Results 41 to 50 of about 9,370,992 (242)
Advanced Science, EarlyView.Mechanical stress activates BAP31 in chondrocytes. BAP31 competes with ATG14 for binding to STX17, disrupting the STX17–ATG14 complex required for autophagosome–lysosome fusion. The resultant autophagic flux blockade drives the generation of autophagy‐derived exosomes, which mediate pathological cartilage calcification in OA. Chondrocyte‐targeted BAP31
Zhi‐hua Xu +13 more
wiley +1 more source
Journal of VirologyAdeno-associated viruses (AAV) are non-enveloped parvoviruses widely utilized for therapeutic gene delivery. The membrane-associated accessory protein (MAAP), a key viral protein, is critical for cellular egress of both infectious and recombinant AAV ...
Robert M. Fusco +5 more
doaj +1 more source
Molecular Therapy: Methods & Clinical Development, 2018 Pre-existing neutralizing antibody (NAb) against adeno-associated virus (AAV) commonly found in primates is a major host barrier that can severely compromise in vivo gene transfer by AAV vectors.
Dan Wang +14 more
doaj +1 more source
The effects of anti-sense interleukin-5 gene transferred by recombinant adeno-associated virus in allergic rats [PDF]
, 2008 The accumulation and infiltration of eosinophils in airways is one of the most important characteristics of asthma, and is mediated partly by secretion of IL-5 from Th2 lymphocytes.
Weining Xiong +6 more
core
, 2022 Adeno-associated virus (AAV)-mediated gene transfer is an efficient method of gene over-expression in the vestibular end organs. However, AAV has limited usefulness for delivering a large gene, or multiple genes, due to its small packaging capacity ...
Jing-Ying Guo (743732) +9 more
core +1 more source
Innate Immunocompetent hiPSC‐Derived Neurospheroids Capture Early CNS Responses to rAAV
Advanced Science, EarlyView.Knowledge of human CNS immune responses to AAV‐based gene therapies remains limited due to the lack of immune‐competent human models. Here, a hiPSC‐derived 3D neuroimmune platform integrating neurospheroids and microglia is established using stirred‐tank bioreactors.
Catarina M. Gomes +14 more
wiley +1 more source
Divergent engagements between adeno-associated viruses with their cellular receptor AAVR
Nature Communications, 2019 Multiple adeno-associated viruses (AAV) use the same receptor (AAVR), but the binding mode is not clear. Here, the authors determine the structures of the AAV1-AAVR and AAV5-AAVR complexes, identify residues necessary for virus entry and compare the ...
Ran Zhang +15 more
doaj +1 more source
Nab Escaping AAV Mutants Isolated from Mouse Muscles
Bio-Protocol, 2018 Neutralizing antibodies (Nabs) are a major challenge in clinical trials of adeno-associated virus (AAV) vector gene therapy, because Nabs are able to inhibit AAV transduction in patients.
Zheng Chai, R. Samulski, Chengwen Li
doaj +1 more source
Advanced Science, EarlyView.ABSTRACT The LRRK2 G2019S mutation, a Parkinson's disease–linked variant, has been associated with depression‐like phenotypes, but mechanisms remain unclear. We chart age‐dependent behavioral changes and astrocyte reactivity in G2019S mice and define a pathway connecting LRRK2 to TFEB/GDNF signaling, cellular energetics, and inflammation.
Longping Yao +5 more
wiley +1 more source
Mutation-independent treatment of autosomal dominant Retinitis Pigmentosa (adRP) [PDF]
, 2010 Viral-mediated gene therapy holds great promise for the treatment of severe inherited retinal diseases, such as Retintitis Pigmentosa (RP), which is caused by mutations in genes preferentially expressed in photoreceptor cells. The availability of vectors
Mussolino, Claudio
core +1 more source