Results 131 to 140 of about 89,643 (298)
, 2016 Use of viral vectors to deliver therapeutic genes to the central nervous system holds promise for the treatment of neurodegenerative diseases and neurotrauma.
Chrisna J LeVaillant +15 more
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Advanced Science, EarlyView.TGFBI deficiency elevates O‐GlcNAcylation, promoting the transcriptional activation of ADAMTS16 and OGT‐mediated post‐translational modification of ADAMTS16 at Ser1170. This leads to its stabilization and accumulation, driving chondrocyte metabolic disorders and senescent phenotypes, ultimately resulting in fibrocartilage dysfunction and TMJOA ...
Xin Liu +12 more
wiley +1 more source
AAV genome loss from dystrophic mouse muscles during AAV-U7 snRNA-mediated exon-skipping therapy
, 2016 In the context of future adeno-associated viral (AAV)-based clinical trials for Duchenne myopathy, AAV genome fate in dystrophic muscles is of importance considering the viral capsid immunogenicity that prohibits recurring treatments.
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Advanced Science, EarlyView.Sepsis triggers hippocampal SIRT7 loss, promoting histone H2B succinylation and activation of the PD‐1/PD‐L1–PINK1 axis. This metabolic‐epigenetic cascade drives aberrant mitophagy and neuronal injury, ultimately causing cognitive deficits. Targeting SIRT7‐dependent succinylation offers a potential strategy to protect brain function during sepsis ...
Na Meng +12 more
wiley +1 more source
AAV-mediated gene therapies for glaucoma and uveitis: Are we there yet?
Glaucoma and uveitis are non-vascular ocular diseases which are among the leading causes of blindness and visual loss. These conditions have distinct characteristics and mechanisms but share a multifactorial and complex nature, making their management ...
Jason Steel (9837167) +2 more
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Doxorubicin accelerates and enhances AAV-based transgene expression in the cat and rat brain
, 2017 PURPOSE: Recombinant adeno-associated viruses (AAV) are widely used vectors for gene transfer into the central nervous system (CNS).However, Low transduction efficiency andthe delay in transgene expression have hampered it from wide application in such
Wu Jihong +8 more
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Transposition efficiency of the AAV/SB transposase hybrid-vector system.
, 2013 Colony forming assay to determine integration efficiencies from the hybrid AAV/SB vector system in HeLa-cells. HeLa-cells were co-infected with AAV-neo and AAV-SB100X at increasing dosages (MOI 100, 1,000, 10,000 and 50,000).
Nadine Müther (413712) +7 more
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Engineered Transformer Base Editor with Enhanced Editing Efficiency
Advanced Science, EarlyView.A highly efficient transformer base editor system achieves robust genomic editing in a humanized mouse model. This work establishes a versatile and translatable platform, opening new avenues for precision gene therapy. ABSTRACT Canonical cytosine base editors (CBEs) achieve precise C‐to‐T conversions without inducing DNA double‐strand breaks (DSBs ...
Bowen Chen +5 more
wiley +1 more source
, 2016 Although adeno-associated virus (AAV)-2 has a broad tissue-host range and can transduce a wide variety of tissue type, some cells, such as erythro-megakaryoblastoid cells, are non-permissive and appear to lack the AAV-2 receptor. However, limited studies
core
Interleukin‐39 is a Prognostic Biomarker and Therapy Target for Sepsis
Advanced Science, EarlyView.Sepsis triggers a significant increase in IL‐39 production, with circulating levels markedly elevated and positively correlated with disease severity and poor clinical prognosis in sepsis patients. Mechanistically, macrophage‐derived IL‐39 activates the GP130 signaling pathway via a ligand‐receptor interaction, thereby fueling the pro‐inflammatory ...
Feng‐zhi Zhang +9 more
wiley +1 more source