Results 101 to 110 of about 326,770 (247)

Effective delivery of large genes to the retina by dual AAV vectors

EMBO Molecular Medicine, 2013
Retinal gene therapy with adeno‐associated viral (AAV) vectors is safe and effective in humans. However, AAV's limited cargo capacity prevents its application to therapies of inherited retinal diseases due to mutations of genes over 5 kb, like Stargardt ...
Ivana Trapani, Pasqualina Colella, Andrea Sommella, Carolina Iodice, Giulia Cesi, Sonia de Simone, Elena Marrocco, Settimio Rossi, Massimo Giunti, Arpad Palfi, Gwyneth J Farrar, Roman Polishchuk, Alberto Auricchio   +12 more
doaj   +1 more source

Universal Method for the Purification of Recombinant AAV Vectors of Differing Serotypes

Molecular Therapy: Methods & Clinical Development, 2018
The generation of clinical good manufacturing practices (GMP)-grade adeno-associated virus (AAV) vectors requires purification strategies that support the generation of vectors of high purity, and that exhibit a good safety and efficacy profile. To date,
Shelley A. Nass, Maryellen A. Mattingly, Denise A. Woodcock, Brenda L. Burnham, Jeffrey A. Ardinger, Shayla E. Osmond, Amy M. Frederick, Abraham Scaria, Seng H. Cheng, Catherine R. O’Riordan   +9 more
doaj   +1 more source

Self-complementary Adeno-associated Virus 2 (AAV)–T Cell Protein Tyrosine Phosphatase Vectors as Helper Viruses to Improve Transduction Efficiency of Conventional Single-Stranded AAV Vectors in Vitro and in Vivo

, 2004
Recombinant vectors based on adeno-associated virus type 2 (AAV) target the liver efficiently, but the transgene expression is limited to ∼5% of hepatocytes.
Yoder, Mervin C., Qing, Keyun, Zhong, Li, Li, Yanjun, Chan, Rebecca J., Chen, Linyuan, Weigel-Kelley, Kirsten A.   +6 more
core   +1 more source

ACSL4‐Dependent Lysosomal Lipid Peroxidation Links WTAP‐Mediated m6A Modification to Intervertebral Disc Degeneration

Advanced Science, EarlyView.
IL‐1β upregulates the protein level of WTAP, which promotes the m6A modification of ACSL4 mRNA in an IGF2BP2‐dependent manner, thereby enhancing its stability. The increased ACSL4 drives lipid peroxidation, leading to lysosomal membrane permeabilization (LMP) and impaired mitophagy, which collectively accelerate intervertebral disc degeneration (IVDD).
Shu Jia, Xu Gao, Laimin Zhu, Haixia Song, Jing Wu, Shang Chen, Sheng Gao, Lu Zhang, Chunyang Meng   +8 more
wiley   +1 more source

Macrophage RSAD2 Couples mtDNA Synthesis With a Self‐Amplifying Inflammatory Circuit to Orchestrate Tissue Repair

Advanced Science, EarlyView.
Macrophage RSAD2 dually regulates CMPK2—the rate‐limiting enzyme for mitochondrial DNA synthesis—by suppressing its ubiquitination and promoting its phosphorylation via Csnk2a2 recruitment. This amplifies mtDNA production, which simultaneously activates a cGAS‐STING‐IRF3‐RSAD2 feedforward loop and the NLRP3 inflammasome, driving a self‐sustaining ...
Haomiao Yuan, Shukui Du, Chunfei Li, Xinjie Li, Shuyang Mu, Anran Qu, Hengxu Yan, Chongchong Xu, Jinnong Yang, Jingkai Sun, Yijun Wang, Jiayang Xiao, Fuyuan Zhang, Linlin Wang, Tiegang Li, Rui Zhao, Dawei Guan   +16 more
wiley   +1 more source

Distinguishing Pseudotransduction and True Transduction Enables Characterization and Bioengineering of Extracellular Vesicle‐Adeno‐Associated Virus Vectors

Journal of Extracellular Vesicles
Adeno‐associated virus (AAV) gene therapies have achieved some clinical success, with multiple products reaching regulatory approval. Encapsulation of AAV vectors within engineered extracellular vesicles (EVs) is an emerging strategy which could help ...
Jonathan D. Boucher, Devin M. Stranford, Hailey I. Edelstein, Danielle Tullman‐Ercek, Neha P. Kamat, Joshua N. Leonard   +5 more
doaj   +1 more source

In vitro TNFα-mediated induction of NF-κB-responsive AAV vectors.

, 2013
HEK-293T cells were transfected with AAV-NF-EGFP (A) or with AAV-NF-hGDNF (B) vectors using the calcium phosphate coprecipitation method. 5×105 cells in 6-wells were transfected with 250 ng DNA.
Abdelwahed Chtarto (290728), Catherine Pythoud (290734), Ralph Clinkers (290737), Liliane Tenenbaum (290738), Marc Levivier (290735), Elias Gebara (290730), Katia Vermoesen (290731), Catherine Melas (290733), Ruth Luthi-Carter (18698), Olivier De Witte (290736), Olivier Bockstael (290729)   +10 more
core   +1 more source

Early Spatial and Contextual Coding Deficits in Hippocampal CA1 Precede Performance Decline in an Alzheimer's Disease Model

Advanced Science, EarlyView.
Two‐photon calcium imaging in behaving 5xFAD mice reveals early CA1 coding deficits before behavioral decline. Task‐related place cell activity and splitter cell trajectory coding are impaired, with reduced retrospective dominance and greater prospective weighting.
Yimei Li, Mary Ann Go, Hualong Zhang, Robertas Aleksynas, Juliana Kim, Mingyang Gao, Seigfred Prado, Magdalena Sastre, Simon R. Schultz   +8 more
wiley   +1 more source

OAF Blocks SIAH1‐Mediated Degradation of SCPX, a Therapeutic Strategy for MASLD

Advanced Science, EarlyView.
This study shows OAF directly binds to SCPX and inhibits its interaction with the E3 ligase SIAH1, thereby protecting SCPX from ubiquitin‐dependent degradation and stabilizing its levels. This OAF‐SCPX axis represents a novel pathway in lipid homeostasis, highlighting OAF as a promising therapeutic candidate for MASLD.
Zongxi Li, Jingyue Song, Kaili Ma, Shunan Tang, Shuqian Wang, Wenya Ji, Chen Lu, Ting Zhang, Huaping Zheng, Xiaowei Zheng, Shanshan Lai, Cheng Deng   +11 more
wiley   +1 more source

Construction and packaging of herpes simplex virus/adeno-associated virus (HSV/AAV) Hybrid amplicon vectors

, 2012
Herpes simplex virus type 1 (HSV-1)-based amplicon vectors conserve most properties of the parental virus: broad host range, the ability to transduce dividing and nondiving cells, and a large transgene capacity.
Saydam, O, Fraefel, C, Glauser, D L
core   +1 more source