Results 101 to 110 of about 79,958 (292)

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

Targeting Endogenous Lipophagy: A Novel Strategy to Enhance MSC Osteogenesis and Mineralization for Senile Osteoporosis Therapy

Advanced Science, EarlyView.
Schematic representation of the role of lipophagy in bone mesenchymal stem cells(MSCs). In healthy MSCs, functional lipophagy efficiently degrades lipid droplets to support oxidative phosphorylation and cellular energy production, thereby facilitating osteogenic differentiation and matrix mineralization.
Chaoqiang Chen, Zhidong Liu, Yanhang Sun, Xiaojun Xu, Zhexiao Lan, Junhao Zhao, Peitao Xu, Guiwen Ye, Jinteng Li   +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

AAV-HGFK1 and Ad-p53 cocktail therapy prolongs survival of mice with colon cancer

, 2008
This study tried to evaluate the application of a novel cancer gene therapy using recombinant adeno-associated virus (AAV) carrying the kringle 1 domain of human hepatocyte growth factor (AAV-HGFK1) in combination with recombinant adenovirus carrying p53
B. Nie, Hsueh, WD, Huo, LF, M. C.M. Lin, Wong, OGW, Lin, MCM, O. G.-W. Wong, B. Jiang, W. D. Hsueh, Kung, HF, Z. Shen, Nie, B, Wen, JB, H.-F. Kung, Shen, Z, Jiang, B, J.-B. Wen, L.-F. Huo   +17 more
core   +1 more source

Mechanosensitive Piezo1/Osteocalcin/Irisin Axis Protects Against Disuse‐Induced Muscle Atrophy

Advanced Science, EarlyView.
Mechanical unloading suppresses bone Piezo1 expression, which reduces circulating undercarboxylated osteocalcin (unOCN). unOCN reduction subsequently exacerbates IMM‐induced Fndc5/Irisin decrease and drives severe muscle atrophy. Bone Piezo1 activation or exogenous osteocalcin/Irisin ameliorate muscle atrophy, while muscle‐specific Gprc6a or Fndc5 ...
Zhaolu Wang, Xiuying Jiang, Xi Sun, Hao Chen, Jianjun Jin, Xin'e Shi   +5 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  

Ultrasound-targeted microbubble destruction enhances AAV mediated gene transfection: human RPE cells in vitro and the rat retina in vivo

, 2009
The present study was performed to investigate the efficacy and safety of Ultrasound-targeted microbubble destruction (UTMD) mediated rAAV2-EGFP to cultured human retinal pigment epithelium (RPE) cells _in vitro_ and the rat retina _in vivo_.
Xiaozhi Zheng, Huiping Wang, Ying Wu, Lianfang Du, Hongli Li, Fan Li   +5 more
core  

Paternal Caffeine Exposure Programs Offspring Stress Vulnerability via Sperm Dlk1‐Dio3 Imprinting‐Directed Remodeling of a Novel Neural Circuit

Advanced Science, EarlyView.
The study elucidates that paternal preconception stress can drive offspring hyperresponsivity of the stress system via hypomethylation of a specific DNA region in sperm. This key link is confirmed in a cohort of prospective fathers: the epigenetic alteration is associated with elevated stress hormone levels.
Mengxi Lu, Gaole Dai, Sen Zhu, Shuai Zhang, Tingting Wang, Yuan Meng, Fang Yang, Xiaoyi Han, Hui Wang, Hao Kou, Dan Xu   +10 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