Results 41 to 50 of about 87,753 (269)

C3- and CR3-dependent microglial clearance protects photoreceptors in retinitis pigmentosa

Journal of Experimental Medicine, 2019
Silverman et al. demonstrate that complement activation features prominently in retinitis pigmentosa in close association with activated microglia. This response mediates adaptive neuroprotection for photoreceptors by facilitating a C3-CR3–dependent ...
Sean M. Silverman, Wenxin Ma, Xu Wang, Lian Zhao, W. Wong   +4 more
semanticscholar   +1 more source

Structure‐Guided Engineering of a Cas12i Nuclease Unlocks Near‐PAMless Genome Editing

Advanced Science, EarlyView.
CRISPR‐Cas nucleases are limited by PAM requirements, restricting genome accessibility. Structure‐guided engineering of the compact Cas12i nuclease SF01 produced three variants with near‐PAMless, enabling efficient editing at diverse 5'‐NNTN‐3' sites. These nucleases expand the editable portion of the human genome more than fourfold, enabling efficient
Qitong Chen, Hanlin Gou, Chao Xu, Sihan Wang, Huitao Zhang, Minglei Song, Mengge Wang, Xingkun Ji, Xiaofei Wei, Yuanyan Tan, Hehua Quan, Pengyu Luo, Hanyu Shou, Pengpeng Liu, Yafeng Liang, Jian‐Kang Zhu   +15 more
wiley   +1 more source

New and emerging technologies for the treatment of inherited retinal diseases: a horizon scanning review. [PDF]

, 2015
The horizon scanning review aimed to identify new and emerging technologies in development that have the potential to slow or stop disease progression and/or reverse sight loss in people with inherited retinal diseases (IRDs).
Michaelides, M, Moore, AT, Simpson, S, Smith, J, Ward, D   +4 more
core  

Rpgrip1 is required for rod outer segment development and ciliary protein trafficking in zebrafish [PDF]

, 2017
The authors would like to thank the Royal Society of London, the National Eye Research Centre, the Visual Research Trust, Fight for Sight, the W.H. Ross Foundation, the Rosetrees Trust, and the Glasgow Children’s Hospital Charity for supporting this work.
A Eblimit, A Hameed, AF Wright, AI Hollander den, B Kennedy, BW Draper, C Insinna, C Turney, CA Murga-Zamalloa, CM Louie, CS Mellersh, D Deretic, D Mustafi, DH Hong, EM Morrow, F Liu, G Stearns, G Ying, H Patil, H Wang, HH Arts, J Kim, J Wang, J Won, JC Booij, JE Westfall, JM Corless, JP Boylan, KL Cerveny, KL Coene, L Sang, MM Humphries, MV Nachury, O Rinner, OL Moritz, P Castagnet, PD Alfinito, PH Balm, R Bachmann-Gagescu, R Roepman, R Roepman, S Gerber, SC Neuhauss, SE Brockerhoff, SR Patnaik, TA Mavlyutov, TP Dryja, V Traverso, VS Lopes, X Shu, X Shu, X Shu., Y Wada., Y Zhao, YV Makhankov   +54 more
core   +4 more sources

CD147/Basigin: From Integrative Molecular Hub to Translational Therapeutic Target

Advanced Science, EarlyView.
This review conceptualizes CD147 as a fundamental “Energy‐Structure Coupler,” physically bridging metabolic flux (via MCTs) with morphogenetic plasticity (via integrins/MMPs) to drive cancer, infection, and autoimmunity. Addressing the “specificity paradox” that limits current translation, the authors chart a strategic roadmap—spanning logic‐gated ...
Xiang‐Min Yang, Huijie Bian, Zhi‐Nan Chen   +2 more
wiley   +1 more source

Therapeutic potential of co-enzyme Q10 in retinal diseases [PDF]

, 2017
Coenzyme Q10 (CoQ10) plays a critical role in mitochondrial oxidative phosphorylation by serving as an electron carrier in the respiratory electron transport chain.
Marcheggiani, Fabio, Reilly, James, Shu, Xinhua, Tiano, Luca, Tohari, Ali Mohammad, Zhang, Xun, Zhou, Xinzhi   +6 more
core   +1 more source

Implantable Drug Delivery Systems for Skeletal Muscles and Eyes

Advanced NanoBiomed Research, EarlyView.
This review highlights the different types of recent implantable drug delivery systems (IDDS) fabricated for a use with skeletal muscles, and with eyes. It presents the developments already made and the current research directions, showing the evolution of IDDS and their great diversity.
Serge Ostrovidov, Murugan Ramalingam, Hongkai Wu, Toshinori Fujie, Yukinari Nakamura, Takeshi Hori, Yuji Nashimoto, Xuetao Shi, Hirokazu Kaji   +8 more
wiley   +1 more source

Gezielte Modulation des spliceosomalen Proteins USP39 durch allosterische Liganden und PROTAC‐induzierte Degradation

Angewandte Chemie, EarlyView.
Proteolysis‐targeting Chimeras (PROTACs) ermöglichen die gezielte Degradation bislang als „undruggable“ geltende Proteine über das zelluläre Ubiquitin–Proteasom‐System. In dieser Studie identifizieren Schäfer et al. thiazolbasierte niedermolekulare Liganden, die allosterisch an die Zinkfinger‐Domäne der Ubiquitin‐spezifischen Protease 39 (USP39) binden
Daniel Schäfer, Cristian Prieto‐Garcia, Jianhui Wang, Marcel Heinz, Vigor Matkovic, Pavel Kielkowski, Sebastian Hasselbeck, Varun Jayeshkumar Shah, Stefan Knapp, Gerhard Hummer, Ivan Dikic, Xinlai Cheng   +11 more
wiley   +1 more source

ARL3 mutations cause Joubert syndrome by disrupting ciliary protein composition [PDF]

, 2018
Joubert syndrome (JBTS) is a genetically heterogeneous autosomal recessive neurodevelopmental ciliopathy. We investigated further the underlying genetic etiology of Joubert syndrome by studying two unrelated families in whom JBTS was not associated ...
Alhashem, Amal, Alkanderi, Sumaya, Alkuraya, Fowzan S., Cairns, George, Edwards, Noel, Elmaghloob, Yasmin, Ewida, Nour, Ismail, Shehab, Miles, Colin G., Molinari, Elisa, Ramsbottom, Simon A., Rice, Sarah J., Sammut, Veronica, Sayer, John A., Shaheen, Ranad, Srivastava, Shalabh, Steel, David H., Stephen, Louise A., White, Kathryn   +18 more
core   +1 more source

Targeting the Spliceosomal Protein USP39 Through Allosteric Ligands and PROTAC‐Induced Degradation

Angewandte Chemie International Edition, EarlyView.
Proteolysis‐targeting chimeras (PROTACs) enable degradation of proteins previously considered undruggable by harnessing the cellular ubiquitin–proteasome system. In this study, Schäfer et al. identify thiazole‐based small molecules that allosterically bind the zinc finger domain of ubiquitin‐specific protease 39 (USP39), a non‐enzymatic scaffold ...
Daniel Schäfer, Cristian Prieto‐Garcia, Jianhui Wang, Marcel Heinz, Vigor Matkovic, Pavel Kielkowski, Sebastian Hasselbeck, Varun Jayeshkumar Shah, Stefan Knapp, Gerhard Hummer, Ivan Dikic, Xinlai Cheng   +11 more
wiley   +1 more source