Results 121 to 130 of about 41,178 (242)

Switching to Glycerol Phenylbutyrate in 48 Patients with Urea Cycle Disorders: Clinical Experience in Spain. [PDF]

J Clin Med, 2022
Martín-Hernández E, Quijada-Fraile P, Correcher P, Meavilla S, Sánchez-Pintos P, de Las Heras Montero J, Blasco-Alonso J, Dougherty L, Marquez A, Peña-Quintana L, Cañedo E, García-Jimenez MC, Moreno Lozano PJ, Murray Hurtado M, Camprodon Gómez M, Barrio-Carreras D, de Los Santos M, Del Toro M, Couce ML, Vitoria Miñana I, Morales Conejo M, Bellusci M.   +21 more
europepmc   +1 more source

Optimized Lipid Nanoparticles with Tail‐Modified Ionizable Lipids for Safer mRNA Delivery

Advanced Science, EarlyView.
Systematic engineering of hydrophobic tail architecture in vitamin B5‐derived ionizable lipids establishes a comprehensive structure–activity relationship framework for mRNA delivery. Combined lipidtail and formulation optimization identifies TM1‐OPT3‐C, a lipid nanoparticle platform that improves efficacy–safety balance through efficient mRNA delivery,
Seo‐Hyeon Bae, Jisun Lee, Jae‐Hun Ahn, Hosam Choi, Huijeong Choi, Jungmin Kim, Minho Kang, Ho Rim Oh, So Hyun Park, Eun Young Oh, Sanghyuk Jeon, Yeeun Lee, Yu‐Sun Lee, Na‐Young Lee, Hee‐Jin Bae, Jina Kwak, Jooil Kim, Nakyung Lee, Beomsun Kang, Ji‐Hye Kim, Eunsaem Song, Daesub Song, Hyunho Yoon, Sang‐Myeong Lee, Hyewon Youn, Kiyoun Lee, Byeong‐Cheol Kang, Jae‐Hwan Nam   +27 more
wiley   +1 more source

Self-Reported Health-Related Quality of Life (HRQoL) in Adults With Urea Cycle Disorders. [PDF]

J Inherit Metab Dis
Coughlin CR, Barber J, Murali CN, Members of the Urea Cycle Disorders Consortium (UCDC), Wilkening G.   +4 more
europepmc   +1 more source

Challenges in the diagnosis and management of urea cycle disorders in Romanian children. [PDF]

Med Pharm Rep, 2021
Pop TL, Grama A, Miclea D, Vulturar R, Benţa G, Grigore M, Simu C.   +6 more
europepmc   +1 more source

Neuron‐Targeted Exosomal Delivery of siRNA Against RIPK3 Slows Neurodegenerative Progression in Alzheimer's Disease

Advanced Science, EarlyView.
Engineered exosomal siRNA delivery platform enables systemic, neuron‐targeted RNA transport across the blood–brain barrier. Surface functionalization with rabies virus glycoprotein‐derived peptide facilitates receptor‐mediated transcytosis, achieving efficient cytosolic delivery and robust gene silencing. Targeting RIPK3 suppresses necroptosis, reduces
Chi Zhang, Jiaqi Zhang, Yuzhi Wang, Yaodong Wang, Peng Lin, Yanling Wang, Zixuan Tang, Jintai Yu, Qin Zhou, Feiyun Cui   +9 more
wiley   +1 more source

(Fe4N/GaN)@GC: A Bifunctional Electrocatalyst for High‐Performance Rechargeable Zinc‐Air Batteries

Advanced Science, EarlyView.
This work demonstrates that an unprecedented Fe4N/GaN heterostructure, stabilized by a graphitized carbon shell, induces interfacial charge redistribution and optimizes oxygen‐intermediate adsorption, enabling highly efficient bifunctional ORR/OER electrocatalysis for rechargeable zinc‐air batteries.
Xin‐Yuan Wei, Sai‐Sai Xie, Xia‐Li Ding, Yuan‐Qi Zhai, Sen Yu, Xiang‐Quan Hu, Rongqian Wu, Jintao Lu, Yan‐Zhen Zheng   +8 more
wiley   +1 more source

Health Outcomes of Patients with Distal Urea Cycle Disorders Detected by Newborn Screening: Data from the Spanish National Registry. [PDF]

Int J Neonatal Screen
Yahyaoui R, Quijada-Fraile P, Blasco-Alonso J, Vives I, Gil Ortega D, Couce ML, Sánchez-Pintos P, García Jiménez MC, Meavilla Olivas S, García Volpe C, de Los Santos Mercedes M, García-Cazorla Á, Felipe-Rucián A, Dougherty-de Miguel L, Morais López A, Bergua Martínez A, Andrade Guerrero JD, Stanescu S, Belanger A, Gil-Campos M, Comino Monroy MJ, Bellusci M, Pérez-Mohand P, Barrio-Carreras D, Pérez B, Martín-Hernández E.   +25 more
europepmc   +1 more source

Probiotic‐Derived Vesicles Rectally Delivered via Thermo‐Gelling Copolymer Promote Mucosal Healing and Reduce Inflammation in Ulcerative Colitis

Advanced Science, EarlyView.
A thermo‐responsive rectal delivery platform integrating probiotic‐derived extracellular vesicles within a hyaluronic acid‐based copolymer enables localized and sustained therapy for ulcerative colitis. The formulation effectively suppresses inflammation, promotes macrophage polarization toward a regenerative phenotype, restores epithelial barrier ...
Ayushi Mairal, Ubaid Tariq, Shreya Mehrotra, Saravanan Matheshwaran, Jan Marsal, Ashok Kumar   +5 more
wiley   +1 more source

Impact of long-term nitrogen scavenger therapy on clinical outcome in individuals with urea cycle disorders. [PDF]

Sci Rep
Posset R, Epp F, Garbade SF, Gleich F, Gropman AL, Nagamani SCS, Hoffmann GF, Kölker S, Zielonka M.   +8 more
europepmc   +1 more source

Direct replacement of oral sodium benzoate with glycerol phenylbutyrate in children with urea cycle disorders. [PDF]

JIMD Rep, 2022
Yeo M, Rehsi P, Dorman M, Grunewald S, Baruteau J, Chakrapani A, Footitt E, Prunty H, McSweeney M.   +8 more
europepmc   +1 more source