Results 71 to 80 of about 8,933,868 (252)
Adeno-associated virus for cystic fibrosis gene therapy
Brazilian Journal of Medical and Biological Research, 2011 Gene therapy is an alternative treatment for genetic lung disease, especially monogenic disorders such as cystic fibrosis. Cystic fibrosis is a severe autosomal recessive disease affecting one in 2500 live births in the white population, caused by ...
S.V. Martini, P.R.M. Rocco, M.M. Morales
doaj +3 more sources
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
Viral Hybrid Vectors for Somatic Integration - Are They the Better Solution? [PDF]
, 2009 The turbulent history of clinical trials in viral gene therapy has taught us important lessons about vector design and safety issues. Much effort was spent on analyzing genotoxicity after somatic integration of therapeutic DNA into the host genome. Based
Anja Ehrhardt +6 more
core +1 more source
Mucopolysaccharidosis type IIIB: a current review and exploration of the AAV therapy landscape
Neural Regeneration ResearchMucopolysaccharidoses type IIIB is a rare genetic disorder caused by mutations in the gene that encodes for N-acetyl-alpha-glucosaminidase. This results in the aggregation of heparan sulfate polysaccharides within cell lysosomes that leads to progressive
Courtney J Rouse +2 more
doaj +1 more source
Potential for cellular stress response to hepatic factor VIII expression from AAV vector
Molecular Therapy: Methods & Clinical Development, 2016 Hemophilia A and B are coagulation disorders resulting from the loss of functional coagulation factor VIII (FVIII) or factor IX proteins, respectively.
Irene Zolotukhin +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
ApoE gene therapy: an overview and update [PDF]
, 2005 Atherosclerosis remains the leading cause of death in industrialized societies. Apolipoprotein E (ApoE) is an attractive candidate to treat hypercholesterolemia and coronary heart disease, as it is a circulating protein with pleiotropic ...
Owen, J.S.
core
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
TOLLIP Inhibits Psoriasis Progression via Suppressing PKM2‐Mediated Glycolysis in Keratinocytes
Advanced Science, EarlyView.In this study, we identify TOLLIP as a critical regulator of psoriasis pathogenesis through its modulation of glycolytic metabolism. Our findings establish the TOLLIP‐PKM2‐glycolysis axis as a key mechanism linking metabolic reprogramming to psoriasis pathogenesis, and propose TOLLIP as a promising therapeutic target.
Xiuhuan Jiang +11 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