Results 161 to 170 of about 143,834 (286)

Precision therapeutics for early onset epilepsy: Allele-selective antisense oligonucleotides for SCN2A-related epilepsy. [PDF]

Mol Ther Nucleic Acids
Kim-McManus O.
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

An antisense method for efficient exon skipping and its application to Duchenne muscular dystrophy. [PDF]

Proc Natl Acad Sci U S A
Feng P, Gao P, Meng S, Yuan Y, Krainer AR, Hua Y.   +5 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 antidote to oncogenic poison exons. [PDF]

Genes Dev
Arvola RM, Singh G.
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

Sydnone-modified nucleosides as versatile tools for bioorthogonal post-synthetic functionalization of antisense oligonucleotides. [PDF]

RSC Chem Biol
Bristiel A, Khuu A, Poulet M, Taran F, Urban D, Guignard R, Guianvarc'h D.   +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

Emerging Nucleic Acid-Based Therapies for Hypercholesterolemia with Focus on a New Modality, Liver-Directed miR-30c Analog C2. [PDF]

Cells
Srivastava RAK.
europepmc   +1 more source

RNA duplex formation and competing endogenous RNA, proposed as mechanisms in regulating expression of natural antisense transcripts- from hypotheses to potential therapeutic applications. [PDF]

Front Mol Biosci
Nakatake R, Okuyama T, Kimura T, Nishizawa M.   +3 more
europepmc   +1 more source