Results 21 to 30 of about 79,958 (292)
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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Frontiers in Molecular Biosciences, 2023 Huntington’s disease (HD) is a multi-tissue failure disorder for which there is no cure. We have previously shown an effective therapeutic approach limited mainly to the central nervous system, based on a synthetic zinc finger (ZF) transcription ...
Michal Mielcarek +3 more
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Wild type AAV, recombinant AAV, and Adenovirus super infection impact on AAV vector mobilization [PDF]
, 2020 Abstract Recombinant 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-
Song, Liujiang +2 more
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Blood, 2021 In this issue of Blood, Konkle et al report that 7 of 8 participants in a phase 1/2 trial of adeno-associated virus (AAV) vector (BAX335) for factor IX (FIX)-Padua gene transfer in patients with hemophilia B did not maintain expression despite steroid intervention, which the authors hypothesize is a result of the innate immune stimulatory effect of ...
openaire +2 more sources
Molecular Therapy: Methods & Clinical DevelopmentDue to the refractiveness of tumor tissues to adeno-associated virus (AAV) transduction, AAV vectors are poorly explored for cancer therapy delivery.
Olaniyi Olarewaju +6 more
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A novel dual mechanism-of-action bispecific PD-1-IL-2v armed by a “βγ-only” interleukin-2 variant
Frontiers in ImmunologyIntroductionInterleukin-2 (IL-2) is one of the first cytokines to be discovered as an immune agonist for cancer immunotherapy. Biased IL-2 variants had been discovered to eliminate Treg activation or enhance the tumor specific T cell cytotoxicity ...
Yongji Jiang +7 more
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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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PEP-TP56 enhances AAV transduction in a serotype-independent manner
HeliyonAdeno-associated viruses (AAVs) have emerged as the most prominent gene therapy vectors. However, the need for high AAV doses to achieve clinical efficacy remains a key challenge to AAV utility in the clinic.
Olaniyi Olarewaju +4 more
doaj +1 more source
AAV Vectors for the Nucleolus [PDF]
Molecular Therapy, 2012 During the replication of wild-type adeno-associated virus (AAV), viral capsids are assembled in the nucleolus,1 and the capsid proteins interact with the nucleolar proteins nucleolin (NCL) and nucleophosmin (NPM1), possibly promoting their transport from the cytoplasm into the nucleolus.2,3 In the case of replication-incompetent AAV vectors, the ...
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AAV-mediated overexpression of Cytl1 induces CF.
, 2016 Control virus or AAV-Cytl1 (5 × 1010 viral genome) was injected into the tail vein of WT mice, and the phenotype of the heart was examined after 8 wks. (A) Picrosirius staining of heart cross-sections from WT mice injected with control virus or AAV-Cytl.
Roger J. Hajjar (142214) +8 more
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