Results 81 to 90 of about 21,795 (202)

Photo‐Catalyzed Labeling of Amyloid Deposits in Human Tissue to Proteotype Amyloidosis Diseases

open access: yesAggregate, Volume 7, Issue 3, March 2026.
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

open access: yesDrug Design, Development and Therapy, 2019
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

open access: yesJournal of Inherited Metabolic Disease, Volume 49, Issue 2, March 2026.
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

Proteomic Analysis of Serum in Cardiac Transthyretin Amyloidosis: Diagnostic and Prognostic Implications for Biomarker Discovery

open access: yesBiomedicines
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

The hydrophobic C‐terminal sequence of transthyretin affects its catalytic kinetics towards amidated neuropeptide Y

open access: yesFEBS Open Bio, 2019
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

open access: yesRespirology Case Reports, Volume 14, Issue 3, March 2026.
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

open access: yesNature Communications
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

open access: yesSmall, Volume 22, Issue 16, 17 March 2026.
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

open access: yesSmall Structures, Volume 7, Issue 3, March 2026.
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)

open access: yesCurrent Problems in Cardiology
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

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