Results 11 to 20 of about 79,958 (292)
Syngeneic AAV Pseudo-particles Potentiate Gene Transduction of AAV Vectors
Molecular Therapy - Methods & Clinical Development, 2017 Adeno-associated virus (AAV) vectors have emerged as a safe and efficient gene therapy platform. One complication is that a significant amount of empty particles have always been generated as impurities during AAV vector production. However, the effects of such particles on AAV vector performance remain unclear.
Qizhao Wang +10 more
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Frontiers in Genome Editing, 2023 Gene therapy is a fast developing field of medicine with hundreds of ongoing early-stage clinical trials and numerous preclinical studies. Genome editing (GE) now is an increasingly important technology for achieving stable therapeutic effect in gene ...
Alena Shakirova +4 more
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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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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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AAV-mediated rescue of Eps8 expression in vivo restores hair-cell function in a mouse model of recessive deafness [PDF]
, 2022 The transduction of acoustic information by hair cells depends upon mechanosensitive stereociliary bundles that project from their apical surface. Mutations or absence of the stereociliary protein EPS8 cause deafness in humans and mice, respectively ...
Yelena Dayn (16300601) +12 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 +5 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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Cell type specific gene delivery using chemically modified AAV vectors [PDF]
, 2022 In the last years the use of Adeno-Associated Viruses (AAVs) as carriers for gene transfer became increasingly popular for both research and clinical applications.
Barenghi, Alessandro
core
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