Familial vasculitides: granulomatosis with polyangitis and microscopic polyangitis in two brothers with differing anti-neutrophil cytoplasm antibody specificity [PDF]
, 2016 Anti-neutrophil cytoplasm antibody (ANCA)-associated vasculitis (AAV) is a group of rare autoimmune diseases. Although the aetiology of AAV is uncertain, it is likely that genetic and environmental factors contribute.
Cairns, T, Prendecki, M, Pusey, CD
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Promising AAV.U7snRNAs vectors targeting DMPK improve DM1 hallmarks in patient-derived cell lines
Frontiers in Cell and Developmental Biology, 2023 Myotonic dystrophy type 1 (DM1) is the most common form of muscular dystrophy in adults and affects mainly the skeletal muscle, heart, and brain. DM1 is caused by a CTG repeat expansion in the 3′UTR region of the DMPK gene that sequesters muscleblind ...
Camila F. Almeida +16 more
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Plectin-1 targeted AAV vector for the molecular imaging of pancreatic cancer
Frontiers in Oncology, 2013 Pancreatic ductal adenocarcinoma (PDAC) is highly malignant disease that is the 4th leading cause of cancer-related death in the US. Gene therapy using AAV vectors to selectively deliver genes to PDAC cells is an attractive treatment option for ...
Prasad R. Konkalmatt +6 more
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To transduce a zebra finch: interrogating behavioral mechanisms in a model system for speech. [PDF]
, 2017 The ability to alter neuronal gene expression, either to affect levels of endogenous molecules or to express exogenous ones, is a powerful tool for linking brain and behavior. Scientists continue to finesse genetic manipulation in mice.
Heston, Jonathan B, White, Stephanie A
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Characterization of Brevibacillus laterosporus Cas9 (BlatCas9) for Mammalian Genome Editing
Frontiers in Cell and Developmental Biology, 2020 Compact CRISPR/Cas9 systems that can be delivered by AAV for in vivo genome editing hold great promise for clinical applications. Brevibacillus laterosporus Cas9 (BlatCas9) is a compact Cas9 nuclease that has been identified for plant genome editing ...
Ning Gao +8 more
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The HLA class I immunopeptidomes of AAV capsid proteins
Frontiers in Immunology, 2023 IntroductionCellular immune responses against AAV vector capsid represent an obstacle for successful gene therapy. Previous studies have used overlapping peptides spanning the entire capsid sequence to identify T cell epitopes recognized by AAV-specific ...
Carlos A. Brito-Sierra +3 more
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Perinatal Gene Transfer to the Liver [PDF]
, 2011 The liver acts as a host to many functions hence raising the possibility that any one may be compromised by a single gene defect. Inherited or de novo mutations in these genes may result in relatively mild diseases or be so devastating that death within
Buckley, SM +6 more
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LDLR-Gene therapy for familial hypercholesterolaemia: Problems, progress, and perspectives [PDF]
, 2010 Coronary artery diseases (CAD) inflict a heavy economical and social burden on most populations and contribute significantly to their morbidity and mortality rates.
Al-Allaf, FA +5 more
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Wild type AAV, recombinant AAV, and Adenovirus super infection impact on AAV vector mobilization [PDF]
, 2020 AbstractRecombinant Adeno-associated viral vector (rAAV) mobilization is a largely theoretical process in which intact AAV vectors spread or “mobilize” from transduced cells and infect additional cells within, or external, of the initial host. This process can be replication independent (vector alone), or replication-dependent (de novo rAAV production ...
Song, Liujiang +2 more
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Frontiers in Neuroanatomy, 2014 When nanoparticles/proteins are infused into the brain, they are often transported to distal sites in a manner that is dependent both on the characteristics of the infusate and the region targeted. We have previously shown that AAV is disseminated within
Ernesto A Salegio +12 more
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