Results 101 to 110 of about 89,643 (298)

In vivo evaluation of tropism and biodistribution of synthetic and natural adeno-associated viral vectors by next-generation sequencing

Биопрепараты: Профилактика, диагностика, лечение
INTRODUCTION. The creation of synthetic adeno-associated virus (AAV) vectors during gene therapy development is a labour-intensive and expensive process. The optimal solution to minimise the time and costs associated with gene therapy development lies in
D. O. Maksimov, D. A. Naumova, E. A. Astakhova, V. V. Artemev, S. A. Biryukov, I. S. Abramov, A. A. Navoikova, N. V. Rudev, S. G. Feoktistova, O. V. Glazova, O. N. Mityaeva, P. Yu. Volchkov   +11 more
doaj   +1 more source

HLA-DRB1 allele frequencies in Japanese patients with MPO-AAV or PR3-AAV or in healthy controls.

, 2016
HLA-DRB1 allele frequencies in Japanese patients with MPO-AAV or PR3-AAV or in healthy controls.
Hiroshi Furukawa (290179), Aya Kawasaki (518768), Shigeto Tohma (290204), Kunihiro Yamagata (413551), Naoyuki Tsuchiya (290203), Takayuki Sumida (12557), Narumi Hasebe (2742082), Yoshihiro Arimura (2742073), Shigeto Kobayashi (2742076), Shoichi Ozaki (413554), Hiroshi Hashimoto (518772), Ken-ei Sada (752034), Hirofumi Makino (25824), Masayoshi Harigai (18940), Nobuyuki Miyasaka (12600), Seiichi Matsuo (588832), Hidehiro Yamada (398062), Misaki Hidaka (2742085), Fumio Hirano (2742079)   +18 more
core   +1 more source

Gut Microbiota‐Isoallolithocholic Acid Crosstalk Promotes Calcium Oxalate Kidney Stone Formation via PARP1‐Mediated Parthanatos

Advanced Science, EarlyView.
ABSTRACT Bile acids have been implicated in calcium oxalate (CaOx) nephrolithiasis. Here, we employ multi‐omics approaches to identify isoallolithocholic acid (isoalloLCA) as the key bile acid elevated in the feces, serum, kidney, and urine of CaOx rats, confirmed by spatial metabolomics.
Zijian Zhou, Ruixi Yu, Xuan Zhou, Feng Shi, Zeyu Lin, Peng Gao, Lujia Wang, Ninghan Feng, Zhong Wu, Dawei Wang   +9 more
wiley   +1 more source

Advancements in CRISPR-based in vivo gene therapy for hemophilia

Frontiers in Genome Editing
Hemophilia is an X-linked hereditary bleeding disorder caused by loss-of-function mutations in the genes encoding coagulation factors, leading to excessive bleeding and potentially being life-threatening.
Xiaoyu Zhang, Xiaoyu Zhang, Kaidi Xu, Shihui Zou, Zhiyao Zhu, Zhiyao Zhu, Shuzhi Teng, Ping Huang   +7 more
doaj   +1 more source

Cellular unfolded protein response against viruses used in gene therapy

Frontiers in Microbiology, 2014
Viruses are excellent vehicles for gene therapy due to their natural ability to infect and deliver the cargo to specific tissues with high efficiency.
Dwaipayan eSen, Balaji eBalakrishnan, Giridhara R. eJayandharan, Giridhara R. eJayandharan   +3 more
doaj   +1 more source

HLA-DPB1 allele frequencies in Japanese patients with MPO-AAV or PR3-AAV or in healthy controls.

, 2016
HLA-DPB1 allele frequencies in Japanese patients with MPO-AAV or PR3-AAV or in healthy controls.
Hiroshi Furukawa (290179), Aya Kawasaki (518768), Shigeto Tohma (290204), Kunihiro Yamagata (413551), Naoyuki Tsuchiya (290203), Takayuki Sumida (12557), Narumi Hasebe (2742082), Yoshihiro Arimura (2742073), Shigeto Kobayashi (2742076), Shoichi Ozaki (413554), Hiroshi Hashimoto (518772), Ken-ei Sada (752034), Hirofumi Makino (25824), Masayoshi Harigai (18940), Nobuyuki Miyasaka (12600), Seiichi Matsuo (588832), Hidehiro Yamada (398062), Misaki Hidaka (2742085), Fumio Hirano (2742079)   +18 more
core   +1 more source

Macrophage PABPC4‐SPP1 Axis Orchestrates Immunosuppression in Colorectal Cancer

Advanced Science, EarlyView.
In the CRC microenvironment, macrophage PABPC4 binds to the 3′UTR of SPP1 mRNA to stabilize its expression, thereby sustaining M2‐like immunosuppressive macrophage polarization. Concurrently, this axis suppresses CD8+ T cell effector functions via CD44 signaling, collectively fostering an immunosuppressive niche that drives tumor progression.
Meng Wang, Fang Yang, Yuan Gao, Liuli Li, Yulan Huang, Hui Yao, Sijie Zhao, Kun Zhao, Tianying Zhang, Hui Heng, Jiaxin Xu, Yuancai Xiang, Weidong Xiao, Shaofan Hu, Hongming Miao   +14 more
wiley   +1 more source

An improved high-resolution method for quantitative separation of empty and filled AAV8 capsids by strong anion exchange HPLC

Frontiers in Bioengineering and Biotechnology
Cell and gene therapy (CGT) is a field of therapeutic medicine that aims to treat, prevent, and cure diseases using engineered cells (stem cells, immune cells, and differentiated adult or fetal cells), vectors [Adeno Associated Virus (AAV), Adeno Virus ...
Samantha Schrecke, Kevin McManus, Cassandra Moshfegh, Jessica Stone, Thuy-Uyen Nguyen, Gustavo Rivas, Ismaeel Muhamed, Daniel A. J. Mitchell   +7 more
doaj   +1 more source

Protein Carrier AAV

ABSTRACT AAV is widely used for efficient delivery of DNA payloads. The extent to which the AAV capsid can be used to deliver a protein payload is unexplored. Here, we report engineered AAV capsids that directly package proteins – Protein Carrier AAV (pcAAV). Nanobodies inserted into the interior of the capsid mediate
Mareike D. Hoffmann, Ryan J. Sorensen, Ajay Extross, Yungui He, Daniel Schmidt   +4 more
openaire   +2 more sources

KLF5 Downregulation Links Impaired BNIP3‐Mediated Mitophagy to Inflammatory Valve Remodeling in Calcific Aortic Valve Disease

Advanced Science, EarlyView.
In human CAVD, KLF5 is reduced in VIC‐rich regions and remodeling/stress‐associated VIC states. In VICs, KLF5 sustains BNIP3 promoter activity and BNIP3‐mediated mitophagy, thereby limiting cytosolic mtDNA accumulation. KLF5 loss weakens mitochondrial quality control and enhances mtDNA‐sensitive STING/NF‐κB/NLRP3 inflammatory signaling under osteogenic
Jin‐Hui Bian, Jia‐Xi Gu, Zhang Yue, Chun‐Ze Yuan, Hai‐Bin Ji, Ao Li, Yu Zhong, Xiu‐Fan Xu, Yu‐Qiu He, Ye‐Fan Jiang, Hong‐Shan Chen, Xiang Chen, Meng‐Lin Chao, Yong‐Feng Shao   +13 more
wiley   +1 more source