Results 171 to 180 of about 9,975 (285)

New Technologies Empower Hemophilic Arthropathy Treatment: Focusing on Regenerative Medicine, Molecular Targeting, and Gene Therapy

open access: yesMedicine Bulletin, EarlyView.
ABSTRACT Hemophilic arthropathy (HA), a major complication of hemophilia, is a chronic osteoarthropathy driven by repeated joint bleeding. Although traditional therapies such as coagulation factor replacement, physical rehabilitation, and surgery can manage symptoms, they cannot fundamentally repair established joint damage or break the pathological ...
Lin Sun   +8 more
wiley   +1 more source

Uncovering the Complexity of Synucleinopathies: An Ongoing Tale Between Proteins and Lipids

open access: yesMovement Disorders, EarlyView.
Abstract Neurodegenerative diseases are pathological states characterized by progressive alterations in brain homeostasis during aging. Synucleinopathies, including Parkinson's disease and dementia with Lewy bodies, are defined neuropathologically by the accumulation of inclusions known as Lewy bodies and Lewy neurites.
Manuel Flores‐León, Tiago F. Outeiro
wiley   +1 more source

The Race to Salvage Glucocerebrosidase: Understanding Small‐Molecule Therapies for GBA1‐Associated Parkinsonism

open access: yesMovement Disorders, EarlyView.
Variants in GBA1, the gene encoding the lysosomal enzyme glucocerebrosidase, cause Gaucher disease and confer an increased risk for parkinsonism. Strategies using small molecules can improve the function of glucocerebrosidase in lysosomes. A clear understanding of the mechanism‐of‐action of these compounds will facilitate development of GBA1‐modulating
Mark J. Henderson   +5 more
wiley   +1 more source

The small molecule CBR-5884 inhibits the Candida albicans phosphatidylserine synthase [PDF]

open access: gold, 2023
Yue Zhou   +11 more
openalex   +1 more source

VPS13A Deficiency Leads to Impaired Lipid Distribution and Alteration of Mitochondrial Calcium Homeostasis in Fibroblasts of VPS13A Disease Patients

open access: yesMovement Disorders, EarlyView.
Abstract Background Membrane contact sites are crucial for the exchange of ions or lipids and thus are critical for the function and maintenance of organelles. VPS13A is a membrane‐residing, bridge‐like protein connecting two membranes to enable bulk lipid transfer. Loss‐of‐function mutations in the VPS13A gene cause VPS13A disease.
Dajana Grossmann   +10 more
wiley   +1 more source

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