Results 81 to 90 of about 65,616 (261)

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

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

Targeting NSUN2‐Mediated m5C Modification Attenuates Chondrocyte Senescence and NLRP3 Activation in Osteoarthritis

Advanced Science, EarlyView.
NSUN2‐mediated m5C modification cooperates with ALYREF to stabilize and export IP3R3 mRNA, increasing IP3R3 expression and Ca2 + overload in chondrocytes. This signaling promotes mitochondrial dysfunction, NLRP3 inflammasome activation, and senescence, thereby accelerating osteoarthritis progression.
Guping Mao, Wei Li, Zhencan Lin, Zengfa Deng, Ming Li, Zongrui Jiang, Changzhao Li, Dianbo Long, Yan Kang   +8 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

Fatty Acid Binding Protein 5 Mediates Astrocytic Pyroptosis and Neuroinflammation in Epilepsy via cGAS/STING Pathway

Advanced Science, EarlyView.
Astrocytic FABP5 promotes mitochondrial stress, cGAS‐STING pathway activation, pyroptosis, and neuroinflammation in epilepsy, contributing to seizure pathology. Genetic targeting of FABP5 or pharmacological inhibition of STING alleviates epileptic phenotypes, highlighting a potential therapeutic strategy for epilepsy.
Chen Chen, Yao Zhao, Yangye Lian, Yifei Hou, Lifen Gong, Tingting Wu, Yulin Yang, Jiaqi Zhu, Xin Wang, Kaihua Zhang, Jing Ding   +10 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

Targeting p21‐High Senescent Kupffer Cells Nanotherapeutically Potentiates Antitumor Immunity in Advanced Hepatocellular Carcinoma with Portal Vein Tumor Thrombus

Advanced Science, EarlyView.
Schematic illustration of a biomimetic SKEV@AAV platform targeting p21‐high senescent Kupffer cells (sKCs) in hepatocellular carcinoma (HCC) with portal vein tumor thrombus (PVTT). p21‐high sKCs accumulate in the PVTT microenvironment and establish an immunosuppressive, SASP‐rich environment that facilitates tumor cell proliferation and immune evasion.
Na Ta, Shuyi Wang, Boyan Zhang, Ning Liu, Yingchen Han, Aoran Liu, Ying Xu, Tingsong Chen, Ye Zhang, Qiuhua Luo, Tao Han   +10 more
wiley   +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  

Pathological Copper Overload Reprograms SOD1 Activation via COMMD1 to Promote Senescence and Fibrosis

Advanced Science, EarlyView.
This study uncovers a previously unrecognized copper‐COMMD1‐SOD1 regulatory axis, revealing that pathological copper overload paradoxically suppresses SOD1 activity by promoting COMMD1‐dependent disruption of SOD1 homodimerization. These findings redefine the regulatory role of copper in SOD1 biology and provide novel mechanistic insight into the ...
Yuqing Liu, Jing Liu, Wenqian Zhou, Yangyang Niu, Yan Zheng, Yiguo Liu, Yingying Zhang, Chen Yu   +7 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