Results 201 to 210 of about 2,820,603 (281)

Long-term experience with gene augmentation therapy in patients with inherited retinal disease associated with biallelic mutations in <b><i>RPE65</i></b>. [PDF]

Med Genet
Lorenz B.
europepmc   +1 more source

MZF1 Inhibits TGFβ‐1/SMAD Signaling by Transcriptionally Activating SnoN to Augment Tumorigenic Potential in Colon Cancer

Advanced Science, EarlyView.
This study identifies MZF1 as a core regulator of colon cancer via SnoN‐mediated TGF‐β suppression, proposes 10Panx Acetate as a targeted inhibitor, and validates MZF1/SnoN as prognostic markers for aggressive disease. ABSTRACT Colon cancer progression is associated with transcriptional and signaling abnormalities.
Siqi Sheng, Mengxi Huang, Yiting Wang, Hongyu Wang, Haimei Feng, Xiaoming Bai, Jie Chen, Ke Xu, Xiaoyuan Chu, Zengjie Lei   +9 more
wiley   +1 more source

Fine-tuning FAM161A gene augmentation therapy to restore retinal function. [PDF]

EMBO Mol Med
Arsenijevic Y, Chang N, Mercey O, El Fersioui Y, Koskiniemi-Kuendig H, Joubert C, Bemelmans AP, Rivolta C, Banin E, Sharon D, Guichard P, Hamel V, Kostic C.   +12 more
europepmc   +1 more source

Compact 9dBEs Enable Efficient and Precise Genome Editing in Mammalian Cells and In Vivo

Advanced Science, EarlyView.
As a compact type II‐D system, the Cas9d‐based platform holds great potential for in vivo applications. Through rational engineering, its derived base editors (9dBEs) enable efficient disease modeling while facilitating single‐vector AAV delivery for in vivo genome editing. These miniature tools offer a robust strategy for basic research and biomedical
Qingquan Xiao, Zhijin Tian, Luqi Weng, Kun Xu, Dingyi Han, Zhaowei Wu, Junnan Li, Shangpu Li, Qijing Zhang, Xiaohui Zhao, Guoling Li, Yinghui Wei, Zhen Gu   +12 more
wiley   +1 more source

Streptococcal Mannose Phosphotransferase System Component IID Is a Novel RANK‐Binding Osteoclastogenic Factor

Advanced Science, EarlyView.
Streptococcal mannose phosphotransferase system component IID (Man‐PTSIID) is identified as a novel RANK‐binding osteoclastogenic factor. By directly binding to RANK and activating NF‐κB independently of TLR2, Man‐PTSIID drives osteoclastogenesis and inflammatory bone destruction, uncovering an unexpected microbial mechanism underlying streptococcal ...
Chaeyeon Park, Yeongkag Kwon, Yeonjin Lim, Jeong Woo Park, Hyun Jung Ji, Dongwook Lee, Jueun Lee, Sung‐Ho Yun, Sungho Jeong, Ho Seong Seo, Kun Cho, Cheol‐Heui Yun, Ok‐Jin Park, Seung Hyun Han   +13 more
wiley   +1 more source

Research Models and Gene Augmentation Therapy for CRB1 Retinal Dystrophies. [PDF]

Front Neurosci, 2020
Boon N, Wijnholds J, Pellissier LP.
europepmc   +1 more source

Natural‐Inspired Engineered Microalgae With Selenium Enrichment Enabled Potent Anti‐Tumor Immunity

Advanced Science, EarlyView.
Se‐Ssp was engineered as a potent immunomodulator for activating innate immunity via the TLR and cGAS‐STING pathways, therefore converting “cold” tumors into “hot” tumors to achieve high efficacy and long‐term therapeutic outcomes. ABSTRACT Immunotherapy has shed light on clinical oncology for several decades.
Yuqi Yang, Xiaowei Cai, Xiaoyan Zhong, Tian Qiu, Qinwei Lu, Jiahao Qin, Fei Gong, Yecheng Huang, Chenyu Jiang, Youdong Chen, Yuan Cheng, Liang Cheng   +11 more
wiley   +1 more source

Assessing the efficacy of Alpha₁-Proteinase inhibitor (human) augmentation therapy for Alpha₁-Antitrypsin deficiency - Related emphysema: Challenges and opportunities. [PDF]

Heliyon
Pierce LR.
europepmc   +1 more source

Inorganic Nanomaterials can Promote or Inhibit Tumor Metastasis Depending on Tumor Microenvironment Composition

Advanced Science, EarlyView.
As engineered nanomaterials gain prominence in cancer imaging and therapy, understanding their impact on tumor progression becomes increasingly critical. We demonstrate that gold and silica‐coated gold nanomaterials can either suppress or enhance breast cancer metastasis through distinct tumor microenvironment interactions, while identifying ...
Kiana Buttiens, Xiaoqi Zheng, Xueqi Fan, Mukaddes Izci, Christy Maksoudian, Irati Perez Gilabert, Tianjiao Chu, Filipa Goncalves, Jonas Dehairs, Johan Swinnen, Bella B. Manshian, Stefaan J. Soenen   +11 more
wiley   +1 more source

Origin of Enhanced Efficiency and Reduced Roll‐Off in Organic Light‐Emitting Diodes Based on Pt(II) Complexes

Advanced Science, EarlyView.
Controlled Pt–Pt fine structure in square‐planar Pt(II) complexes governs metal–metal‐to‐ligand charge‐transfer (MMLCT) state formation, exciton dynamics, and organic light‐emitting diode performance. Short Pt–Pt contacts in Pt(fppz)2 enable coherent metal–metal coupling, sub‐microsecond emission and an external quantum efficiency of 29%, whereas ...
Atul Shukla, Sarah K. M. McGregor, Julian A. Steele, Innes K. Gale, Kiyoshi Miyata, Monirul Hassan, Christian McDonald, Romain J. Lepage, Eduardo Solano, Pilankatta K. Ramya, Cherumuttathu H. Suresh, Takumi Ehara, Chihaya Adachi, Ken Onda, Shih‐Chun Lo, Ebinazar B. Namdas   +15 more
wiley   +1 more source