Results 51 to 60 of about 36,553 (255)

Common AAV gene therapy vectors show nonselective transduction of ex vivo human brain tissue

Molecular Therapy: Methods & Clinical Development
The ability to deliver a therapeutic sequence to a specific cell type in the human brain would make possible innumerable therapeutic options for some of our most challenging diseases; however, studies on adeno-associated virus (AAV) vector tropism have ...
JP McGinnis, Joshua Ortiz-Guzman, Maria Camila Guevara, Sai Mallannagari, Benjamin D.W. Belfort, Suyang Bao, Snigdha Srivastava, Maria Morkas, Emily Ji, Angela Addison, Evelyne K. Tantry, Sarah Chen, Ying Wang, Zihong Chen, Kalman A. Katlowitz, Jeffrey J. Lange, Melissa M. Blessing, Carrie A. Mohila, M. Cecilia Ljungberg, Guillermo Aldave, Ali Jalali, Akash Patel, Sameer A. Sheth, Howard L. Weiner, Shankar Gopinath, Ganesh Rao, Akdes Serin Harmanci, Daniel J. Curry, Benjamin R. Arenkiel   +28 more
doaj   +1 more source

Preclinical Assessment of Immune Responses to AAV (adeno-associated virus) Vectors

Frontiers in Immunology, 2014
Transitioning to human trials from preclinical models resulted in the emergence of inhibitory AAV vector immune responses which has become a hurdle for sustained correction.
Etiena eBasner-Tschakarjan, Enoch eBijjiga, Ashley Thomas Martino   +2 more
doaj   +1 more source

Inhibition of VEGF‐Induced Angiogenesis and Vascular Leakage by Bicistronic Co‐Expression of Aflibercept and COMP‐Ang1

Advanced Healthcare Materials, EarlyView.
A bicistronic rAAV8 vector encoding Aflibercept (Afb) and COMP‐cAng1 simultaneously coordinates VEGF clearance and Tie2‐mediated vessel maturation. In a 3D angiogenesis‐on‐a‐chip model, rAAV8‐Afb/cAng1 effectively reverses pathological barrier breakdown under clinical disease‐mimicking challenges.
Bong‐Kyu Kim, Jaehong Min, Sang‐Hee Park, Subeen Shin, Sangbin Yu, Jeong‐Eun Park, Sujin Kim, Wooju Park, Jihoon Ko, Sunjong Kwon   +9 more
wiley   +1 more source

Aiming for spatial and temporal control of gene expression

eLife
Bioluminescent imaging is helping researchers better understand the effectiveness of tissue regeneration enhancers delivered to injured heart tissue by different adeno-associated virus vectors.
Stanislao Igor Travisano, Ching-Ling Lien   +1 more
doaj   +1 more source

Gut Microbiota Regulates Systemic Inflammatory Response and Compensatory Anti‐Inflammatory Response Syndromes by Targeting PF4+ Macrophages in Acute Pancreatitis

Advanced Science, EarlyView.
ABSTRACT Acute pancreatitis (AP) begins with pancreatic local inflammation, leading to the onset of systemic inflammatory response syndrome (SIRS), followed by compensatory anti‐inflammatory response syndrome (CARS), which causes immune paralysis and higher mortality rate.
Liwei Liu, Guanqun Li, Dongxu Lu, Haoran Ding, Tianqi Lu, Yuhang Sui, Can Zhang, Yu Xie, Rui Kong, Hua Chen, Xuewei Bai, Hongtao Tan, Dongbo Xue, Xianzhi Meng, Le Li, Bei Sun   +15 more
wiley   +1 more source

Adipocyte‐Derived Leptolin Enhances Energy Expenditure and Prevents Obesity

Advanced Science, EarlyView.
We identified a novel adipokine, which we named leptolin. In humans, leptolin levels in white adipose tissue were positively correlated with exercise and negatively associated with body mass index. We observed elevated leptolin in serum from athletes and lower leptolin in serum from obese individuals.
Jiarui Liu, Bingwei Wang, Zhijie Su, Xiaoxv Han, Miao He, Yun Zhao, Yujia Hou, Daotong Li, Weiguang Zhang, Lihua Qin, Ke Wang, Yanchun Li, Yi Yan, Siwang Yu, Xiaoshuai Huang, Tairan Yuwen, Jianyuan Luo, Ruimao Zheng   +17 more
wiley   +1 more source

When size matters: A novel compact Cas12a variant for in vivo genome editing.

PLoS Biology
A new study characterizes and improves a novel small Cas12a variant before adapting it for in vivo genome editing by delivery via adeno-associated virus (AAV) vectors, showcasing the potential of small CRISPR systems and their compatibility with viral ...
Felix Bubeck, Dirk Grimm
doaj   +1 more source

Agnuside Stabilizes the Complex I Assembly Factor NDUFAF6 to Reinforce Mitochondrial Efficiency and Thermogenic Responsiveness

Advanced Science, EarlyView.
ABSTRACT Brown and beige adipocytes dissipate energy as heat, yet effective strategies to enhance their mitochondrial efficiency remain limited. Here, we identify Agnuside (AGN) as a selective stabilizer of the complex I assembly factor NDUFAF6. AGN directly binds cytosolic NDUFAF6, suppresses its ubiquitination, prolongs its half‐life, and facilitates
Qingwen Zhao, Li Xie, Qianzhuo Wang, Shuying Dai, Bei Li, Yingjuan Zhang, Zhe Sun, Yue Gao   +7 more
wiley   +1 more source

Human Hepatocyte Transduction with Adeno-Associated Virus Vector

, 2022
As the adeno-associated virus (AAV) vectors hold unique advantages over other viral vectors, AAV gene therapy has accumulated rapid progress and development. Liver-targeted gene therapy by AAV vectors has been successfully applied in clinical trials for many diseases.
Song, Zhenwei, Shao, Wenwei, Song, Liujiang, Pei, Xieolei, Li, Chengwen   +4 more
openaire   +2 more sources

Phosphorylation‐Facilitated CKB Lactylation At K11 By GCN5 Enhances Creatine Kinase Activity and Mitigates Neuronal Damage After Cerebral Ischemia‐Reperfusion

Advanced Science, EarlyView.
This study uncovers hierarchical coordination between K11 lactylation and S199 phosphorylation of CKB in cerebral ischemia‐reperfusion injury. Such dual modifications potentiate CKB enzymatic function, remodel energy metabolism, alleviate oxidative stress and neuronal damage, and represent a viable therapeutic target for stroke treatment.
Chao Duan, Ruolin Zhang, Shihui Ding, Ying Feng, Tao Shen, Ziwei Wang, Wendai Bao, Ke Shui, Jun Qin, Jun Chen, Min Zhang, Zijing Ren, Dongmei Zhao, Xingrong Guo, Zhibing Ai, Xin Yang, Peiyang Zhou, Zhiqiang Dong   +17 more
wiley   +1 more source