Results 101 to 110 of about 116,785 (352)
Performance of AAV8 vectors expressing human factor IX from a hepatic-selective promoter following intravenous injection into rats [PDF]
, 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
Production of recombinant adeno-associated virus
, 2000 Currently, rAAV appears to be one of the most promising vectors for gene therapy applications. Attractive features of the vector include nonpathogenicity, the ability to infect nondividing cells, escape from host immune responses, and integration into the host genome.
G P, Gao, J M, Wilson, N A, Wivel
openaire +2 more sources
Advanced Science, EarlyView.Elevated exogenous palmitic acid promotes the S‐palmitoylation of SRPK1 in endothelial cells, a dynamic process governed by ZDHHC24 and APT1. This post‐translational modification strengthens the interaction between SRPK1 and the E3 ubiquitin ligase MIB1, thereby facilitating the proteasomal degradation of SRPK1.
Xiao‐Hui Tan +11 more
wiley +1 more source
RNA interference approaches for treatment of HIV-1 infection. [PDF]
, 2015 HIV/AIDS is a chronic and debilitating disease that cannot be cured with current antiretroviral drugs. While combinatorial antiretroviral therapy (cART) can potently suppress HIV-1 replication and delay the onset of AIDS, viral mutagenesis often leads to
Bobbin, Maggie L +2 more
core +1 more source
Advanced Science, EarlyView.This study presents a novel “in vivo–in vitro” therapeutic strategy for spinal cord injury by leveraging magnetically responsive piezoelectric nanomaterials. These nanomaterials enable targeted delivery of localized electrical stimulation at the injury site through noninvasive external magnetic actuation, thereby promoting axonal regeneration and ...
Zhihang Xiao +9 more
wiley +1 more source
Association between adeno‐associated virus genomic titers and intracellular plasmid levels
Engineering ReportsThe recombinant adeno‐associated viral (rAAV) vector is one of the most effective viral vectors in gene therapy because of its low immunogenicity, high transduction efficiency, broad tissue specificity, and long‐term transgene expression ability. HEK293T
Kyoko Masumi‐Koizumi +4 more
doaj +1 more source
Potential Application of the CRISPR/Cas9 System against Herpesvirus Infections. [PDF]
, 2018 The CRISPR/Cas9 system has been applied in the genome editing and disruption of latent infections for herpesviruses such as the herpes simplex virus, Epstein⁻Barr virus, cytomegalovirus, and Kaposi's sarcoma-associated herpesvirus.
Chen, Yuan-Chuan +3 more
core +2 more sources
Advanced Science, EarlyView.This study shows that integrin receptor CD49a (Itga1 gene) is significantly upregulated in hyperactivated microglia and microglia‐specific knockdown of Itga1 rescues neuroinflammation and neurodegeneration in a chronic Parkinson's disease (PD) model by targeting PGAM5‐mediated mitochondrial dysfunction and NLRP3 activation. Targeted inhibition of CD49a
Huanpeng Lu +6 more
wiley +1 more source
Targeting DNA‐LNPs to Endothelial Cells Improves Expression Magnitude, Duration, and Specificity
Advanced Science, EarlyView.Attaching antibodies against endothelial cell surface proteins redirects the delivery and expression of DNA‐lipid nanoparticles to organs of interest. Our targeted nanoparticles enable organ‐selective DNA expression in the endothelium of the lungs, brain, or spleen, providing a therapeutic platform for dozens of endothelial‐centric diseases.
Nicolas Marzolini +24 more
wiley +1 more source