Results 41 to 50 of about 31,732 (250)

Codon-Optimization of Wild-Type Adeno-Associated Virus Capsid Sequences Enhances DNA Family Shuffling while Conserving Functionality

Molecular Therapy: Methods & Clinical Development, 2019
Adeno-associated virus (AAV) vectors have become one of the most widely used gene transfer tools in human gene therapy. Considerable effort is currently being focused on AAV capsid engineering strategies with the aim of developing novel variants with ...
Marti Cabanes-Creus, Samantha L. Ginn, Anais K. Amaya, Sophia H.Y. Liao, Adrian Westhaus, Claus V. Hallwirth, Patrick Wilmott, Jason Ward, Kimberley L. Dilworth, Giorgia Santilli, Arkadiusz Rybicki, Hiroyuki Nakai, Adrian J. Thrasher, Adrian C. Filip, Ian E. Alexander, Leszek Lisowski   +15 more
doaj   +1 more source

Insight into the mechanisms of enhanced retinal transduction by the engineered AAV2 capsid variant ‐7m8 [PDF]

Biotechnology and Bioengineering, 2016
ABSTRACTRecently, we described a modified AAV2 vector—AAV2‐7m8—having a capsid‐displayed peptide insertion of 10 amino acids with enhanced retinal transduction properties. The insertion of the peptide referred to as 7m8 is responsible for high‐level gene delivery into deep layers of the retina when virus is delivered into the eye's vitreous.
Khabou, Hanen, Desrosiers, Mélissa, Winckler, Céline, Fouquet, Stéphane, Auregan, Gwenaëlle, Bemelmans, Alexis-Pierre, Sahel, José-Alain, Dalkara, Deniz   +7 more
openaire   +2 more sources

Inhibition of VEGF‐Induced Angiogenesis and Vascular Leakage by Bicistronic Co‐Expression of Aflibercept and COMP‐Ang1

Advanced Healthcare Materials, EarlyView.
A bicistronic rAAV8 vector encoding Aflibercept (Afb) and COMP‐cAng1 simultaneously coordinates VEGF clearance and Tie2‐mediated vessel maturation. In a 3D angiogenesis‐on‐a‐chip model, rAAV8‐Afb/cAng1 effectively reverses pathological barrier breakdown under clinical disease‐mimicking challenges.
Bong‐Kyu Kim, Jaehong Min, Sang‐Hee Park, Subeen Shin, Sangbin Yu, Jeong‐Eun Park, Sujin Kim, Wooju Park, Jihoon Ko, Sunjong Kwon   +9 more
wiley   +1 more source

Resolving Heterogeneity of Targeted Lipid Nanoparticles Through Solution‐Based Biophysical Analyses

Advanced Materials, EarlyView.
AF4‐UV‐DLS‐MALS‐SAXS resolves previously inaccessible targeted lipid nanoparticle (tLNP) subpopulations that differ in size, shape, and composition. Correlation of subpopulation‐resolved biophysical properties with in vivo RNA delivery reveals that targeted placental transfection is associated with distinct tLNP subpopulations rather than ensemble ...
Hannah C. Geisler, Hannah C. Safford, Ajay S. Thatte, Marshall S. Padilla, Elisa Battistini, Hannah M. Yamagata, Violet M. Ullman, Alex Chan, Benjamin E. Nachod, Anushka Agrawal, Maxwell B. Watkins, Jesse B. Hopkins, Andrew Tsourkas, Kushol Gupta, Michael J. Mitchell   +14 more
wiley   +1 more source

Adeno‐associated virus serotype 2 capsid variants for improved liver‐directed gene therapy

