Results 1 to 10 of about 31,732 (250)
Modifying immune responses to adeno-associated virus vectors by capsid engineering [PDF]
Molecular Therapy: Methods & Clinical Development, 2023 De novo immune responses are considered major challenges in gene therapy. With the aim to lower innate immune responses directly in cells targeted by adeno-associated virus (AAV) vectors, we equipped the vector capsid with a peptide known to interfere ...
Martin Bentler +13 more
doaj +7 more sources
Strategies of AAV capsid engineering for targeted delivery to brain, muscle, and retina [PDF]
Frontiers in Molecular BiosciencesAdeno-associated virus (AAV) vectors are widely used for in vivo gene delivery to the central nervous system (CNS), muscle, and retina, but many clinically used capsids show limited potency in human tissues, necessitating high systemic doses that ...
Xinyuan Xu
doaj +7 more sources
Systematic multi-trait AAV capsid engineering for efficient gene delivery [PDF]
Nature Communications, 2022 Broadening gene therapy applications requires manufacturable vectors that efficiently transduce target cells in humans and preclinical models. Conventional selections of adeno-associated virus (AAV) capsid libraries are inefficient at searching the vast ...
Fatma-Elzahraa Eid +15 more
doaj +6 more sources
CapBuild: a cloud-native tool for adeno-associated virus capsid engineering. [PDF]
Nucleic Acids ResAbstract Adeno-associated virus (AAV) capsid engineering is essential for advancing gene therapy but remains limited by structural complexity and computational constraints. To address these challenges, we developed CapBuild, a cloud-native web server that streamlines AAV capsid prediction, assembly and engineering.
Klein AH +6 more
europepmc +4 more sources
Cell Reports, 2018 Summary: The adeno-associated virus (AAV) vector is a preferred delivery platform for in vivo gene therapy. Natural and engineered variations of the AAV capsid affect a plurality of phenotypes relevant to gene therapy, including vector production and ...
Anna C. Maurer +5 more
doaj +3 more sources
Advances in endothelial cell targeting by AAV vectors [PDF]
Acta Biochimica PolonicaAdeno-associated virus (AAV) vectors have become a cornerstone of in vivo gene delivery. However, although the endothelium is the first cellular interface encountered after systemic delivery, native AAV serotypes exhibit poor endothelial transduction ...
Milena Cichon +3 more
doaj +2 more sources
Overcoming Immunological Challenges Limiting Capsid-Mediated Gene Therapy With Machine Learning
Frontiers in Immunology, 2021 A key hurdle to making adeno-associated virus (AAV) capsid mediated gene therapy broadly beneficial to all patients is overcoming pre-existing and therapy-induced immune responses to these vectors.
Anna Z. Wec +6 more
doaj +1 more source
Completion of the AAV Structural Atlas: Serotype Capsid Structures Reveals Clade-Specific Features
Viruses, 2021 The capsid structures of most Adeno-associated virus (AAV) serotypes, already assigned to an antigenic clade, have been previously determined. This study reports the remaining capsid structures of AAV7, AAV11, AAV12, and AAV13 determined by cryo-electron
Mario Mietzsch +6 more
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AAV Engineering for Improving Tropism to the Central Nervous System
Biology, 2023 Adeno-associated virus (AAV) is a non-pathogenic virus that mainly infects primates with the help of adenoviruses. AAV is being widely used as a delivery vector for in vivo gene therapy, as evidenced by five currently approved drugs and more than 255 ...
Muhammad S. Ghauri, Li Ou
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Chemical modification of AAV9 capsid with N-ethyl maleimide alters vector tissue tropism
Scientific Reports, 2023 Although more adeno-associated virus AAV-based drugs enter the clinic, vector tissue tropism remains an unresolved challenge that limits its full potential despite that the tissue tropism of naturally occurring AAV serotypes can be altered by genetic ...
Patrick L. Mulcrone +6 more
doaj +1 more source