Gene Therapy
Papers 4422
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#1Warut Tulalamba (Vrije Universiteit Brussel)H-Index: 3
#2Jonas Weinmann (Heidelberg University)
Last.Dirk Grimm (Heidelberg University)H-Index: 38
view all 7 authors...
The human musculature is a promising and pivotal target for human gene therapy, owing to numerous diseases that affect this tissue and that are often monogenic, making them amenable to treatment and potentially cure on the genetic level. Particularly attractive would be the possibility to deliver clinically relevant DNA to muscle tissue from a minimally invasive, intravenous vector delivery. To date, this aim has been approximated by the use of Adeno-associated viruses (AAV) of different serotyp...
#1Laura Rodriguez-Estevez (UNC: University of North Carolina at Chapel Hill)
#2Priyadarsini Asokan (UNC: University of North Carolina at Chapel Hill)H-Index: 1
Last.Teresa Borrás (UNC: University of North Carolina at Chapel Hill)H-Index: 15
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The trabecular meshwork (TM) of the eye is responsible for maintaining physiological intraocular pressure (IOP). Dysfunction of this tissue results in elevated IOP, subsequent optic nerve damage and glaucoma, the world’s leading cause of irreversible blindness. IOP regulation by delivering candidate TM genes would offer an enormous clinical advantage to the current daily-drops/surgery treatment. Initially, we showed that a double-stranded AAV2 (scAAV2) transduced the human TM very efficiently, w...
#1Jian Kang (Tongji University)
#2Zhenhuan LiH-Index: 1
Last.Guanghui Xu (Tongji University)
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In this study, we aimed to investigate the therapeutic effect of miR-21 in the treatment of spinal cord injury (SCI) as well as its underlying molecular mechanisms. Real-time PCR and western blot were performed to measure the expression of miR-21, PTEN, and PDCD4 in SCI rats. Locomotion recovery assessment, Nissl staining, IHC assay, and TUNEL assay were utilized to observe the therapeutic effect of miR-21 in the treatment of SCI. Bioinformatics analysis and luciferase assay were conducted to es...
#1Wenwei Shao (UNC: University of North Carolina at Chapel Hill)H-Index: 1
#2Xiaolei Pei (UNC: University of North Carolina at Chapel Hill)H-Index: 1
Last.Chengwen Li (UNC: University of North Carolina at Chapel Hill)H-Index: 23
view all 11 authors...
Although therapeutic outcomes have been achieved in hemophilia patients after delivery of clotting factor genes to the liver using adeno-associated virus (AAV) vectors, it is well known that the preclinical results generated from hemophilia animal models have not been directly predictive of successful translation in humans. To address this discrepancy humanized mouse models have recently been used to predict AAV transduction efficiency for human hepatocytes. In this study we evaluated AAV vector...
#1Mariana Santana Dias (UFRJ: Federal University of Rio de Janeiro)
#2Victor Guedes de Araujo (UFRJ: Federal University of Rio de Janeiro)
Last.Hilda Petrs-Silva (UFRJ: Federal University of Rio de Janeiro)H-Index: 9
view all 7 authors...
Adeno-associated virus vectors (rAAV) are currently the most common vehicle used in clinical trials of retinal gene therapy, usually delivered through subretinal injections to target cells of the outer retina. However, targeting the inner retina requires intravitreal injections, a simple and safe procedure, which is effective for transducing the rodent retina, but still of low efficiency in the eyes of primates. We investigated whether adjuvant pharmacological agents may enhance rAAV transductio...
#1Sijing ZhouH-Index: 5
#2Ke Zhu (Anhui Medical University)
Last.Ran Wang (Anhui Medical University)H-Index: 7
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We aimed to investigate how estrogen (ES) is implicated in the pathogenesis of pulmonary arterial hypertension (PAH) potentially by reducing the extent of vascular remodeling in females. HE assay, Western Blot, IHC, and real-time PCR were carried out to observe the role of ES in regulating miR-133a expression and the levels of MYOSLID, SRF, CTGF, and vascular remodeling in rats. In addition, MTT assay and flow cytometry were utilized to observe how ES affects cell proliferation and cell cycle in...
#1Esteban Fiore (CONICET: National Scientific and Technical Research Council)H-Index: 9
#2Luciana M. Dominguez (Austral University)H-Index: 2
Last.Guillermo Mazzolini (CONICET: National Scientific and Technical Research Council)H-Index: 30
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Extracellular vesicles (EVs) can mediate mesenchymal stromal cells (MSCs) paracrine effects. We aimed to evaluate the therapeutic potential of human umbilical cord perivascular cells (HUCPVCs) engineered to produce Insulin Growth Factor like-I (IGF-I) in experimental liver fibrosis and the role of EVs in this effect. HUCPVCs were engineered to produce human IGF-I (AdhIGF-I) or green fluorescence protein (AdGFP) using adenoviruses, and EVs were isolated from their conditioned medium (CM). In vitr...
#1Sheng Lu (SJTU: Shanghai Jiao Tong University)H-Index: 1
#2Zheng-Gang Zhu (SJTU: Shanghai Jiao Tong University)H-Index: 1
Last.Wen-Cong Lu (SHU: Shanghai University)H-Index: 1
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Colorectal cancer (CRC) is the third most common type of cancer. In recent decades, genomic analysis has played an increasingly important role in understanding the molecular mechanisms of CRC. However, its pathogenesis has not been fully uncovered. Identification of genes related to CRC as complete as possible is an important way to investigate its pathogenesis. Therefore, we proposed a new computational method for the identification of novel CRC-associated genes. The proposed method is based on...
1 CitationsSource
#1Anna K. Blakney (Imperial College London)H-Index: 11
#2Paul F. McKay (Imperial College London)H-Index: 16
Last.Robin J. Shattock (Imperial College London)H-Index: 7
view all 5 authors...
Self-amplifying RNA (saRNA) is a promising biotherapeutic tool that has been used as a vaccine against both infectious diseases and cancer. saRNA has been shown to induce protein expression for up to 60 days and elicit immune responses with lower dosing than messenger RNA (mRNA). Because saRNA is a large (~9500 nt), negatively charged molecule, it requires a delivery vehicle for efficient cellular uptake and degradation protection. Lipid nanoparticles (LNPs) have been widely used for RNA formula...
#1Yong Zeng (NIH: National Institutes of Health)H-Index: 13
#2Haohua Qian (NIH: National Institutes of Health)H-Index: 12
Last.Ronald A. Bush (NIH: National Institutes of Health)H-Index: 17
view all 6 authors...
Recombinant adeno-associated virus (rAAV) has been widely used for gene delivery in animal models and successfully applied in clinical trials for treating inherited retinal disease. Although subretinal delivery of AAVs can effectively transduce photoreceptors and/or retinal pigmental epithelium (RPE), cells most affected by inherited retinal diseases, the procedure is invasive and complicated, and only delivers the gene to a limited retinal area. AAVs can also be delivered intravitreally to the ...
Top fields of study
Genetic enhancement
Gene delivery
Molecular biology