Results 11 to 20 of about 89,643 (298)
Association of the AAV-PRO questionnaire with established outcome measures in AAV [PDF]
Rheumatology, 2023 Abstract Objectives The ANCA-associated vasculitis (AAV) patient-reported outcome (AAV-PRO) questionnaire was developed to capture the impact of AAV and its treatment. We investigated the association of specific AAV-PRO domains with disease activity and extent, damage, depression, health-related ...
Annika Maunz +5 more
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AAV genome modification for efficient AAV production
Heliyon, 2023 The adeno-associated virus (AAV) is one of the most potent vectors in gene therapy. The experimental profile of this vector shows its efficiency and accepted safety, which explains its increased usage by scientists for the research and treatment of a wide range of diseases.
Walaa Asaad +6 more
openaire +3 more sources
AAV-8 and AAV-9 Vectors Cooperate with Serum Proteins Differently Than AAV-1 and AAV-6 [PDF]
Molecular Therapy - Methods & Clinical Development, 2018 Under intravenous delivery, recombinant adeno-associated vectors (rAAVs) interact with blood-borne components in ways that can critically alter their therapeutic efficiencies. We have previously shown that interaction with human galectin 3 binding protein dramatically reduces rAAV-6 efficacy, whereas binding of mouse C-reactive protein improves rAAV-1 ...
Denard, Jérôme +9 more
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Progress in the use of adeno-associated viral vectors for gene therapy [PDF]
, 2004 The development of safe and efficient gene transfer vectors is crucial for the success of gene therapy trials. A viral vector system promising to meet these requirements is based on the apathogenic adeno-associated virus (AAV-2), a member of the ...
Markus Braun-Falco +7 more
core +1 more source
AAV-CRISPR gene editing is negated by pre-existing immunity to Cas9
, 2023 Adeno-associated viral (AAV) vectors are a leading candidate for the delivery of CRISPR-Cas9 for therapeutic genome editing in vivo. However, AAV-based delivery involves persistent expression of the Cas9 nuclease, a bacterial protein.
Mark R. Tanner (8928290) +10 more
core +1 more source
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
doaj +1 more source
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
doaj +1 more source
Molecular Therapy, 2015 The paper recently published in Nature by Gardner et al.1 marks yet another milestone in the development of adeno-associated virus (AAV) as an effective gene delivery vehicle, and is an appropriate way to highlight the fiftieth anniversary of the discovery of AAV. Using simple intramuscular injections in primates, Gardner et al.
Muzyczka, Nicholas, Berns, Kenneth I
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T Cell-Mediated Immune Responses to AAV and AAV Vectors [PDF]
Frontiers in Immunology, 2021 Adeno-associated virus (AAV)-mediated gene transfer has benefited patients with inherited diseases, such as hemophilia B, by achieving long-term expression of the therapeutic transgene. Nevertheless, challenges remain due to rejection of AAV-transduced cells, which in some, but not all, patients can be prevented by immunosuppression. It is assumed that
openaire +3 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
doaj +1 more source