Results 21 to 30 of about 70,372 (263)

Gene Transfer in Rodent Nervous Tissue Following Hindlimb Intramuscular Delivery of Recombinant Adeno-Associated Virus Serotypes AAV2/6, AAV2/8, and AAV2/9

Neuroscience Insights, 2019
Recombinant adeno-associated virus (rAAV) vectors have emerged as the safe vehicles of choice for long-term gene transfer in mammalian nervous system. Recombinant adeno-associated virus–mediated localized gene transfer in adult nervous system following ...
Asad Jan, Mette Richner, Christian B Vægter, Jens R Nyengaard, Poul H Jensen   +4 more
doaj   +1 more source

Adeno-Associated Virus Vectors and Hematology [PDF]

Blood, 1999
Gene therapists have a special fondness for hematology. Initially, this was due to the many inherited diseases that could potentially be cured by ex vivo genetic modification of hematopoietic stem cells. Once the early murine leukemia virus (MLV) vectors were found to transduce only a small percentage of primate stem cells, the search began for other ...
D W, Russell, M A, Kay
openaire   +2 more sources

Familial and genetic overlap between Sjögren’s disease and other autoimmune diseases

Frontiers in Immunology
PurposeTo evaluate familial clustering of Sjögren’s disease (SjD) with other autoimmune diseases and to characterize shared genetic architecture using an integrative genomic approach.MethodsMeta-analysis identified studies assessing autoimmune disease ...
Hitomi Ono-Minagi, Hitomi Ono-Minagi, Takuma Ohnishi, Natalie Atyeo, Kentaro Okuno, Peter D. Burbelo, John A. Chiorini   +6 more
doaj   +1 more source

Expression Profiles of Bovine Adeno-Associated Virus and Avian Adeno-Associated Virus Display Significant Similarity to That of Adeno-Associated Virus Type 5 [PDF]

Journal of Virology, 2006
ABSTRACT We present the first detailed expression profiles of nonprimate-derived adeno-associated viruses, namely, bovine adeno-associated virus (B-AAV) and avian adeno-associated virus (A-AAV), which were obtained after the infection of cell lines derived from their natural hosts.
Jianming, Qiu, Fang, Cheng, David J, Pintel   +2 more
openaire   +2 more sources

Virus Binding and Internalization Assay for Adeno-associated Virus

Bio-Protocol, 2017
The binding and internalization of adeno-associated virus (AAV) is an important determinant of viral infectivity and tropism. The ability to dissect these two tightly connected cellular processes would allow better understanding and provide insight on ...
Garrett Berry, Victor Tse
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: from defective virus to effective vector

Virology Journal, 2005
The initial discovery of adeno-associated virus (AAV) mixed with adenovirus particles was not a fortuitous one but rather an expression of AAV biology.
Gonçalves Manuel AFV
doaj   +1 more source

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 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

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