Results 61 to 70 of about 326,770 (247)
Molecular Therapy: Methods & Clinical Development, 2020 Adeno-associated virus (AAV) vectors have been commonly purified through density gradient ultracentrifugation (DGUC) or column chromatography methods.
Zhe Yu +5 more
doaj +1 more source
Advanced Science, EarlyView.The pre‐regenerative vascular niche (PVN) is essential for nerve repair, yet its endothelial blueprint remains unclear. We identified angiogenic ECs (AECs) as the dominant pre‐regenerative subset and found that antler blood–derived exosomes (AB‐EXO) promote repair via IMP3.
Jinsheng Huang +11 more
wiley +1 more source
Viral vector-mediated RNA interference in the retina [PDF]
, 2010 RNA interference (RNAi) is a highly conserved post-transcriptional gene silencing process triggered by double-stranded RNA (dsRNA) in eukaryotic cells.
Georgiadis, A.
core
AAV-CRISPR gene editing is negated by pre-existing immunity to Cas9
, 2023 Adeno-associated viral (AAV) vectors are a leading candidate for the delivery of CRISPR-Cas9 for therapeutic genome editing in vivo. However, AAV-based delivery involves persistent expression of the Cas9 nuclease, a bacterial protein.
Mark R. Tanner (8928290) +10 more
core +1 more source
Advanced Science, EarlyView.. ABSTRACT Neural presbycusis disrupts auditory timing and speech perception, yet its molecular basis remains poorly understood. This study identifies Cntn2 as the Ranvier's nodal molecule significantly downregulated in aged auditory nerves. Cntn2 heterozygous knockout (Cntn2+/−) mice showed elevated auditory brainstem response thresholds and prolonged
Penghui Chen +8 more
wiley +1 more source
Humoral immunity to AAV vectors in gene therapy: challenges and potential solutions
, 2013 International audienceGene transfer trials with adeno-associated virus (AAV) vectors have initiated to unveil the therapeutic potential of this approach, with some of the most exciting results coming from clinical studies of gene transfer for hemophilia ...
Mingozzi, F., Pavani, G., Masat, E.
core +4 more sources
Nature Communications, 2022 Recombinant adeno-associated viral (AAV) vectors are a promising gene delivery platform, but ongoing clinical trials continue to highlight a relatively narrow therapeutic window.
Trevor J. Gonzalez +14 more
doaj +1 more source
Molecular Therapy: Methods & Clinical Development, 2022 Recent clinical successes have intensified interest in using adeno-associated virus (AAV) vectors for therapeutic gene delivery. The liver is a key clinical target, given its critical physiological functions and involvement in a wide range of genetic ...
Marti Cabanes-Creus +14 more
doaj +1 more source
Advanced Science, EarlyView.. ABSTRACT Neuroinflammation driven by microglial activation is a defining feature of Alzheimer's disease (AD), yet the molecular mechanisms sustaining this proinflammatory state remain unclear. Here, we identify the deubiquitinase OTUD7B as a critical regulator of microglial activation and AD pathology.
Luyao Li +15 more
wiley +1 more source
Advanced Science, EarlyView.The present study shows that Nrf2 directly binds to the Gm26550 promoter, thereby activating Gm26550 transcription and increasing its expression. Mechanistically, Gm26550 promotes IGF1 expression by functionally antagonizing miR‐26a‐5p‐mediated repression and sequestering the RBP KHSRP, thereby enhancing hippocampal neuronal synaptic plasticity and ...
Hongfang Wang +12 more
wiley +1 more source