Results 41 to 50 of about 65,616 (261)

Ablation of Bscl2/seipin in hepatocytes does not cause metabolic dysfunction in congenital generalised lipodystrophy

Disease Models & Mechanisms, 2020
Mutations affecting the BSCL2 gene cause the most severe form of congenital generalised lipodystrophy (CGL). Affected individuals develop severe metabolic complications including diabetes and hepatic steatosis.
George D. Mcilroy, Sharon E. Mitchell, Weiping Han, Mirela Delibegović, Justin J. Rochford   +4 more
doaj   +1 more source

AAV Vectors for the Nucleolus [PDF]

Molecular Therapy, 2012
During the replication of wild-type adeno-associated virus (AAV), viral capsids are assembled in the nucleolus,1 and the capsid proteins interact with the nucleolar proteins nucleolin (NCL) and nucleophosmin (NPM1), possibly promoting their transport from the cytoplasm into the nucleolus.2,3 In the case of replication-incompetent AAV vectors, the ...
openaire   +2 more sources

Modulation of miR‐23b Wnt/β‐catenin Axis Strengthens Endothelial Barrier Properties

Advanced Science, EarlyView.
Early blood‐brain barrier (BBB) disruption contributes to stroke and CNS disease pathology. miR‐23b was identified as a regulator of BBB integrity in brain endothelial cells. Inhibition of miR‐23b enhanced barrier‐associated properties, promoted repair‐related signaling, and reduced BBB leakage in experimental stroke models, supporting further ...
Victor Anthony Martinez, Rose E. Presby, Sara Nakanishi, Pallavi Gaur, Ugur Akcan, Johannes Daugaard Krog, Iben Daugaard Krog, Lipi Das, David S. Johnson, Danny Jamoul, Douglas Jury, Brian Lawson, Jacob Körbelin, Lewis M. Brown, Vilas Menon, Dritan Agalliu, Irene Munk Pedersen   +16 more
wiley   +1 more source

Intracisternal delivery of NFkappaB-inducible scAAV2/9 reveals locoregional neuroinflammation induced by systemic kainic acid treatment.

Frontiers in Molecular Neuroscience, 2014
We have previously demonstrated disease-dependent gene delivery in the brain using an AAV vector responding to NFB activation as a probe for inflammatory responses.
Olivier eBockstael, Liliane eTenenbaum, Deniz eDalkara, Catherine eMelas, Olivier eDe Witte, Marc eLevivier, Abdelwahed eChtarto   +6 more
doaj   +1 more source

A Toolkit for Targeted Neuromodulation of Striatal Direct Pathway Neurons Rescues Parkinsonian Motor Deficits in Mice

Advanced Science, EarlyView.
An adeno‐associated virus (AAV) toolkit enables selective anatomical and functional targeting of striatal D1‐MSNs through retrograde transduction. Enhanced capsids and engineered enhancers drive robust transgene expression across murine and primate models.
Zexuan Hong, Yujing Zhang, Junjiao Zhang, Hanhe Liu, Qiwei Liu, Yanglei Li, Lixin Yang, Lixia Li, Zhongjie Liu, Zhen Yuan, Zhonghua Lu, Yefei Chen, Yuantao Li, Yuwu Jiang, Taian Liu   +14 more
wiley   +1 more source

No CpGs for AAVs? [PDF]

Blood, 2021
In this issue of Blood, Konkle et al report that 7 of 8 participants in a phase 1/2 trial of adeno-associated virus (AAV) vector (BAX335) for factor IX (FIX)-Padua gene transfer in patients with hemophilia B did not maintain expression despite steroid intervention, which the authors hypothesize is a result of the innate immune stimulatory effect of ...
openaire   +2 more sources

FGF13 Deficiency Ameliorates Paclitaxel‐Induced Neuropathic Pain by Inhibiting VASH1‐Mediated Microtubule Detyrosination to Promote Mitophagy

Advanced Science, EarlyView.
FGF13 is upregulated in DRG neurons of PIPNP model mice. DRG neuron‐specific knockout of FGF13 ameliorates PIPNP symptoms. Mechanistically, FGF13 potentiates microtubule detyrosination by promoting VASH1 binding to microtubules. FGF13 knockout suppresses VASH1‐mediated microtubule detyrosination and promotes α‐tubulin tyrosination.
Yiming Dong, Yidan Wang, Simeng Lv, Zishan Dong, Kaixi Zhi, Xiuhua Guo, Xuyan Li, Ruoxi Yu, Yiyi Zhang, Siyuan Cheng, Chuan Wang   +10 more
wiley   +1 more source

ANKS1B in the Nucleus Accumbens Controls Escalated Cocaine Self‐Administration via Regulating CBP‐FoxO3 Complex

Advanced Science, EarlyView.
ANKS1B in the nucleus accumbens plays a critical role in the transition from controlled to escalated cocaine intake. Mechanistically, ANKS1B interacts with CBP to epigenetically suppress FoxO3 through H3K27 acetylation. The ANKS1B‐CBP‐FoxO3 signaling cascade presents a novel theraputic target for the treatment of cocaine addiction.
Liping Yang, Xiaoxuan Wu, Xuan Chen, Chao Peng, Zihang Li, Shumin Gao, Shiqiu Meng, Jing Dong, Dong Wu, Liying Lv, Ying Han, Yanxue Xue, Lin Lu, Jie Shi, Jianfeng Liu, Yan Sun   +15 more
wiley   +1 more source

An in vivo systemic massively parallel platform for deciphering animal tissue-specific regulatory function

Frontiers in Genetics
Introduction: Transcriptional regulation is an important process wherein non-protein coding enhancer sequences play a key role in determining cell type identity and phenotypic diversity.
Ashley R. Brown, Ashley R. Brown, Grant A. Fox, Grant A. Fox, Irene M. Kaplow, Irene M. Kaplow, Irene M. Kaplow, Alyssa J. Lawler, Alyssa J. Lawler, BaDoi N. Phan, BaDoi N. Phan, BaDoi N. Phan, Lahari Gadey, Lahari Gadey, Morgan E. Wirthlin, Morgan E. Wirthlin, Easwaran Ramamurthy, Easwaran Ramamurthy, Gemma E. May, Ziheng Chen, Qiao Su, Qiao Su, C. Joel McManus, Robert van de Weerd, Robert van de Weerd, Andreas R. Pfenning, Andreas R. Pfenning, Andreas R. Pfenning   +27 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