Results 151 to 160 of about 171,175 (241)

Brain region-resolved pharmacodynamics of an antisense oligonucleotide targeting human SNCA 3'UTR in BAC-hSNCA rats. [PDF]

Transl Neurodegener
Naki M, Kattan FG, Pantazopoulou M, Skea S, Zisis D, Pilalis E, Fouka M, Koronaiou E, Fotis C, Riess O, Chatziioannou A, Alexopoulos LG, Stefanis L, Doxakis E.   +13 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

ALG-020572, an Antisense Oligonucleotide for the Treatment of Chronic Hepatitis B Virus Infection Discontinued for Drug-Induced Liver Injury. [PDF]

J Viral Hepat
Agarwal K, Forton D, Zen Y, Wu M, Lin TI, Le K, Chanda S, Blatt L, Fry J, Gane E.   +9 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

An antisense oligonucleotide targeting STK25 as a long-acting therapeutic candidate for early MASLD. [PDF]

Mol Ther Nucleic Acids
Dong A, Li N, Lang X, Chen S, Qin Y, Wang Y, Wang H, Guo M, Yan A, Song K, Bi J, Ren G, Lai F, Yang J, Dang Y.   +14 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

Meta-analysis of adverse events in clinical studies with antisense oligonucleotide therapies. [PDF]

Mol Ther Nucleic Acids
Vermeer C, Venema RR, Birnie E, Bolling MC, Knoers N, Bremer J, van den Akker PC.   +6 more
europepmc   +1 more source

Emergencies in Amyotrophic Lateral Sclerosis

Muscle &Nerve, EarlyView.
ABSTRACT Emergencies are frequent in people living with amyotrophic lateral sclerosis (pALS), especially as the disease progresses, and can necessitate urgent evaluation and intervention. Progressive weakness in ALS inevitably increases fall risk, making discussion of fall prevention strategies integral to caring for pALS.
S. Pinar Uysal, Katherine A. Dudley, Stacey Sullivan, Kate T. Brizzi, Katherine M. Burke, Sydney Hall, Kiersten Pease, Joshua N. Goldstein, James D. Berry, Doreen T. Ho   +9 more
wiley   +1 more source

WDR91, an endosomal maturation protein, promotes antisense oligonucleotide activity. [PDF]

Mol Ther Nucleic Acids
Chen S, Zheng T, Li D.
europepmc   +1 more source

Givinostat enhances antisense oligonucleotide efficacy in the <i>mdx52</i> model of Duchenne muscular dystrophy. [PDF]

Mol Ther Nucleic Acids
Phongsavanh X, Gastaldi C, Mottais A, Doisy M, Le Coz O, de Haan S, Spitali P, Garcia L, Goyenvalle A.   +8 more
europepmc   +1 more source