Walter
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Article
| Open AccessHuman MiniPromoters for ocular-rAAV expression in ON bipolar, cone, corneal, endothelial, Müller glial, and PAX6 cells
- Andrea J. Korecki
- , Jorge L. Cueva-Vargas
- & Elizabeth M. Simpson
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Brief Communication
| Open AccessSingle-cell mapping of focused ultrasound-transfected brain
- A. S. Mathew
- , C. M. Gorick
- & R. J. Price
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Brief Communication
| Open AccessDevelopment of a dual hybrid AAV vector for endothelial-targeted expression of von Willebrand factor
- Elena Barbon
- , Charlotte Kawecki
- & Federico Mingozzi
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Article |
Restored microRNA-519a enhances the radiosensitivity of non-small cell lung cancer via suppressing EphA2
- Shulei Gong
- , Yu Li
- & Wanfu Men
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Article
| Open AccessSite-specific modifications to AAV8 capsid yields enhanced brain transduction in the neonatal MPS IIIB mouse
- Janine A. Gilkes
- , Benjamin L. Judkins
- & Coy D. Heldermon
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Review Article |
Effects of oncolytic viruses and viral vectors on immunity in glioblastoma
- Penghao Liu
- , Yaning Wang
- & Yu Wang
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Brief Communication |
Molecular targeting of vulnerable RNA sequences in SARS CoV-2: identifying clinical feasibility
- G. Ian Gallicano
- , John L. Casey
- & Samiksha Mahapatra
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Review Article |
Self-amplifying RNA vaccines for infectious diseases
- Kristie Bloom
- , Fiona van den Berg
- & Patrick Arbuthnot
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Article |
Long noncoding RNA CA3-AS1 suppresses gastric cancer migration and invasion by sponging miR-93-5p and targeting BTG3
- Xiao-Yu Zhang
- , Hai-Wen Zhuang
- & Jin Dou
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Article |
Adenovirus vector-mediated YKL-40 shRNA attenuates eosinophil airway inflammation in a murine asthmatic model
- Ling Wang
- , Aihua Bao
- & Suqin Ben
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Review Article |
Gene therapy and gene correction: targets, progress, and challenges for treating human diseases
- Matthew R. Cring
- & Val C. Sheffield
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Article |
Overexpression of miR-144-3p alleviates polycystic ovaries syndrome through targeting expression of HSP-70
- Bing Qu
- , Qinghong Zhao
- & Yan Zhang
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Article |
Silencing of long non-coding RNA FOXD2-AS1 inhibits the progression of gallbladder cancer by mediating methylation of MLH1
- Jun Gao
- , Chao Dai
- & Fan Zhou
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Article |
Therapeutic benefit after intracranial gene therapy delivered during the symptomatic stage in a feline model of Sandhoff disease
- Victoria J. McCurdy
- , Aime K. Johnson
- & Douglas R. Martin
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Article |
LncRNA SNHG16 promotes colorectal cancer cell proliferation, migration, and epithelial–mesenchymal transition through miR-124-3p/MCP-1
- Zhi-Yuan Chen
- , Xiang-Yang Wang
- & Ya Peng
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Limited potential of AAV-mediated gene therapy in transducing human mesenchymal stem cells for bone repair applications
- Sofia Bougioukli
- , Morgan Chateau
- & Jay R. Lieberman
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Article |
Lentiviral gene therapy vectors encoding VIP suppressed diabetes-related inflammation and augmented pancreatic beta-cell proliferation
- Fulya Erendor
- , Elif Ozgecan Sahin
- & Salih Sanlioglu
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Article
| Open AccessInvestigation of the safety and feasibility of AAV1/SERCA2a gene transfer in patients with chronic heart failure supported with a left ventricular assist device – the SERCA-LVAD TRIAL
- A. R. Lyon
- , D. Babalis
- & S. E. Harding
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Review Article |
Gene therapeutic strategies and relevant clinical trials in neuromuscular disorder in China
- Chaoping Hu
- & Xihua Li
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Comment |
Retinal gene therapy: an eye-opener of the 21st century
- Anne Louise Askou
- , Thomas Stax Jakobsen
- & Thomas J. Corydon
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Review Article |
An overview of development in gene therapeutics in China
- Dawei Wang
- , Kang Wang
- & Yujia Cai
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Article |
LncRNA SNHG16 induces proliferation and fibrogenesis via modulating miR-141-3p and CCND1 in diabetic nephropathy
- Xiaohong Jiang
- , Qianying Ru
- & Shan Huang
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Review Article |
Optimization of antisense-mediated exon skipping for Duchenne muscular dystrophy
- Kasia Dzierlega
- & Toshifumi Yokota
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Article |
Treating Bietti crystalline dystrophy in a high-fat diet-exacerbated murine model using gene therapy
- Bin Qu
- , Shijing Wu
- & Wei Li
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Article |
Sigma-1 receptor activity in primary sensory neurons is a critical driver of neuropathic pain
- Seung Min Shin
- , Fei Wang
- & Hongwei Yu
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Review Article |
Gene therapy for cardiovascular diseases in China: basic research
- Jiali Deng
- , Mengying Guo
- & Junjie Xiao
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Article
| Open AccessAAV-mediated cardiac gene transfer of wild-type desmin in mouse models for recessive desminopathies
- T. Ruppert
- , M. B. Heckmann
- & O. J. Müller
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Article
| Open AccessMuscle overexpression of Klf15 via an AAV8-Spc5-12 construct does not provide benefits in spinal muscular atrophy mice
- Nina Ahlskog
- , Daniel Hayler
- & Melissa Bowerman
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Comment
| Open AccessToward a new framework for the development of individualized therapies
- Peter Marks
- & Celia Witten
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Article |
Targeted gene therapy into a safe harbor site in human hematopoietic progenitor cells
- Fatima Rodriguez-Fornes
- , Oscar Quintana-Bustamante
- & Guillermo Guenechea
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Article |
MiR-140-5p inhibits oxidized low-density lipoprotein-induced oxidative stress and cell apoptosis via targeting toll-like receptor 4
- Huifang Liu
- , Ziming Mao
- & Fengling Chen
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Review Article |
Reactivation of FMR1 gene expression is a promising strategy for fragile X syndrome therapy
- Ekaterina M. Shitik
- , Anastasia A. Velmiskina
- & Dmitry V. Yudkin
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Article |
AAV3-miRNA vectors for growth suppression of human hepatocellular carcinoma cells in vitro and human liver tumors in a murine xenograft model in vivo
- Ling Yin
- , Geoffrey D. Keeler
- & Arun Srivastava
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Particulate mediators of the bystander effect linked to suicide and interferon-β transgene expression in melanoma cells
- Lucrecia Agnetti
- , Chiara Fondello
- & Liliana M. E. Finocchiaro
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