Results 21 to 30 of about 396,665 (375)

Adeno-Associated Virus Toolkit to Target Diverse Brain Cells.

Annual Review of Neuroscience, 2022
Recombinant adeno-associated viruses (AAVs) are commonly used gene delivery vehicles for neuroscience research. They have two engineerable features: the capsid (outer protein shell) and cargo (encapsulated genome).
Rosemary C. Challis, Sripriya Ravindra Kumar, Xinhong Chen, David Goertsen, G. Coughlin, A. Hori, Miguel R. Chuapoco, T. Otis, T. F. Miles, V. Gradinaru   +9 more
semanticscholar   +1 more source

LAMP3 transfer via extracellular particles induces apoptosis in Sjögren’s disease

Scientific Reports, 2023
Sjögren’s disease (SjD) is an autoimmune disease that affects exocrine tissues and is characterized by increased apoptosis in salivary and lacrimal glands.
Tsutomu Tanaka, Hiroyuki Nakamura, Duy T. Tran, Blake M. Warner, Yan Wang, Tatsuya Atsumi, Masayuki Noguchi, John A. Chiorini   +7 more
doaj   +1 more source

Efficient CRISPR editing with a hypercompact Cas12f1 and engineered guide RNAs delivered by adeno-associated virus

Nature Biotechnology, 2021
Gene therapy would benefit from a miniature CRISPR system that fits into the small adeno-associated virus (AAV) genome and has high cleavage activity and specificity in eukaryotic cells.
Do Yon Kim, J. M. Lee, Su Bin Moon, H. Chin, Seyeon Park, Youjung Lim, Daesik Kim, T. Koo, J. Ko, Yong-Sam Kim   +9 more
semanticscholar   +1 more source

Adeno-associated Virus Gene Therapy for Hemophilia.

Annual Review of Medicine, 2022
In vivo gene therapy is rapidly emerging as a new therapeutic paradigm for monogenic disorders. For almost three decades, hemophilia A (HA) and hemophilia B (HB) have served as model disorders for the development of gene therapy.
B. Samelson-Jones, Lindsey A. George
semanticscholar   +1 more source

Adeno‐associated virus (AAV)-based gene therapy for glioblastoma

Cancer Cell International, 2021
Glioblastoma (GBM) is the most common and malignant Grade IV primary craniocerebral tumor caused by glial cell carcinogenesis with an extremely poor median survival of 12–18 months.
Xin Xu, Wenli Chen, Wenjun Zhu, Jing Chen, Bin Ma, Jianxia Ding, Zaichuan Wang, Yifei Li, Yeming Wang, Xiaochun Zhang   +9 more
doaj   +1 more source

Infectious Entry Pathway of Adeno-Associated Virus and Adeno-Associated Virus Vectors [PDF]

Journal of Virology, 2000
ABSTRACT We have investigated the infectious entry pathway of adeno-associated virus (AAV) and recombinant AAV vectors by assessing AAV-mediated gene transfer and by covalently conjugating fluorophores to AAV and monitoring entry by fluorescence microscopy.
R. Jude Samulski, Rose Wilcher, Jeffrey S. Bartlett   +2 more
openaire   +3 more sources

Surface immobilization of hexa-histidine-tagged adeno-associated viral vectors for localized gene delivery. [PDF]

, 2010
Adeno-associated viral (AAV) vectors, which are undergoing broad exploration in clinical trials, have significant promise for therapeutic gene delivery because of their safety and delivery efficiency.
Bethi, SR, Gujraty, K, Jang, J-H, Kane, RS, Koerber, JT, Schaffer, DV   +5 more
core   +5 more sources

Clinical and laboratory evaluation of sicca complaints: distinctive aspects of primary, secondary and non-Sjogren syndrome

Advances in Rheumatology, 2022
Background Sjögren Syndrome (SS) is a systemic autoimmune disease with a wide spectrum of manifestations that can lead to misdiagnosis. This study describes and compares demographic, clinical, serological, and histopathological data from subjects with SS
Fabíola Reis de Oliveira, Ana Carolina F. Motta, Carolina Maria Módulo, Denny Marcos Garcia, John A. Chiorini, Paulo Louzada-Junior, Eduardo Melani Rocha   +6 more
doaj   +1 more source

Strategies to package recombinant Adeno-Associated Virus expressing the N-terminal gasdermin domain for tumor treatment

Nature Communications, 2021
Pyroptosis induced by the N-terminal gasdermin domain (GSDMNT) holds great potential for anti-tumor therapy. However, due to the extreme cytoxicity of GSDMNT, it is challenging to efficiently produce and deliver GSDMNT into tumor cells.
Yuan Lu, Wenbo He, Xin Huang, Yu He, Xiaojuan Gou, Xiaoke Liu, Zhe Hu, Weize Xu, K. Rahman, Shan Li, Sheng Hu, Jie Luo, Gang Cao   +12 more
semanticscholar   +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.
Fang Cheng, David J. Pintel, Jianming Qiu   +2 more
openaire   +3 more sources