Results 21 to 30 of about 31,732 (250)
Molecular Therapy: Methods & Clinical Development, 2019 Parvoviruses are highly attractive templates for the engineering of safe, efficient, and specific gene therapy vectors, as best exemplified by adeno-associated virus (AAV).
Julia Fakhiri +14 more
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Adipose Tissue: An Emerging Target for Adeno-associated Viral Vectors
Molecular Therapy: Methods & Clinical Development, 2020 Adipose tissue is one of the largest organs, playing important roles in physiology and pathologies of multiple diseases. However, research related to adeno-associated virus (AAV) targeting adipose tissue has been left far behind studies carried out in ...
Rhiannon Bates, Wei Huang, Lei Cao
doaj +1 more source
Non-canonical amino acid incorporation into AAV5 capsid enhances lung transduction in mice
Molecular Therapy: Methods & Clinical Development, 2023 Gene therapy using recombinant adeno-associated virus (rAAV) relies on safe, efficient, and precise in vivo gene delivery that is largely dependent on the AAV capsid.
Hao Chang +8 more
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Molecular Therapy: Nucleic Acids, 2014 Recently, we described a peptide-modified AAV2 vector (AAV-GMN) containing a capsid-displayed peptide that directs in vivo brain vascular targeting and transduction when delivered intravenously.
James C Geoghegan +5 more
doaj +1 more source
Adeno-Associated Virus (AAV) Gene Delivery: Dissecting Molecular Interactions upon Cell Entry
Viruses, 2021 Human gene therapy has advanced from twentieth-century conception to twenty-first-century reality. The recombinant Adeno-Associated Virus (rAAV) is a major gene therapy vector. Research continues to improve rAAV safety and efficacy using a variety of AAV
Edward E. Large +4 more
doaj +1 more source
The Cold Case Files of rAAV Capsid Influence on Transduction: New Leads
VirusesAdeno-associated virus (AAV) is a prevalent vector in viral gene therapy. Given its importance, significant efforts focus on engineering the capsid residues on the exterior surface to increase cell/tissue-specific binding to cellular receptors or to ...
Sara K. Powell
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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
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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