Results 81 to 90 of about 21,795 (202)
Photo‐Catalyzed Labeling of Amyloid Deposits in Human Tissue to Proteotype Amyloidosis Diseases
Amyloid‐ID, a ThT‐like photocatalytic probe, enables proteotyping of amyloid deposits by ROS‐mediated protein labeling. Applied to patient biopsies, amyloid‐ID identifies potential fibrinogen‐derived amyloid in laryngeal amyloidosis, revealing a mechanically sensitive fibrillation mechanism linked to vocal cord friction.
Huan Feng +10 more
wiley +1 more source
Inotersen: new promise for the treatment of hereditary transthyretin amyloidosis
Veena Mathew,1 Annabel K Wang1,2 1Department of Neurology, UCI ALS and Neuromuscular Center, University of California, Irvine, Orange, CA, USA; 2Neurology Section, Tibor Rubin VA Medical Center, Long Beach, CA, USA Abstract: Hereditary transthyretin ...
Mathew V, Wang AK
doaj
RNA‐Based Therapies for Inherited Metabolic Disorders
ABSTRACT Inherited metabolic disorders (IMDs) are a diverse and complex group of genetic conditions resulting from deficiencies in enzymes, transporters, or cofactors. These deficiencies lead to metabolic dysfunction and severe clinical consequences. Despite significant progress in understanding their molecular basis, treatment options remain limited ...
Reddy Sreekanth Vootukuri +5 more
wiley +1 more source
Background/Objectives: Having serum biomarkers available for cardiac transthyretin amyloidosis (ATTR-CA) would be beneficial for diagnosis and prognosis. This study aimed to identify potential ATTR-CA biomarkers through proteomic analysis.
Joanna Waś +9 more
doaj +1 more source
Transthyretin (TTR) is a transporter for thyroid hormone and retinol binding protein that has recently been reported to have proteolytic activity against certain substrates, including amidated neuropeptide Y (NPY).
Sukanya Tangthavewattana +2 more
doaj +1 more source
Conservative Management of Bilateral Spontaneous Pneumothorax
Bilateral simultaneous spontaneous pneumothorax is a rare occurrence that generally requires prompt intervention. We report a case of simultaneous bilateral spontaneous pneumothorax that was conservatively managed. ABSTRACT Bilateral simultaneous spontaneous pneumothorax is a rare occurrence that generally requires prompt intervention. We report a case
Hanson Siu, Chuan T. Foo
wiley +1 more source
Structure of ATTRv-F64S fibrils isolated from skin tissue of a living patient
Amyloid transthyretin-derived (ATTR) amyloidosis is a degenerative, systemic disease characterized by transthyretin fibril deposition in organs like the heart, kidneys, liver, and skin.
Jun Yu +7 more
doaj +1 more source
Nature Inspired Delivery Vehicles for CRISPR‐Based Genome Editing
The review highlights nature‐inspired nanocarriers for CRISPR delivery, emphasizing viral vectors, extracellular vesicles, liposomes, and lipid nanoparticles. It discusses their roles in improving specificity, minimizing immunogenicity, and overcoming barriers in genome editing. Recent advancements, challenges, and therapeutic applications are explored,
Elizabeth Maria Clarissa +4 more
wiley +1 more source
Lipid Nanoparticles in Drug Delivery: Overcoming Challenges and Unlocking New Frontiers
Lipid nanoparticles (LNPs) have revolutionized nucleic acid delivery, yet challenges in formulation, stability, and targeted efficacy persist. This review explores how advances in microfluidic manufacturing, precision targeting, and endosomal escape are reshaping LNP design.
Jordan Kambanis +7 more
wiley +1 more source
Hereditary transthyretin amyloidosis (ATTRv)
Hereditary transthyretin (TTR) amyloidosis (ATTRv amyloidosis) is a devastating disease characterized by broad range of clinical manifestations, including predominantly neurological, predominantly cardiac, and mixed phenotypes. This wide phenotypic variability hindered timely disease diagnosis and risk stratification in the past, especially in ...
Filippos Triposkiadis +8 more
openaire +3 more sources

