Results 31 to 40 of about 4,965,597 (147)
Molecular Therapy: Methods & Clinical DevelopmentGene therapies using recombinant adeno-associated virus (AAV) vectors have demonstrated considerable clinical success in the treatment of genetic disorders.
Suzanne Scott +11 more
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BiomoleculesAdeno-associated virus (AAV) vectors have emerged as the leading platform for retinal gene therapy due to their favorable safety profile, low immunogenicity, and ability to mediate long-term transgene expression within the immune-privileged ocular ...
Jiayu Huang +3 more
doaj +1 more source
Rationally Engineered AAV Capsids Improve Transduction and Volumetric Spread in the CNS [PDF]
Molecular Therapy - Nucleic Acids, 2017 Adeno-associated virus (AAV) is the most common vector for clinical gene therapy of the CNS. This popularity originates from a high safety record and the longevity of transgene expression in neurons. Nevertheless, clinical efficacy for CNS indications is lacking, and one reason for this is the relatively limited spread and transduction efficacy in ...
Department of Translational Science and Molecular Medicine, Michigan State University, Grand Rapids, MI 49503, USA ( host institution ) +10 more
openaire +4 more sources
Molecular Therapy: Methods & Clinical DevelopmentAdeno-associated virus (AAV) is the most widely used vector for in vivo gene transfer. A major limitation of capsid engineering is the incomplete understanding of the consequences of multiple amino acid variations on AAV capsid stability resulting in ...
Tiziana La Bella +8 more
doaj +1 more source
Generation of enhanced gene delivery vectors by directed evolution of adeno-associated virus [PDF]
, 2006 Despite promising advance in the development of viral vectors based on AAV for human gene therapy, several major hurdles for a more general use remain.
Endell, Jan
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Cellular phosphoinositides and the maturation of bluetongue virus, a non-enveloped capsid virus. [PDF]
, 2013 BACKGROUND: Bluetongue virus (BTV), a member of Orbivirus genus in the Reoviridae family is a double capsid virus enclosing a genome of 10 double-stranded RNA segments. A non-structural protein of BTV, NS3, which is associated with cellular membranes and
Roy, Polly +3 more
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Asian Journal of Pharmaceutical Sciences, 2023 Virus-capsid mimicking mucus-permeable nanoparticles are promising oral insulin carriers which surmount intestinal mucus barrier. However, the impact of different virus-capsid mimicking structure remains unexplored.
Zhixiang Cui +7 more
doaj +1 more source
Controlling AAV Tropism in the Nervous System with Natural and Engineered Capsids [PDF]
, 2016 More than one hundred naturally occurring variants of adeno-associated virus (AAV) have been identified, and this library has been further expanded by an array of techniques for modification of the viral capsid. AAV capsid variants possess unique antigenic profiles and demonstrate distinct cellular tropisms driven by differences in receptor binding ...
Michael J, Castle +3 more
openaire +2 more sources
A Protein-Capsid-Based System for Cell Delivery of Selenocysteine
, 2018 Selenocysteine (Sec) has received a lot of attention as a potential anticancer drug. However, its broad cytotoxicity limits its therapeutic usefulness. Thus, Sec is an attractive candidate for targeted drug delivery.
Kenneth J. Woycechowsky (2054407) +4 more
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Bacteriophages and their structural organisation [PDF]
, 2012 Viruses are extremely small infectious particles that are not visible in a light microscope, and are able to pass through fine porcelain filters. They exist in a huge variety of forms and infect practically all living systems: animals, plants ...
E.V. Orlova, Orlova, Elena
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