Results 41 to 50 of about 214,719 (303)

Silencing of UTX Mitigates Aging-Associated Cardiac Fibrosis via Blocking Cardiac Fibro blaststo-Myofibroblasts Trans-Differentiation

Anatolian Journal of Cardiology, 2023
Background: Cardiac fibrosis increases with age. Fibroblast activation plays an essential role in cardiac fibrosis. Histone modifications are involved in various chromatin-dependent processes.
Chao Li, Tiantian Lin, Delin Li, Daoyuan Si, Huan Sun, Sibao Yang, Zhongfan Zhang, Qian Zhang, Kaiyao Shi   +8 more
doaj   +1 more source

Onasemnogene Abeparvovec in Type I Spinal Muscular Atrophy: 24‐Month Follow‐Up From the Italian Registry

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Onasemnogene abeparvovec (OA) is an AAV9‐based gene therapy for spinal muscular atrophy type I (SMA I). Real‐world outcomes show increased response variability compared to clinical trials, and follow‐up data beyond 12–18 months are limited.
Marika Pane, Giorgia Coratti, Chiara Cutrì, Antonio Varone, Riccardo Masson, Adele D'Amico, Valeria Sansone, Sonia Messina, Federica Ricci, Chiara Ticci, Claudio Bruno, Caterina Agosto, Francesca Benedetti, Antonella Pini, Sabrina Siliquini, Massimiliano Filosto, Alberto Zambon, Ilaria Bitetti, Maria Rosaria Manna, Claudia Dosi, Riccardo Zanin, Stefano Parravicini, Roberto De Sanctis, Giulia Stanca, Michela Catteruccia, Michele Tosi, Irene Mizzoni, Emilio Albamonte, Valentina Franchino, Maria Sframeli, Ilaria Cavallina, Elena Procopio, Michele Sacchini, Simone Morando, Noemi Brolatti, Federica Trucco, Gaia Scarpini, Elena Briganti, Beatrice Berti, Concetta Palermo, Daniela Leone, Stefano C. Previtali, Eugenio Mercuri, the ITASMAC working group   +43 more
wiley   +1 more source

Adeno-Associated Virus (AAV) Versus Immune Response

Viruses, 2019
Decades ago, Friedmann and Roblin postulated several barriers to gene therapy, including tissue targeting, delivery across the blood⁻brain barrier (BBB), and host immune responses. These issues remain pertinent till today.
Joseph Rabinowitz, Ying Kai Chan, Richard Jude Samulski   +2 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
core  

Onasemnogene Abeparvovec in Patients With SMA: Interim Results of the RESTORE Registry in Japan

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective There are limited real‐world data regarding the safety and effectiveness of onasemnogene abeparvovec (OA; Zolgensma) infusion, a one‐time gene replacement therapy, for Japanese patients with spinal muscular atrophy (SMA). We aimed to improve understanding of the real‐world outcomes for OA in Japan.
Kayoko Saito, Kamal Benguerba, Ken Tsuchida, Kazushige Yazawa, Isao Tsumiyama, Hiromitsu Kayama, Sandra P. Reyna, Farid Khan, Richard S. Finkel   +8 more
wiley   +1 more source

Hydrogel Microtube Drug Carrier for Catheter‐Based Intravascular Therapy

Advanced Healthcare Materials, EarlyView.
This paper proposes hydrogel microtube carriers composed of barium alginate gel, enabling long‐term vascular retention without interrupting blood flow. Intravascular treatments have long been attracting attention for therapeutic efficacy, yet practical delivery methods remained unestablished.
Shota Sato, Hiroki Ohta, Naoki Takakura, Kentaro Kasa, Teppei Komatsu, Dai Kejia, Misato Yoshioka, Hirotaka James Okano, Hiroaki Onoe   +8 more
wiley   +1 more source

Adeno-Associated Virus VP1u Exhibits Protease Activity

Viruses, 2019
Adeno-associated viruses (AAVs) are being developed for gene delivery applications, with more than 100 ongoing clinical trials aimed at the treatment of monogenic diseases.
Justin J. Kurian, Renuk Lakshmanan, William M. Chmely, Joshua A. Hull, Jennifer C. Yu, Antonette Bennett, Robert McKenna, Mavis Agbandje-McKenna   +7 more
doaj   +1 more source

Performance of AAV8 vectors expressing human factor IX from a hepatic-selective promoter following intravenous injection into rats

, 2008
Background: Vectors based on adeno-associated virus-8 (AAV8) have shown efficiency and efficacy for liver-directed gene therapy protocols following intravascular injection, particularly in relation to haemophilia gene therapy. AAV8 has also been proposed
Amit Nathwani, Baker, AH, Lorraine M Work, Tracey Graham, McIntosh, JB, Jenny McIntosh, Nathwani, A, Work, LM, Graham, T, McIntosh, J, Andrew H Baker   +10 more
core   +1 more source

Piezo2 Mediates a Vicious Cycle of “Mechanical Homeostasis Imbalance—Inflammation” in Sensory Nerves and the Cartilage Endplate

Advanced Science, EarlyView.
In a lumbar spine instability model, dorsal root ganglion cells mediate the perception of relevant mechanical stresses through Piezo2 and subsequently release CGRP. CGRP activates the NF‐κB signaling pathway in cartilage endplate cells through the receptor RAMP1.
Hanpeng Xu, Wen Geng, Zhi Du, Yifan Du, Di Wu, Bide Tong, Huaizhen Liang, Xingyu Zhou, Zixuan Ou, Junyu Wei, Kun Wang, Yu Song, Wenbin Hua, Yan Xu, Wencan Ke, Bingjin Wang, Cao Yang   +16 more
wiley   +1 more source

Pharmacology of Recombinant Adeno-associated Virus Production

Molecular Therapy: Methods & Clinical Development, 2018
Recombinant adeno-associated viral (rAAV) vectors have been used in more than 150 clinical trials with a good safety profile and significant clinical benefit in many genetic diseases.
Magalie Penaud-Budloo, Achille François, Nathalie Clément, Eduard Ayuso   +3 more
doaj   +1 more source