Results 151 to 160 of about 144,032 (247)

Enhanced splicing modulation by NMA-modified antisense oligonucleotides. [PDF]

Nucleic Acids Res
Ling K, Prakash TP, Yu J, Jackson M, Chun SJ, Bachmann G, Post N, Greenlee S, Soriano A, Hunyara JL, Norris DA, Jafar-Nejad P, Swayze EE, Bennett CF, Rigo F.   +14 more
europepmc   +1 more source

Enhanced CRISPR‐cas systems for genome editing and molecular diagnostics

Interdisciplinary Medicine, EarlyView.
This review systematically summarizes the major optimization strategies for CRISPR‐Cas systems, focusing on the engineering of Cas proteins, guide RNAs, reporter probes, and the incorporation of chemical additives. The aim is to enhance editing precision and diagnostic capability, thereby expanding the CRISPR system's potential for broader biomedical ...
Sheng Li, Han Li, Bojie Chen, Ling Jiang, Keyu Long, Jiaqi Lang, Xingxiang Pu, Erhu Xiong   +7 more
wiley   +1 more source

Half-Life as a Therapeutic Design Axis: Targeting Short-Lived lncRNAs With Antisense Oligonucleotides. [PDF]

IUBMB Life
Tani H.
europepmc   +1 more source

Precision Medicine in Neurodegeneration with Brain Iron Accumulation (NBIA) Disorders: An Update on Emerging Treatments

Movement Disorders Clinical Practice, EarlyView.
Abstract Background Neurodegeneration with Brain Iron Accumulation (NBIA) is a heterogeneous group of heritable, mostly recessive, progressive neurodegenerative diseases characterized by iron deposition in the basal ganglia and brainstem. There are no solid global epidemiological data on prevalence and incidence of NBIA subtypes, but registry data and ...
Susanne A. Schneider, Divyani Garg, Vassilena Iankova, Thomas Klopstock   +3 more
wiley   +1 more source

Exon-skipping antisense oligonucleotides for H3.3K27M-altered diffuse midline glioma therapy. [PDF]

Mol Ther Nucleic Acids
Yang L, Zhang Q, Wilkinson JE, Deshpande S, Krainer AR.   +4 more
europepmc   +1 more source

Genetic Etiologies of Dystonia with Anarthria/Aphonia

Movement Disorders Clinical Practice, EarlyView.
Abstract Background Dystonia with anarthria and/or aphonia (DAnAp) represents a distinctive phenotype manifesting across lifespan. Frequently associated with genetic disorders, early recognition is critical for diagnosis and management. Objectives To provide practical recommendations for the clinical evaluation of patients with DAnAp, enhancing ...
Anika Ménétrey, Eriberto Rayco, Maziar Emamikhah Abarghouei, Susan H. Fox, Anthony E. Lang, George M. Ibrahim, Carolina Gorodetsky, Christos Ganos   +7 more
wiley   +1 more source

SafeSense: An open-access safety atlas for antisense oligonucleotides adverse events in human. [PDF]

Mol Ther Nucleic Acids
Vaknin N, Ziv A, Chernobylsky T, Avkin-Nachum S, Rechavi G, Dominissini D, Mor N, N=1 Collaborative.   +7 more
europepmc   +1 more source

SPG4 Hereditary Spastic Paraplegia: From Etiology to Therapy

Movement Disorders, EarlyView.
Abstract Hereditary spastic paraplegias (HSPs) comprise a heterogeneous group of heritable neurodegenerative disorders resulting from mutations in a wide variety of genes. HSP locomotor symptoms include lower limb weakness and spasticity that arise from progressive degeneration of corticospinal axons projecting from the motor cortex to the distal ...
Emanuela Piermarini, Peter W. Baas
wiley   +1 more source

Antisense Oligonucleotides as a Gene-Silencing Strategy Regulating Cytosolic G6PDH in <i>Hordeum vulgare</i>. [PDF]

Plants (Basel)
Aquilone A, Perrotta MM, Landi S, Esposito S.   +3 more
europepmc   +1 more source

Redefining CHI3L1: Therapeutic Opportunities at the Crossroads of Immune Suppression and Disease Progression

Medicinal Research Reviews, EarlyView.
ABSTRACT Chitinase‐3‐like‐1 (CHI3L1, also known as YKL‐40) has been recognized as a biomarker of inflammation and tissue remodeling and has now emerged as a pseudoenzymatic immune checkpoint. Recent structural, immunological, and translational studies redefine it as an active regulator of immune suppression rather than a passive disease marker. Despite
Kirti Upmanyu, Saurabh Upadhyay, Moustafa T. Gabr   +2 more
wiley   +1 more source