Results 151 to 160 of about 72,467 (253)

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

Modulating NLRP3 splicing with antisense oligonucleotides to control pathological inflammation. [PDF]

Nucleic Acids Res
Klein R, Onyuru J, Centa JL, Viera EM, Putnam CD, Hoffman HM, Hastings ML.   +6 more
europepmc   +1 more source

Systematic Review of the Huntington's Disease Drug Development Pipeline, 2014 to 2025

Movement Disorders, EarlyView.
Abstract Background In the past decade, significant advances have improved our understanding of the mechanisms underlying HD pathobiology leading to several putative therapeutic targets for HD. Objective The aim was to describe the Huntington's disease (HD) drug development clinical pipeline.
Pavlina Konstantinova, Jenny Townhill, Juliana Bronzova, Viktoria Andreeva, Swati Sathe, Cristina Sampaio   +5 more
wiley   +1 more source

Restoring chloride efflux in cystic fibrosis with TMEM16a antisense oligonucleotides. [PDF]

Mol Ther
Mitri C, Rousselet N, Bardin P, Dias Wickramanayaka M, Foussignière T, Dupuis G, Leblanc M, Gournet V, Sonneville F, Corvol H, Tabary O.   +10 more
europepmc   +1 more source

RNA Therapeutics: Focus on Antisense Oligonucleotides in the Nervous System. [PDF]

Biomol Ther (Seoul)
Ertural B, Çiçek BN, Kurnaz IA.
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

Freezing shock monocytes deliver antisense oligonucleotides via liposomes for the treatment of idiopathic pulmonary fibrosis. [PDF]

Asian J Pharm Sci
Li H, Wu X, Li J, Han L, Liu H, Jiang Q, Liu B, Xia Q, Li Z, Li X, Gu S, Xu A, Zhou H, Gu X, Xu Z, Ai X, Yang C.   +16 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

Targeting rare splicing defects: Antisense oligonucleotides offer a therapeutic strategy in FRDA. [PDF]

Mol Ther Nucleic Acids
Kerkhof LMC, Buijsen RAM.
europepmc   +1 more source

<i>DUX4</i> reduction and muscle function improvement by subcutaneous delivery of gapmer antisense oligonucleotides. [PDF]

Mol Ther Nucleic Acids
Zhang A, Lim KRQ, Chen Z, Yokota T, Chen YW.   +4 more
europepmc   +1 more source