Results 111 to 120 of about 131,722 (303)

Decoding Undesirable Inflammatory Responses of Nucleic Acid‐Delivering Lipid Nanoparticles

Advanced Science, EarlyView.
Lipid nanoparticles (LNPs) enable efficient nucleic acid delivery, but their immunogenicity is a double‐edged sword. This review explores LNP‐driven innate and adaptive immunity, covering lipid components, endosomal escape, and nucleic acid sensing.
Ruimin Hu, Yin Dou, Chenwen Li, Yuying Mu, Huiying Ouyang, Wenhao Shen, Jianxiang Zhang   +6 more
wiley   +1 more source

Insights into DNA platination within unusual structural settings [PDF]

, 2011
2D [1H, 15N] HSQC NMR spectroscopy has been used to monitor reaction and product formation between [Pt(15NH3)2I2] and nucleic acids possessing irregular topologies and containing site specific phosphorothioate substitution in the phosphodiester backbone.
Harvie, Stephanie, Parkinson, John A, Wilson, Owen   +2 more
core   +2 more sources

ZDHHC18‐Mediated Palmitoylation of ORF3a Promotes SARS‐CoV‐2 Pathogenesis by Antagonizing TRIM16‐Mediated Ubiquitination and Proteasomal Degradation

Advanced Science, EarlyView.
Palmitoylation by ZDHHC18 blocks ORF3a K27‐linked ubiquitination mediated by TRIM16, thereby preventing its proteasomal degradation and strengthening viral pathogenesis. Targeting palmitoylation through a pharmacological inhibitor (2‐BP), a competitive inhibitory peptide (OPIP), or adenovirus‐mediated knockdown of ZDHHC18 expression presents a ...
Sidi Yang, Kun Li, Lihong Liu, Linsen Zeng, Qifeng Deng, Jiacheng Huang, Xiaoran Dong, Xin Wang, Jianwei Liang, Hongchao Liu, Hong Peng, Yuxin Lin, Xiaolu Xie, Yuzhen Ye, Tiefeng Xu, Zhaohuan Wang, Chun‐Mei Li, Deyin Guo   +17 more
wiley   +1 more source

Antisense Oligonucleotide Gene Therapy for Neuromuscular Disorders

University of Ottawa Journal of Medicine, 2018
Antisense oligonucleotides (ASOs) are synthetic, single-stranded DNA molecules that can bind to specific mRNA sequences and alter protein expression.
Ryan Gotesman
doaj   +1 more source

Evidence for regulated expression of Telomeric Repeat-containing RNAs (TERRA) in parasitic trypanosomatids [PDF]

, 2017
The Telomeric Repeat-containing RNAs (TERRA) participate in the homeostasis of telomeres in higher eukaryotes. Here, we investigated the expression of TERRA in Leishmania spp.
Damasceno, Jeziel D., Silva, Gabriel L.A., Tosi, Luiz R.O., Tschudi, Christian   +3 more
core   +1 more source

Therapy for Myhre Syndrome: Goals, Misconceptions, and Current Agents

American Journal of Medical Genetics Part C: Seminars in Medical Genetics, EarlyView.
ABSTRACT Myhre Syndrome (MYHRS, MIM #139210) is a rare, multisystem connective tissue disorder caused by recurrent heterozygous gain‐of‐function pathogenic variants in the SMAD4 gene, a key player in TGF‐β signaling and a regulator of extracellular matrix homeostasis.
Alessandro De Falco, Alfonso Manuel D'Alessio, Nicola Brunetti‐Pierri   +2 more
wiley   +1 more source

Retro-1-Oligonucleotide Conjugates. Synthesis and Biological Evaluation

Molecules, 2019
Addition of small molecule Retro-1 has been described to enhance antisense and splice switching oligonucleotides. With the aim of assessing the effect of covalently linking Retro-1 to the biologically active oligonucleotide, three different derivatives ...
Jordi Agramunt, Enrique Pedroso, Silvia M. Kreda, Rudolph L. Juliano, Anna Grandas   +4 more
doaj   +1 more source

High Prevalence of SOD1 Pathogenic Variants in the UK Biobank: Implications for Early Intervention in Amyotrophic Lateral Sclerosis

Annals of Neurology, EarlyView.
Objective SOD1 is the second most frequently mutated gene in European patients with amyotrophic lateral sclerosis (ALS). Given the recent authorization of SOD1‐targeted antisense oligonucleotides for SOD1‐ALS, prompt screening for SOD1 mutations in patients with ALS patients is highly recommended.
Delia Gagliardi, Chiara Villella, Matteo Zanovello, Virginia Iacobelli, Stefania Corti, Giacomo Pietro Comi, Pietro Fratta, Henry Houlden, Arianna Tucci, Dario Ronchi   +9 more
wiley   +1 more source

Longitudinal Phenotypic Trajectories in GNAO1‐Related Disorders: Defining Disease Progression and Clinical Profiles

Annals of Neurology, EarlyView.
Objective Pathogenic variants in GNAO1 cause a spectrum of epilepsy, movement disorders, and developmental impairment. Clinical heterogeneity complicates prognosis and therapeutic development. We present the first longitudinal natural history study of GNAO1‐related disorders (GNAO1‐RD) to delineate phenotypic trajectories. Methods Sixty‐six individuals
Jana Domínguez‐Carral, Ana María Domínguez Cobo, Sol Balsells, Anna Aguilar‐Ros, Chu‐Ting Chang, William G. Ludlam, Kathryn Yang, Katerina Bernardi, Micaela Chinigioli, Ainara Salazar‐Villacorta, Veronica Di Pisa, Nuria Lamagrande‐Casanova, Elena González‐Alguacil, Beatriz De la Casa‐Fages, Akihisa Okumura, Josefina Rodríguez, Ayush Agarwal, Daniela Muñoz‐Chesta, Carolina Reynoso‐Osnayo, Amy Lin, Brahim Tabarki, Jobaida Parvin, Adolfo Alberto Gallo, Andreia Forno, Fabian Maass, Johnny Montiel Blanco, Salomé Nasif, Elizabeth Jennions, Jorge Luis Ramón‐Gómez, Helene Verhelst, Juan José Nieto Barceló, Dunja Čokolić Petrović, Luz Victoria García Ruiz, Christoph van Riesen, Paulo Rego Sousa, Maria del Pilar Massaro Sanchez, Husnea Ara Khan, Wejdan Hakami, Jennifer Friedman, Iván Espinoza‐Quinteros, Monica Troncoso, Divyani Garg, Micaela Pauni, Hirokazu Kurahashi, María Concepción Miranda‐Herrero, Anna Duat‐Rodriguez, Luca Soliani, Manju A. Kurian, Angeles Schteinschnaider, Siddharth Srivastava, Darius Ebrahimi‐Fakhari, Kirill A. Martemyanov, Juan Darío Ortigoza‐Escobar   +52 more
wiley   +1 more source

XASH-3, a novel Xenopus achaete-scute homolog, provides an early marker of planar neural induction and position along the mediolateral axis of the neural plate [PDF]

, 1993
We have isolated a novel Xenopus homolog of the Drosophila achaete-scute genes, called XASH-3. XASH-3 expression is neural specific and is detected as early as stage 11 1/2, making it one of the earliest markers of neural induction so far described ...
Anderson, David J., Bars, Julie, Collazo, Andres, Shih, John, Zimmerman, Kathryn   +4 more
core