Results 111 to 120 of about 5,628,138 (356)

Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus. [PDF]

, 2013
Usher 1 patients are born profoundly deaf and then develop retinal degeneration. Thus they are readily identified before the onset of retinal degeneration, making gene therapy a viable strategy to prevent their blindness.
Boye, S, Boye, SE, Chiodo, V, Dyka, F, Fofo, H, Hauswirth, WW, Lopes, VS, Louie, CM, Williams, DS   +8 more
core   +1 more source

Aptamer‐Based Delivery Systems for VEGF and NGF Modulation in Ocular Therapies

Advanced Healthcare Materials, EarlyView.
Aptamer‐based delivery systems targeting NGF and VEGF hold significant potential for precise and sustained treatment of complex diseases affecting both the anterior and posterior segments of the eye. This review critically summarizes current strategies for NGF and VEGF delivery as well as VEGF sequestration, providing a clinical perspective on how ...
Nadine Best, Gizem Karabiyik, Gerd Geerling, Sarah Barbara Zwingelberg, Gregor Lang, Martin Humenik   +5 more
wiley   +1 more source

The Leishmania major BBSome subunit BBS1 is essential for parasite virulence in the mammalian host [PDF]

, 2013
Bardet–Biedl syndrome (BBS) is a human genetic disorder with a spectrum of symptoms caused by primary cilium dysfunction. The disease is caused by mutations in one of at least 17 identified genes, of which seven encode subunits of the BBSome, a protein ...
Doehl, Johannes, Farrant, Katie, Hodgkinson, Michael R., Paape, Daniel, Price, Helen P., Smith, Deborah F., Stark, Meg   +6 more
core   +1 more source

Centrin2 regulates CP110 removal in primary cilium formation [PDF]

Journal of Cell Biology, 2015
Primary cilia are antenna-like sensory microtubule structures that extend from basal bodies, plasma membrane–docked mother centrioles. Cellular quiescence potentiates ciliogenesis, but the regulation of basal body formation is not fully understood. We used reverse genetics to test the role of the small calcium-binding protein, centrin2, in ciliogenesis.
Prosser, Suzanna L., Morrison, Ciaran G.
openaire   +2 more sources

Bioengineered 3D hPSC‐Cholangiocyte Ducts With Physiological Signals for Biliary Disease Modeling

Advanced Healthcare Materials, EarlyView.
Tian and colleagues generated a bioengineered bile duct from human pluripotent stem cell (hPSC)‐derived intrahepatic cholangiocytes within a high‐throughput, 384‐well platform to systematically examine the influence of biliary physiological signals including fluid flow, stromal cells and bile acids, and models intrahepatic biliary disease progression ...
Britney Tian, Mina Ogawa, Manami Kondo, Gabrielle Langeveld, Ling Jun Huan, Feng Zhang, Andrew Hollinger, Sara Deir, Christine Bear, Boyang Zhang, Shinichiro Ogawa   +10 more
wiley   +1 more source

The centrosomal deubiquitylase USP21 regulates Gli1 transcriptional activity and stability [PDF]

, 2016
USP21 is a centrosome-associated deubiquitylase (DUB) that has been implicated in the formation of primary cilia - crucial organelles for the regulation of the Hedgehog (Hh) signaling pathway in vertebrates. Here, we identify KCTD6 - a cullin-3 E3-ligase
Bertsoulaki, Erithelgi, Clague, Michael J., Cucchi, Danilo, DE SMAELE, Enrico, Heride, Claire, Rigden, Daniel J., Urbé, Sylvie   +6 more
core   +1 more source

Disruption of Dhcr7 and Insig1/2 in cholesterol metabolism causes defects in bone formation and homeostasis through primary cilium formation

Bone Research, 2020
Human linkage studies suggest that craniofacial deformities result from either genetic mutations related to cholesterol metabolism or high-cholesterol maternal diets.
A. Suzuki, Kenichi Ogata, Hiroki Yoshioka, Junbo Shim, C. Wassif, F. Porter, J. Iwata   +6 more
semanticscholar   +1 more source

Respiratory Organ‐on‐a‐Chip for Disease Modeling: From Architecture to Functional Integration

Advanced Healthcare Materials, EarlyView.
Respiratory organ‐on‐a‐chip (ROC) models capture key mechanical and cellular cues of the human respiratory system, enabling quantitative dissection of disease mechanisms. This review links ROC architectures to disease modeling, functional integration, and commercialization, and proposes a decision framework that aligns model complexity with mechanistic
Jinzhuo Hu, Yongjie Tang, Sidi Liu, Tao Xu, Qian Liu   +4 more
wiley   +1 more source

A Proximity Mapping Journey into the Biology of the Mammalian Centrosome/Cilium Complex

Cells, 2020
The mammalian centrosome/cilium complex is composed of the centrosome, the primary cilium and the centriolar satellites, which together regulate cell polarity, signaling, proliferation and motility in cells and thereby development and homeostasis in ...
Melis Dilara Arslanhan, Dila Gulensoy, Elif Nur Firat-Karalar   +2 more
doaj   +1 more source

Transmission Electron Microscopy Tilt-Series Data from In-Situ Chondrocyte Primary Cilia

Data, 2021
The primary cilium has recently become the focus of intensive investigations into understanding the physical structure and processes of eukaryotic cells.
Michael J. Jennings, Timothy C. A. Molteno, Robert J. Walker, Jennifer J. Bedford, John P. Leader, Tony Poole   +5 more
doaj   +1 more source