Results 81 to 90 of about 144,727 (384)
Preclinical Assessment of Immune Responses to AAV (adeno-associated virus) Vectors
Frontiers in Immunology, 2014 Transitioning to human trials from preclinical models resulted in the emergence of inhibitory AAV vector immune responses which has become a hurdle for sustained correction.
Etiena eBasner-Tschakarjan +2 more
doaj +1 more source
GM1 Ganglioside Modifies α-Synuclein Toxicity and is Neuroprotective in a Rat α-Synuclein Model of Parkinson\u27s Disease. [PDF]
, 2019 While GM1 may interact with α-synuclein in vitro to inhibit aggregation, the ability of GM1 to protect against α-synuclein toxicity in vivo has not been investigated.
Aras, Radha +5 more
core +1 more source
Liver-specific knockout of arginase-1 leads to a profound phenotype similar to inducible whole body arginase-1 deficiency [PDF]
, 2016 Arginase-1 (Arg1) converts arginine to urea and ornithine in the distal step of the urea cycle in liver. We previously generated a tamoxifen-inducible Arg1 deficient mouse model (Arg1-Cre) that disrupts Arg1 expression throughout the whole body and leads
Al-Dirbashi, Osama Y. +5 more
core +1 more source
Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain
Nature Biotechnology, 2015 Recombinant adeno-associated viruses (rAAVs) are commonly used vehicles for in vivo gene transfer. However, the tropism repertoire of naturally occurring AAVs is limited, prompting a search for novel AAV capsids with desired characteristics.
Benjamin E. Deverman +10 more
semanticscholar +1 more source
Annals of the Rheumatic Diseases, 2019 Background: ANCA associated vasculitis is an autoimmune disease classified by clinical phenotype and serology. A genetic predisposition has been determined which differs between PR3-ANCA and MPO-ANCA patients. Experimental models have confirmed the pathogenicity of ANCA for certain aspects of pathology and have defined a role for alternative ...
openaire +2 more sources
Advanced Materials, EarlyView.This study develops a multifunctional CRISPR‐dCas9‐based OMV platform termed OMV‐C9I12, which facilitates the coexpression of CXCL9 and IL‐12 within tumor cells. This platform enhances T cell recruitment and activation, synergizes with anti‐PD‐1/PD‐L1 immunotherapy, and amplifies antitumor T cell immunity.
Hongjin Wang +22 more
wiley +1 more source
Viral Vector-based Improvement of Optic Nerve Regeneration: Characterization of Individual Axons\u27 Growth Patterns and Synaptogenesis in a Visual Target [PDF]
, 2015 Lack of axon growth ability in the central nervous system poses a major barrier to achieving functional connectivity after injury. Thus, a non-transgenic regenerative approach to reinnervating targets has important implications in clinical and research ...
Blackmore, Murray G. +3 more
core +1 more source
Advances and Strategies in Enhancing mRNA Cancer Vaccines
Advanced Materials, EarlyView.Messenger RNA (mRNA) vaccines offer a powerful approach for cancer immunotherapy, but their clinical impact remains limited by delivery challenges and suboptimal immune activation. This review discusses key biological barriers and design strategies—including structural optimization, immunomodulation, organ targeting delivery, and advanced nanocarriers ...
Miao Zhang +4 more
wiley +1 more source
AAV vectors for FRET-based analysis of protein-protein interactions in photoreceptor outer segments
Frontiers in Neuroscience, 2016 Fluorescence resonance energy transfer (FRET) is a powerful method for the detection and quantification of stationary and dynamic protein-protein interactions.
Elvir Becirovic +19 more
doaj +1 more source
Genetic Engineering Methods in Primary T Cells
Advanced Therapeutics, EarlyView.Primary T cells can be engineered to confer them with novel therapeutic functions, allowing them to treat a variety of conditions. Genetic engineering can be either stable or transient, aiming to either express or inhibit a target gene. This review discusses the various genetic engineering tools available as well as their characteristics and ...
Anthony Youssef, Hui‐Shan Li
wiley +1 more source