Results 91 to 100 of about 115,653 (351)

Performance of AAV8 vectors expressing human factor IX from a hepatic-selective promoter following intravenous injection into rats [PDF]

open access: yes, 2008
Background: Vectors based on adeno-associated virus-8 (AAV8) have shown efficiency and efficacy for liver-directed gene therapy protocols following intravascular injection, particularly in relation to haemophilia gene therapy. AAV8 has also been proposed
Baker, AH   +4 more
core   +2 more sources

METTL1‐Mediated M7G tRNA Modification Promotes Residual Liver Regeneration After Hepatectomy via Translational Control

open access: yesAdvanced Science, EarlyView.
METTL1 and its mediated m7G tRNA modification are significantly up‐regulated after partial hepatectomy (PHx). Overexpression of METTL1 improves the efficiency of liver regeneration after PHx, increases the proliferation of hepatocytes, and accelerates the recovery of liver function.
Manling Huang   +18 more
wiley   +1 more source

Novel Cytotoxic Vectors Based on Adeno-Associated Virus

open access: yesToxins, 2010
Vectors based on adeno-associated virus (AAV) are promising tools for gene therapy. The production of strongly toxic vectors, for example for cancer-directed gene transfer, is often unfeasible due to uncontrolled expression of toxic genes in vector ...
Johannes Kohlschütter   +2 more
doaj   +1 more source

Iron Overload Mediates the Differential Cell Fate of Astrocytes from Neurons and Its Regulatory Mechanisms in Ischemic Stroke

open access: yesAdvanced Science, EarlyView.
Upon ischemia/reperfusion‐induced iron overload condition, neurons and astrocytes exhibit similar oxidative stress changes but opposite alterations in endogenous antioxidant defense mechanisms, leading to divergent ferroptotic outcomes. The iron deposition and neuronal loss are increased within the perilesional cortex of stroke patients ...
Yi Guo   +17 more
wiley   +1 more source

LncRNA THUMPD3‐AS1 Regulates Behavioral and Synaptic Structural Abnormalities in Schizophrenia via miR‐485‐5p and ARHGAP8

open access: yesAdvanced Science, EarlyView.
Schizophrenia (SCZ) is linked to synaptic structural deficits driven by dysregulated noncoding RNAs. Using the novel ceRNAxis pipeline, THUMPD3‐AS1/miR‐485‐5p/ARHGAP8 is identified as a key regulator of actin cytoskeletal remodeling through noncanonical RhoB/C‐ROCK2 signaling. Modulating this axis ameliorates SCZ‐like phenotypes in mice and aligns with
Xiaojuan Gong   +9 more
wiley   +1 more source

A novel polyethyleneimine-coated adeno-associated virus-like particle formulation for efficient siRNA delivery in breast cancer therapy: preparation and in vitro analysis

open access: yesInternational Journal of Nanomedicine, 2012
Wei Shao1, Arghya Paul1, Sana Abbasi1, Parminder S Chahal2, Jimmy A Mena2, Johnny Montes2, Amine Kamen2, Satya Prakash11Biomedical Technology and Cell Therapy Research Laboratory, Department of Biomedical Engineering and Artificial Cells and Organs ...
Shao W   +7 more
doaj  

To transduce a zebra finch: interrogating behavioral mechanisms in a model system for speech. [PDF]

open access: yes, 2017
The ability to alter neuronal gene expression, either to affect levels of endogenous molecules or to express exogenous ones, is a powerful tool for linking brain and behavior. Scientists continue to finesse genetic manipulation in mice.
Heston, Jonathan B, White, Stephanie A
core   +1 more source

Treatment with Minicircle DNA Expressing a FGF23 Fragment in a Clinically relevant Mouse Model of X‐Linked Hypophosphatemic Rickets

open access: yesAdvanced Science, EarlyView.
The pathogenic role of PHEX isn't fully determined, and there is no radical cure for X‐linked hypophosphatemic rickets (XLHR). This study makes the first attempt to perform gene therapy using a minicircle DNA (MC‐DNA) vector expressing a fragment of FGF23 (amino acids 180‐251) in Phex‐T1349C mice and suggests MC‐DNA as a promisingly safe and effective ...
Huixiao Wu   +20 more
wiley   +1 more source

Engineering a serum-resistant and thermostable vesicular stomatitis virus G glycoprotein for pseudotyping retroviral and lentiviral vectors. [PDF]

open access: yes, 2013
Vesicular stomatitis virus G glycoprotein (VSV-G) is the most widely used envelope protein for retroviral and lentiviral vector pseudotyping; however, serum inactivation of VSV-G pseudotyped vectors is a significant challenge for in vivo gene delivery ...
Hwang, B-Y, Schaffer, DV
core  

Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy [PDF]

open access: yes, 2018
Mutations in the gene encoding dystrophin, a protein that maintains muscle integrity and function, cause Duchenne muscular dystrophy (DMD). The deltaE50-MD dog model of DMD harbors a mutation corresponding to a mutational “hotspot” in the human DMD gene.
Amoasii, L   +12 more
core   +2 more sources

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