Results 111 to 120 of about 32,046 (240)
Transthyretin amyloid cardiomyopathy
, 2020 Transthyretin amyloid cardiomyopathy (ATTR-CM) resulting from deposition of transthyretin amyloid fibrils in the heart is an underrecognised cause of heart failure in the elderly and is associated with a poor life expectancy.
Hansen, Peter Riis, Krakauer, Martin
core +1 more source
Three-dimensional structure of the Transthyretin-Retinol-Binding Protein Complex
, 2002 Transthyretin (TTR), formerly called prealbumin, one of the transporters of the hormone thyroxine and retinol-binding protein (RBP), the specific carrier of vitamin A, forms, under physiological conditions, a macromolecular complex that prevents ...
MONACO, Ugo Luigi
core
Genome Editing for Glycogen Storage Diseases
Journal of Inherited Metabolic Disease, Volume 49, Issue 5, September 2026.ABSTRACT Gene therapy has been developed for several glycogen storage diseases and has advanced into clinical trials. However, the limitations of these gene therapies with regard to stability following treatment early in life have led to the development of genome editing.
Troy von Beck +2 more
wiley +1 more source
Smart Design: Integrating Artificial Intelligence and Gene Editing for Advanced mRNA Therapeutics
MedComm – Biomaterials and Applications, Volume 5, Issue 3, September 2026.The challenges of mRNA therapy and the application of artificial intelligence and gene editing in the field of mRNA drugs. ABSTRACT Artificial intelligence (AI) and gene editing are increasingly being applied to the design and evaluation of mRNA therapeutics.
Haixing Shi +11 more
wiley +1 more source
Inhibition of Transthyretin Fibrillogenesis Using a Conformation Specific Antibody [PDF]
, 2012 Immunoglobulin-mediated inhibition of amyloid fibril formation in vivo is a promising strategy for the treatment of protein misfolding diseases such as the amyloidoses.
Bugyei-Twum, Antoinette
core +2 more sources
Discover MedicineAmyloidosis is a systemic disorder characterized by extracellular protein deposition, with transthyretin amyloidosis being the most common subtype. Carpal tunnel syndrome is a frequent early manifestation, yet detailed MRI characterization of amyloid ...
Jean Pierre Abdallah +4 more
doaj +1 more source
PLoS ONEBackgroundHeart failure is a clinical syndrome resulting from numerous pathological conditions. One cause of heart failure, transthyretin cardiac amyloidosis, presents insidiously with common and seemingly unrelated symptoms.
Sherry L Ball +3 more
doaj +1 more source
European Journal of Neurology, Volume 33, Issue 9, September 2026.There are no tools to support early detection of ATTRv signs/symptoms or to identify risk of transition to clinically detectable disease in carriers of pathogenic ATTRv variants. This retrospective cohort study describes the ACNA, a brief 23‐item assessment for use in routine clinical visits.
Isabel Conceição +8 more
wiley +1 more source
Combating ageing beyond the cell: Emerging roles of extracellular proteostasis
The FEBS Journal, Volume 293, Issue 18, Page 5558-5584, September 2026.Ageing challenges the body's ability to maintain a stable and functional proteome, leading to protein damage and aggregation both inside and outside cells. This review focuses on the less well understood mechanisms of extracellular protein quality control and how they become disrupted in ageing, particularly in neurodegenerative diseases.
Siddharth R. Venkatesh +7 more
wiley +1 more source
Journal of the Peripheral Nervous System, Volume 31, Issue 3, September 2026.ABSTRACT Background and Aims The Val122Ile ATTR Amyloidosis has traditionally been linked to cardiac manifestations. Recent studies suggest that neuropathy may be relevant. In this study, we characterized its peripheral nerve manifestations in depth. Methods This was a national, multicenter, observational, retrospective study.
Anna Paula Paranhos +16 more
wiley +1 more source