Results 161 to 170 of about 6,268,347 (286)

Mutant KRAS‐m6A Epitranscriptome Axis Promotes Colorectal Cancer and is a Therapeutic Target

Advanced Science, EarlyView.
Mutant KRAS‐m6A axis promotes colorectal cancer (CRC) progression. Mutant KRAS stabilizes METTL3, leading to increased m6A‐modified BCL9L mRNA and translation of BCL9L protein. BCL9L in turn mediates TGF‐β secretion to induce differentiation of Treg and an immunosuppressive microenvironment.
Danyu Chen, Pingmei Huang, Ruizhi Tao, Fenfen Ji, Qinyao Wei, Lok Hin Ko, Lianxin Zhou, Huarong Chen, Alvin Cheung, Wei Kang, Ko Fai To, Jun Yu, Chi Chun Wong   +12 more
wiley   +1 more source

Clinical response to systemic AAV gene therapy in a large animal model of late-stage lysosomal storage disease. [PDF]

Gene Ther
Hunter JE, Molony CM, Clarke DL, Panek W, Vite CH, Chawla S, Poptani H, Wolfe JH.   +7 more
europepmc   +1 more source

SIRT7‐Mediated H2BK120 Succinylation Drives Aberrant Mitophagy in Sepsis‐Associated Cognitive Dysfunction

Advanced Science, EarlyView.
Sepsis triggers hippocampal SIRT7 loss, promoting histone H2B succinylation and activation of the PD‐1/PD‐L1–PINK1 axis. This metabolic‐epigenetic cascade drives aberrant mitophagy and neuronal injury, ultimately causing cognitive deficits. Targeting SIRT7‐dependent succinylation offers a potential strategy to protect brain function during sepsis ...
Na Meng, Jiateng Zhou, Xiaoyu Guo, Ting Hong, Xuelian Li, Xingyu Wei, Chanhua Zhang, Xixue Zhang, Songbin Liu, Yulin Zhang, Liangfang Yao, Lina Huang, Weidong Gu   +12 more
wiley   +1 more source

New insights in pediatric metabolic medicine: pathogenesis of cholesterol biosynthesis defects and innovative therapies for Pompe disease [PDF]

, 2007
Rossi, Emiliano
core   +1 more source

Naturally occurring lysosomal storage disease consistent with neuronal ceroid lipofuscinosis in a group of 5 related captive rhesus macaques (<i>Macaca mulatta</i>). [PDF]

Vet Pathol
Olstad K, Pastenkos G, Barrera-Zarate J, Kanthaswamy S, Sammak R, Lemoy MJ.   +5 more
europepmc   +1 more source

Lysosomal storage disease spectrum in nonimmune hydrops fetalis: a retrospective case control study. [PDF]

Prenat Diagn, 2020
Al-Kouatly HB, Felder L, Makhamreh MM, Kass SL, Vora NL, Berghella V, Berger S, Wenger DA, Luzi P.   +8 more
europepmc   +1 more source

Multifunctional Nanovaccine‐Radiotherapy Synergy Drives Anti‐Tumor Immunity via Multi‐Cascade Cell Death for Pancreatic Cancer Therapy

Advanced Science, EarlyView.
Machine‐learning‐driven transcriptomic analysis identifies FSTL3 as a key tumor‐microenvironment regulator in desmoplastic pancreatic adenocarcinoma. A cRGD‐modified liposome co‐loading gemcitabine and siFSTL3 serves as an irradiation‐triggered in situ nanovaccine. It synergizes with radiotherapy to induce ferroptosis and immunogenic cell death through
Chengyi Huang, Yushu Han, Xiang Liu, Chuanhao Wang, Xiaofei Zhu, Di Chen, Yangyang Gong, Liang Chen, Wenjuan Chen, Zhenyuan Miao, Bufu Tang, Huojun Zhang   +11 more
wiley   +1 more source

Self‐Propelled HPB@Lip@AB Nanomotors Ameliorate Dry Eye Disease

Advanced Science, EarlyView.
A self‐propelled HPB@Lip@AB nanomotor system is developed for dry eye disease (DED) therapy, facilitating rapid penetration across ocular surface barriers while integrating hydrogen therapy with multi‐enzyme‐like antioxidant activity. The nanoplatform restores mitochondrial function, suppresses oxidative stress, inflammation, and apoptosis, and ...
Jing Li, Xi Wan, Xinru Su, Zechuan Li, Yue Wang, Ying Wang, Ruofei Li, Huangzhao Wei, Jianbin Zhang, Shuai Wang   +9 more
wiley   +1 more source

Choroid plexus-targeted viral gene therapy for alpha-mannosidosis, a prototypical neurometabolic lysosomal storage disease. [PDF]

Hum Mol Genet
Choi EY, Wolfe JH, Kaler SG.
europepmc   +1 more source

Lysosomal storage diseases

Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease, 1995
openaire   +2 more sources