Results 111 to 120 of about 89,643 (298)

Adeno-Associated Virus (AAV) Gene Therapy pada Hemofilia: A Systematic Review

Majalah Kedokteran Andalas
Hemofilia A (HA) dan Hemofilia B (HB) adalah kelainan perdarahan terkait kromosom X yang disebabkan oleh defisiensi faktor koagulasi Faktor VIII (FVIII) atau Faktor IX (FIX).
Winda Angriani, Fitrah Afdhal
doaj   +1 more source

SerpinA3 is an Endogenous TGF‐β Receptor Antagonist that Attenuates Cardiac Fibroblast Activation and Fibrotic Remodeling

Advanced Science, EarlyView.
SerpinA3 acts as an endogenous TGF‐β receptor antagonist that binds to the extracellular domain of TGFR‐1, thereby preventing TGFR‐1–TGFR‐2 complex formation. This receptor‐level blockade suppresses TGF‐β/Smad2/3 signaling, attenuates cardiac fibroblast activation, and extracellular matrix deposition, and ultimately alleviates pressure overload–induced
Hui Wang, Jieyu Cui, Chunlu Huang, Wanyi Guo, Tong Liu, Yi Zhu, Shuai Shao, Kangyin Chen, Jinlong He, Qiankun Bao   +9 more
wiley   +1 more source

Advancing AAV vector manufacturing: challenges, innovations, and future directions for gene therapy

Frontiers in Molecular Medicine
Adeno-Associated Virus (AAV) vectors are at the forefront of gene therapy, offering transformative therapeutic potential for many genetic disorders.
N. Charan S. S. Kowshik, Pushpendra Singh   +1 more
doaj   +1 more source

SCTR-Expressing Astrocyte-Serotonergic Neuron Signaling Drives Sexual Dimorphism in Depression Through KAT7/H4ac-Foxc2 Epigenetic Axis in an AMPK-Dependent Manner. [PDF]

Adv Sci (Weinh)
Astrocytic SCTR modulates depressive‐related phenotypes via AMPKα1, whereas lowered Mt1 blunts Megalin‐dependent AMPKα2 signaling in serotonergic neurons. Diminished AMPKα2 hinders KAT7 nuclear translocation, curtails KAT7 occupancy and H4K8ac at the Foxc2 promoter, and represses Foxc2 transcription, revealing cell‐type‐specific AMPK‐linked epigenetic ...
Meng F, Liu J, Dai J, Wu M, Wang W, Zhang M, Jiang S, Cao Y, Xu L, Du L, Wang D, Zhao D, Qin G, Wang D, Li W, Li C.   +15 more
europepmc   +2 more sources

AAV-mediated rescue of Eps8 expression in vivo restores hair-cell function in a mouse model of recessive deafness

, 2022
The transduction of acoustic information by hair cells depends upon mechanosensitive stereociliary bundles that project from their apical surface. Mutations or absence of the stereociliary protein EPS8 cause deafness in humans and mice, respectively ...
Yelena Dayn (16300601), Adam J Carlton (16300112), Tsung-Chang Sung (436234), Steve D M Brown (16300607), Richard Goodyear (4460608), Michael R Bowl (16300604), Jing-Yi Jeng (16259351), Stuart L Johnson (9120284), Guy Richardson (4460482), Uri Manor (775070), Federico Ceriani (16269425), Walter Marcotti (224237), Colbie Chinowsky (16300598)   +12 more
core  

An RNA‐Binding Protein RBMS1 Promotes Endothelial Cell Autophagy to Maintain Vascular Homeostasis and Suppress Deep Vein Thrombosis

Advanced Science, EarlyView.
RBMS1, an RNA‐binding protein, restores vascular endothelial homeostasis by enhancing ATG3‐mediated autophagy, thereby attenuating thrombus formation and suggesting a potential avenue for further therapeutic exploration in DVT. ABSTRACT Deep vein thrombosis (DVT) is a prevalent vascular disorder characterized by aberrant coagulation within the deep ...
Chu Chu, Xinkui Liu, Nannan Fan, Xiaoyan Yu, Qiaoqiao Han, Yunhong Zhang, Wei Li, Yongqing Cai, Miaomiao Zhou, Fang Li, Feifei Shi, Zhen Zhang, Bin Wang, Xia Li   +13 more
wiley   +1 more source

Development of recombinant adeno-associated virus empty capsids as a reference standard for quality control of gene therapy products

Биопрепараты: Профилактика, диагностика, лечение
INTRODUCTION. The development of adeno-associated virus (AAV)-based gene therapy products in Russia requires establishing reference standards, which are used throughout the pharmaceutical development cycle, and monitoring their stability during the ...
A. V. Tumaev, D. Yu. Voloshin, E. S. Berdinskikh, E. L. Sakhibgaraeva, E. V. Golovin, E. N. Subcheva, O. O. Vasileva, A. A. Galieva, A. A. Chuvashov, E. S. Novikova, A. V. Karabelsky   +10 more
doaj   +1 more source

An AAV-Based NF-κB-Targeting Gene Therapy (rAAV-DMP-miR533) to Inflammatory Diseases

Journal of Inflammation Research, 2022
Tao Luo,1 Yile Wang,1 Hailin Tang,1 Fei Zhou,2 Ying Chen,3 Bing Pei,4 Jinke Wang1 1State Key Laboratory of Bioelectronics, Southeast University, Nanjing, 210096, People’s Republic of China; 2School of Food Engineering and Biotechnology, Hanshan Normal ...
Luo T, Wang Y, Tang H, Zhou F, Chen Y, Pei B, Wang J   +6 more
doaj  

ACSL4‐Dependent Lysosomal Lipid Peroxidation Links WTAP‐Mediated m6A Modification to Intervertebral Disc Degeneration

Advanced Science, EarlyView.
IL‐1β upregulates the protein level of WTAP, which promotes the m6A modification of ACSL4 mRNA in an IGF2BP2‐dependent manner, thereby enhancing its stability. The increased ACSL4 drives lipid peroxidation, leading to lysosomal membrane permeabilization (LMP) and impaired mitophagy, which collectively accelerate intervertebral disc degeneration (IVDD).
Shu Jia, Xu Gao, Laimin Zhu, Haixia Song, Jing Wu, Shang Chen, Sheng Gao, Lu Zhang, Chunyang Meng   +8 more
wiley   +1 more source

Editorial: Gene therapy 2.0: Biotechnology for circuit engineering and complex therapeutic approaches

Frontiers in Bioengineering and Biotechnology, 2022
Luís Quintino, Luís Pereira de Almeida, Cecilia Lundberg   +2 more
doaj   +1 more source