Results 11 to 20 of about 65,616 (261)
Humoral immune response to AAV [PDF]
Frontiers in Immunology, 2013 Adeno-associated virus (AAV) is a member of the family parvoviridae that has been widely used as a vector for gene therapy because of its safety profile, its ability to transduce both dividing and non-dividing cells, and its low immunogenicity.
Roberto eCalcedo, James M Wilson
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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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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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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
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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
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Adeno-Associated Virus (AAV) - Based Gene Therapies for Retinal Diseases: Where are We?
The Application of Clinical Genetics, 2023 Divya Ail, Hugo Malki, Emilia A Zin, Deniz Dalkara Sorbonne Université, INSERM, CNRS, Department of Therapeutics, Institut de la Vision, Paris, 75012, FranceCorrespondence: Divya Ail, Department of Therapeutics, Institut de la Vision, 17 Rue Moreau ...
Ail D, Malki H, Zin EA, Dalkara D
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