Results 71 to 80 of about 89,643 (298)
Molecular Therapy: Methods & Clinical Development, 2020 Delivery of therapeutic transgenes with adeno-associated viral (AAV) vectors for treatment of myopathies has yielded encouraging results in animal models and early clinical studies.
Jennifer B. Kwon +7 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
Cross-Packaging and Capsid Mosaic Formation in Multiplexed AAV Libraries
Molecular Therapy: Methods & Clinical Development, 2020 Generation and screening of libraries of adeno-associated virus (AAV) variants have emerged as a powerful method for identifying novel capsids for gene therapy applications.
Pauline F. Schmit +13 more
doaj +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
Relative expression in cultured RPE with AAV-OTX2S versus AAV-GFP control transduced cells.
, 2016 Relative expression in cultured RPE with AAV-OTX2S versus AAV-GFP control transduced cells.
José-Alain Sahel (19376) +14 more
core +1 more source
Advanced Science, EarlyView.This study develops an MMP‐9‐responsive protein release system that transforms resident cells into drug depots through in situ genetic engineering. Anchored to the membrane, the therapeutic protein TIMP1 is selectively released upon MMP‐9 cleavage.
Yuan Zhang +11 more
wiley +1 more source
Advanced Science, EarlyView.Using single‐cell transcriptomic and epigenomic profiling, researchers mapped microglial dysfunction across the lifespan of db/db mice. The authors uncovered a nonlinear microglial remodeling featuring early inflammatory priming, a mid‐stage transcriptional inflection, and late dysfunction.
Jizhou Zhang +6 more
wiley +1 more source
Molecular Therapy, 2003 One of the many hurdles that face gene therapists is the requirement to optimize gene delivery systems in such a way that both efficient and selective transgene production is achieved in vivo within a chosen cell or tissue. Exploiting native vector tropism can sometimes achieve this goal irrespective of additional modifications. For example, adenoviral
openaire +2 more sources
Advanced Science, EarlyView.FES‐derived MGE spheroids exhibit progenitor‐stage alterations in developmental trajectory and hypoxia‐responsive transcriptional programs, followed by functional disruption. Gestational hypoxia recapitulates impaired progenitor proliferation, shortened cell‐cycle progression, interneuron developmental abnormalities, and schizophrenia‐like behaviors in
Peiyan Ni +17 more
wiley +1 more source
Molecules, 2019 Adeno-Associated Viruses (AAV) are widely used gene-therapy vectors for both clinical applications and laboratory investigations. The titering of different AAV preparations is important for quality control purposes, as well as in comparative studies ...
Mengtian Cui +7 more
doaj +1 more source