Hepatology, EarlyView., 2022
Abstract Background and Aims Current liver‐directed gene therapies look for adeno‐associated virus (AAV) vectors with improved efficacy. With this background, capsid engineering is explored. Whereas shuffled capsid library screenings have resulted in potent liver targeting variants with one first vector in human clinical trials, modifying natural ...
Nadja Meumann, Marti Cabanes‐Creus, Moritz Ertelt, Renina Gale Navarro, Julie Lucifora, Qinggong Yuan, Karin Nien‐Huber, Ahmed Abdelrahman, Xuan‐Khang Vu, Liang Zhang, Ann‐Christin Franke, Christian Schmithals, Albrecht Piiper, Annabelle Vogt, Maria Gonzalez‐Carmona, Jochen T. Frueh, Evelyn Ullrich, Philip Meuleman, Steven R. Talbot, Margarete Odenthal, Michael Ott, Erhard Seifried, Clara T. Schoeder, Joachim Schwäble, Leszek Lisowski, Hildegard Büning   +25 more
wiley   +1 more source

Engineering a highly durable adeno-associated virus receptor for analytical applications

Molecular Therapy: Methods & Clinical Development, 2023
Adeno-associated virus (AAV) is a major viral vector used in gene therapy. There are multiple AAV serotypes, and many engineered AAV serotypes are developed to alter their tissue tropisms with capsid modification.
Kouhei Yoshida, Yuji Tsunekawa, Kento Kurihara, Kazuya Watanabe, Yuriko Makino-Manabe, Mikako Wada, Toru Tanaka, Teruhiko Ide, Takashi Okada   +8 more
doaj   +1 more source

Programmable Functional Silicification of DNA Origami Nanostructures

Advanced Materials, EarlyView.
This work introduces additional functionality into silica‐coated DNA origami nanostructures using non‐standard silica precursors. A fluorescent precursor enables enhanced intracellular tracking, while a disulfide‐containing reagent yields redox‐responsive, degradable silica coatings.
Anna V. Baptist, Lasse Guericke, Philipp Mauker, Oliver Thorn‐Seshold, Amelie Heuer‐Jungemann   +4 more
wiley   +1 more source

Modified AAV5 capsid for improved brain biodistribution following direct injection in preclinical models

Molecular Therapy: Methods & Clinical Development
Gene therapies based on adeno-associated virus vectors hold strong potential for the treatment of central nervous system disorders. However, systemic delivery is limited by the blood-brain barrier, off-target effects, immune responses, and vector loss ...
Sebastian N. Kieper, Elisabeth A. Spronck, Meriem Bourajjaj, Roberto D.V.S. Morais, M. Leontien van der Bent, Seyda Acar Broekmans, Charlot van Rooijen, Rhodé van Westen, Giorgia Squeri, Giso Brasser, Morgane Wartel, Stijn J.H. Waaijer, C. Warner Hoornenborg, Tycho M. Hoogland, Richard Porter, Anggakusuma   +15 more
doaj   +1 more source

Deep Contrastive Learning for High‐Throughput Prediction of Drug Resistance Mutations from Sequences

Advanced Science, EarlyView.
This study presents DeepMutDTA, a deep learning framework aimed at predicting mutation‐induced changes in protein‐drug interactions and prioritizing variants potentially linked to drug resistance. Trained on large‐scale data, it incorporates SimSiam‐MuTF, a label‐aware contrastive fine‐tuning strategy that encourages separation between WT and MT ...
Xiaowen Hu, Pan Zhang, Shangqian Wu, Hao Sun, Minwei Li, Sophia Tsoka, Zizhang Sheng, Lei Deng   +7 more
wiley   +1 more source

αFAP-specific nanobodies mediate a highly precise retargeting of modified AAV2 capsids thereby enabling specific transduction of tumor tissues

Molecular Therapy: Methods & Clinical Development
Due to the refractiveness of tumor tissues to adeno-associated virus (AAV) transduction, AAV vectors are poorly explored for cancer therapy delivery.
Olaniyi Olarewaju, Franziska Held, Pamela Curtis, Cynthia Hess Kenny, Udo Maier, Tadas Panavas, Francois du Plessis   +6 more
doaj   +1 more source