Results 131 to 140 of about 144,727 (384)

A Generic Method for Fast and Sensitive Detection of Adeno-Associated Viruses Using Modified AAV Receptor Recombinant Proteins

Molecules, 2019
Adeno-Associated Viruses (AAV) are widely used gene-therapy vectors for both clinical applications and laboratory investigations. The titering of different AAV preparations is important for quality control purposes, as well as in comparative studies ...
Mengtian Cui, Yabin Lu, Can Tang, Ran Zhang, Jing Wang, Yang Si, Shan Cheng, Wei Ding   +7 more
doaj   +1 more source

Protein Carrier AAV

ABSTRACTAAV 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).
Mareike D. Hoffmann, Ryan J. Sorensen, Ajay Extross, Yungui He, Daniel Schmidt   +4 more
openaire   +2 more sources

Post‐Translational Modifications in Cilia and Ciliopathies

Advanced Science, EarlyView.
This review synthesizes current understanding of post‐translational modifications (PTMs) in ciliary proteins and emphasizes their roles in ciliary formation, homeostasis, and signaling. This review also discusses the implication of PTM dysregulation in ciliopathies and explores therapeutic strategies targeting PTM‐modifying enzymes.
Jie Ran, Jun Zhou
wiley   +1 more source

In Vivo AAV-CRISPR/Cas9–Mediated Gene Editing Ameliorates Atherosclerosis in Familial Hypercholesterolemia

Circulation, 2019
Supplemental Digital Content is available in the text. Background: Mutations in low-density lipoprotein (LDL) receptor (LDLR) are one of the main causes of familial hypercholesterolemia, which induces atherosclerosis and has a high lifetime risk of ...
Huan Zhao, Yan Li, Lingjuan He, Wenjuan Pu, Wei Yu, Yi Li, Yan-ting Wu, Chenming Xu, Yuda Wei, Q. Ding, B. Song, He-feng Huang, Bin Zhou   +12 more
semanticscholar   +1 more source

From Code to Life: The AI‐Driven Revolution in Genome Editing

Advanced Science, EarlyView.
This review explores AI‐driven advancements in genome editing, focusing on CRISPR optimization, enhanced targeting precision, reduced off‐target effects, and novel tool design. It addresses ethical challenges, data biases, and highlights future prospects in precision medicine, agriculture, and synthetic biology.
Zhidong Li, Wasi Ullah Khan, Genxiang Bai, Chao Dong, Jungang Wang, Youpeng Zhang, Chong Wang, Hongbin Zhang, Wenyi Wang, Ming Luo, Fei Chen   +10 more
wiley   +1 more source

Post-selected weak measurement beyond the weak value

, 2010
Closed expressions are derived for the quantum measurement statistics of pre-and postselected gaussian particle beams. The weakness of the pre-selection step is shown to compete with the non-orthogonality of post-selection in a transparent way.
L. Diósi, M. V. Berry, Tamás Geszti, Y. Aharonov   +3 more
core   +1 more source

Metabolic Stress‐Induced Choline Kinase α (CHKA) Activation in Endothelial Subpopulation Contributes to Diabetes‐Associated Microvascular Dysfunction

Advanced Science, EarlyView.
This study uncovers CHKA as a pivotal driver of vascular dysfunction in diabetic retinopathy and highlights its role in endothelial dysfunction through NAD⁺‐SIRT1‐Notch signaling. Single‐cell and functional analyses, combined with clinical and genetic validation, suggest CHKA as a promising therapeutic target for the prevention and treatment of ...
Ling Ren, Linyu Zhang, Yun Bai, Chang Huang, Xiaosa Li, Fanfei Ma, Wan Mu, Mudi Yao, Chang Jiang, Xiangjun Chen, Qin Jiang, Biao Yan   +11 more
wiley   +1 more source

Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration

Nature Communications, 2018
Gene therapy mediated by recombinant adeno-associated virus (AAV) vectors is a promising treatment for systemic monogenic diseases. However, vector immunogenicity represents a major limitation to gene transfer with AAV vectors, particularly for vector re-
Amine Meliani, F. Boisgérault, R. Hardet, S. Marmier, F. Collaud, G. Ronzitti, C. Leborgne, Helena Costa Verdera, Marcelo Simon Sola, S. Charles, A. Vignaud, L. van Wittenberghe, Giorgia Manni, O. Christophe, F. Fallarino, C. Roy, Alicia M. Michaud, P. Ilyinskii, T. Kishimoto, F. Mingozzi   +19 more
semanticscholar   +1 more source

Restoration of SIRT3 Expression in Aged Mice Alleviates UUO‐Induced Renal Fibrosis by Reducing GSK‐3β Hyperacetylation

Advanced Science, EarlyView.
SIRT3 expression is significantly downregulated in the aged kidney, rendering the kidney more vulnerable to fibrotic changes in obstructive nephropathy. By promoting GSK‐3β deacetylation, SIRT3 indirectly inhibits Wnt/β‐catenin signaling, thereby suppressing renal fibrosis.
Jing Wang, Xiang Ren, Huan Lu, Zihao Guo, Xing Li, Yiqun Tian, Yisheng Yin, Zhenliang Qin, Kun Yun, Minglong Wu, Gang Chen, Xiaoyong Zeng   +11 more
wiley   +1 more source

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