Results 161 to 170 of about 1,703,096 (348)

Precision therapies for genetic epilepsies in 2025: Promises and pitfalls

Epilepsia Open, EarlyView.
Abstract By targeting the underlying etiology, precision therapies offer an exciting paradigm shift to improve the stagnant outcomes of drug‐resistant epilepsies, including developmental and epileptic encephalopathies. Unlike conventional antiseizure medications (ASMs) which only treat the symptoms (seizures) but have no effect on the underlying ...
Shuyu Wang, Emilio Perucca, Samuel F. Berkovic, Piero Perucca   +3 more
wiley   +1 more source

Targeting MGAT4A‐Mediated N‐Glycosylation as a Therapeutic Strategy to Inhibit Glioblastoma Stem Cell Invasion

Exploration, EarlyView.
ABSTRACT Glioblastoma (GBM) is the most aggressive type of primary brain tumor, and its invasive properties contribute significantly to incomplete surgical resection and tumor recurrence. Despite extensive clinical efforts to mitigate GBM invasion, targeted therapies against GBM stem cells (GSCs), which drive invasion, remain incompletely understood ...
Rui Niu, Meichen Liu, Qingshan Yang, Jinlong Yin   +3 more
wiley   +1 more source

Ultrasmall nanoparticles for co-delivery of antisense oligonucleotides targeting miR-21 and miR-210 to treat glioblastoma

Journal of Nanobiotechnology
Glioblastoma (GBM) is the most common and aggressive type of brain tumour, with less than 5% of patients surviving more than 5 years. Despite decades of research to understand the underlying pathophysiological causes, it has witnessed very slow progress ...
Ravi Raj Singh, Ritu Kulshreshtha, Amirali Popat   +2 more
doaj   +1 more source

When Rare Is Not Small: Amyotrophic Lateral Sclerosis Initiatives and Therapy

Exploration, EarlyView.
In the precision‐medicine era, rare diseases must not be sidelined in translational infrastructure. The Mr. Cai Lei—led “Ice‐Breaking Team” turns an amyotrophic lateral sclerosis patient community into a sustainable ecosystem, realigning philanthropy, data, and research and development to reshape rare‐disease pipelines and guide precision therapies ...
Yang Liu, Xue Xia, Yingfang Chen, Haoying Yang, Xiaochen Chen, Lei Cai, Bingyang Shi   +6 more
wiley   +1 more source

Exosome-mediated delivery of antisense oligonucleotides targeting α-synuclein ameliorates the pathology in a mouse model of Parkinson's disease

Neurobiology of Disease, 2020
Jiaolong Yang, Shilin Luo, Ji-Chun Zhang, Ting Yu, Zhihui Fu, Yongfa Zheng, Ximing Xu, Chaoyang Liu, Mingxia Fan, Zhentao Zhang   +9 more
semanticscholar   +1 more source

Antisense nucleic acids—tough delivery [PDF]

Nature Reviews Urology, 2013
openaire   +1 more source

Next‐generation sequencing in newborn screening: Current status, challenges, and future perspectives

Pediatric Investigation, EarlyView.
Newborn screening shifts from biochemical to genetic methods. Global exploration is promising but requires overcoming challenges and international collaboration to optimize implementation. ABSTRACT Newborn screening (NBS) is a key public health intervention that improves children's health outcomes by enabling precise intervention through the early ...
Zhelan Huang, Wenhao Zhou
wiley   +1 more source

Treatment of pediatric epilepsy

Pediatric Investigation, EarlyView.
Anti‐seizure medications are the first‐line treatment for the vast majority of children with epilepsy, with the advantages of non‐invasive wide adaptability. Surgery is the main treatment for drug‐resistant epilepsy and lesion‐related epilepsy, which can cure some cases of epilepsy in children. A ketogenic diet is often an add‐on therapy.
Junxiao Li, Tinghong Liu, Chang Liu, Jie Deng, Shijie Wu, Suhui Kuang, Xiaotong Li, Zhirong Wei, Shuli Liang   +8 more
wiley   +1 more source

RNA degradomics and proteomics reveal the mechanism of dsProsβ1‐mediated proteasome targeting in Psylliodes chrysocephala

Pest Management Science, EarlyView.
RNA degradomics revealed dsProsβ1‐derived siRNA‐mediated mRNA cleavage events, mainly at uracil‐guanine and adenine‐adenine pairs. Proteasome inhibition via dsProsβ1 increased mitochondrial and cytoskeletal proteins while reducing translation‐related and mRNA‐binding proteins.
Doga Cedden, Gözde Güney, Michael Rostás, Stefan Scholten   +3 more
wiley   +1 more source

Gene‐specific double‐stranded RNAs induce mortality in the South African mealybug Delottococcus aberiae

Pest Management Science, EarlyView.
Gene‐specific double‐stranded RNAs effectively silence essential genes in the invasive citrus pest Delottococcus aberiae, leading to significant mortality through both microinjection and oral delivery. These results provide proof of concept for RNA interference‐based biopesticides as selective and sustainable tools for citrus integrated pest management.
Carolina Gallego‐Giraldo, Alberto Urbaneja, Omar Ruíz‐Rivero, Verónica Aragonés, Marie Guijarro‐Guijarro, José‐Antonio Daròs, Meritxell Perez‐Hedo   +6 more
wiley   +1 more source