Results 41 to 50 of about 73,809 (216)

Innate Immunocompetent hiPSC‐Derived Neurospheroids Capture Early CNS Responses to rAAV

Advanced Science, EarlyView.
Knowledge of human CNS immune responses to AAV‐based gene therapies remains limited due to the lack of immune‐competent human models. Here, a hiPSC‐derived 3D neuroimmune platform integrating neurospheroids and microglia is established using stirred‐tank bioreactors.
Catarina M. Gomes, Gabriela Silva, Mafalda Aleixo, Marta Gomes, Daniel Simão, Stephan J. Holtkamp, Diana D. Lobo, Pradeep Harish, Rosalind Jenkins, Lekh N. Dahal, Rui J. Nobre, Luís de Pereira de Almeida, Mark Trautwein, Paula M. Alves, Catarina Brito   +14 more
wiley   +1 more source

Adeno-associated virus for cystic fibrosis gene therapy

Brazilian Journal of Medical and Biological Research, 2011
Gene therapy is an alternative treatment for genetic lung disease, especially monogenic disorders such as cystic fibrosis. Cystic fibrosis is a severe autosomal recessive disease affecting one in 2500 live births in the white population, caused by ...
S.V. Martini, P.R.M. Rocco, M.M. Morales
doaj   +3 more sources

Disrupted TFEB/GDNF–cAMP/ATP Coupling Underlies Astrocytic Dysfunction and Depression in LRRK2 G2019S Parkinson's Mice

Advanced Science, EarlyView.
ABSTRACT The LRRK2 G2019S mutation, a Parkinson's disease–linked variant, has been associated with depression‐like phenotypes, but mechanisms remain unclear. We chart age‐dependent behavioral changes and astrocyte reactivity in G2019S mice and define a pathway connecting LRRK2 to TFEB/GDNF signaling, cellular energetics, and inflammation.
Longping Yao, Maryam Hatami, Sumeyye Koc, Qing Jiang, Thomas Skutella, Yuanfeng Zhang   +5 more
wiley   +1 more source

Mucopolysaccharidosis type IIIB: a current review and exploration of the AAV therapy landscape

Neural Regeneration Research
Mucopolysaccharidoses type IIIB is a rare genetic disorder caused by mutations in the gene that encodes for N-acetyl-alpha-glucosaminidase. This results in the aggregation of heparan sulfate polysaccharides within cell lysosomes that leads to progressive
Courtney J Rouse, Victoria N Jensen, Coy D Heldermon   +2 more
doaj   +1 more source

Potential for cellular stress response to hepatic factor VIII expression from AAV vector

Molecular Therapy: Methods & Clinical Development, 2016
Hemophilia A and B are coagulation disorders resulting from the loss of functional coagulation factor VIII (FVIII) or factor IX proteins, respectively.
Irene Zolotukhin, David M Markusic, Brett Palaschak, Brad E Hoffman, Meera A Srikanthan, Roland W Herzog   +5 more
doaj   +1 more source

Pre‐Regenerative Endothelial Cells Empowered by Antler Blood Exosomes Orchestrate Peripheral Nerve Repair

Advanced Science, EarlyView.
The pre‐regenerative vascular niche (PVN) is essential for nerve repair, yet its endothelial blueprint remains unclear. We identified angiogenic ECs (AECs) as the dominant pre‐regenerative subset and found that antler blood–derived exosomes (AB‐EXO) promote repair via IMP3.
Jinsheng Huang, Qiang Chen, Aihua Jin, Xiaoqi Yang, Hui Zhou, Weiquan Gong, Jin Cheng, Jun Dai, Minjin Wang, Meihua Piao, Yuanyi Wang, Nan Zhou   +11 more
wiley   +1 more source

Microglial Deubiquitinase OTUD7B Stabilizes STAT3 to Promote Neuroinflammation and Cognitive Decline in Alzheimer's Disease

Advanced Science, EarlyView.
. ABSTRACT Neuroinflammation driven by microglial activation is a defining feature of Alzheimer's disease (AD), yet the molecular mechanisms sustaining this proinflammatory state remain unclear. Here, we identify the deubiquitinase OTUD7B as a critical regulator of microglial activation and AD pathology.
Luyao Li, Hao Tang, Qin Yu, Yuyang Zhang, Binglu Shi, Ying Kong, Leiyu Xu, Jingjing Shao, Chenghong Hu, Lingyu She, Zhe Wang, Haiyi Chen, Aleksandr V. Samorodov, Gang Cao, Xia Zhao, Yi Wang   +15 more
wiley   +1 more source

TOLLIP Inhibits Psoriasis Progression via Suppressing PKM2‐Mediated Glycolysis in Keratinocytes

Advanced Science, EarlyView.
In this study, we identify TOLLIP as a critical regulator of psoriasis pathogenesis through its modulation of glycolytic metabolism. Our findings establish the TOLLIP‐PKM2‐glycolysis axis as a key mechanism linking metabolic reprogramming to psoriasis pathogenesis, and propose TOLLIP as a promising therapeutic target.
Xiuhuan Jiang, Guoying Miao, Na Shen, Qingxia Han, Xiaoru Han, Zichang Qiao, Kexin Feng, Zhuo Tan, Yaguang Wang, Xiuhua Liu, Chen Wu, Zhenzhen Yan   +11 more
wiley   +1 more source

Gm26550 Modulates Learning and Memory by Increasing IGF1 mRNA Expression and Stability in Nrf2−/− Mice

Advanced Science, EarlyView.
The present study shows that Nrf2 directly binds to the Gm26550 promoter, thereby activating Gm26550 transcription and increasing its expression. Mechanistically, Gm26550 promotes IGF1 expression by functionally antagonizing miR‐26a‐5p‐mediated repression and sequestering the RBP KHSRP, thereby enhancing hippocampal neuronal synaptic plasticity and ...
Hongfang Wang, Ziyao Wang, Dongyue Zuo, Zhaowen Su, Bowen Song, Jiamin Gao, Yizhou Zhang, Ruiting Zhao, Linjie Sun, Mengdi Li, Yirui Fan, Dandan Geng, Lei Wang   +12 more
wiley   +1 more source

In Situ Genetic Engineering Facilitates MMP‐9‐Responsive TIMP1 Release Through Proteolytic Cleavage to Boost Diabetic Wound Healing

Advanced Science, EarlyView.
This study develops an MMP‐9‐responsive protein release system that transforms resident cells into drug depots through in situ genetic engineering. Anchored to the membrane, the therapeutic protein TIMP1 is selectively released upon MMP‐9 cleavage.
Yuan Zhang, Jiani Deng, Man Yee Cheung, Tingting Fu, Xufeng Qi, Dongqing Cai, Jianmin Sun, Gang Lu, Peng Shi, Wai‐Yee Chan, Xu Li, Hui Zhao   +11 more
wiley   +1 more source