CTNS-RD-04, an ex vivo gene therapy for cystinosis, showed reduced white-cell cystine levels and an acceptable safety profile ...

Cystinuria occurs in one out of every 7,000 in the world, one out of every 10,000 -20,000 in Japan, but its curative treatment has not been established. AGT1/SLC7A13, a protein, is an unknown factor ...

Cystine stones are a type of kidney stone. They are also called renal calculi, nephrolithiasis, or urolithiasis, and they are caused by a genetic condition. Here’s what to know about cystine crystals ...

Using molecules that closely mimic the structure of the dimeric amino acid L-cystine, chemists have discovered a way to suppress formation of large L-cystine crystals. The finding, reported in Science ...

CYSTINE-STORAGE disease (cystinosis or Lignac-> Fanconi disease) is an uncommon but intriguing disorder, the etiology and pathogenesis of which are unknown. The cardinal pathological finding is the ...

Fibroblasts derived from patients with cystinosis, an autosomal recessive condition, accumulate the disulfide amino acid cystine within lysosomes. The metabolic defect leading to the cystine ...

Cystine Stones contain cystine, a product of the amino acids cysteine and methionine. Amino acids are the building blocks of proteins. Thus the simplest way to reduce the amount of cystine in the body ...

Despite the advances in the diagnosis and treatment of breast cancer, breast cancers still cause significant mortality. For some patients, especially those with triple-negative breast cancer, current ...

It has previously been reported that cystinuria, as it appears clinically, represents at least three biochemically and genetically distinct diseases. 13 The present studies were undertaken to ...

The ability of cystine dimethylester (CDME) to load lysosomes with cystine has been used to establish the basic defect in cystinosis: defective cystine exodus from lysosomes. Using CDME loading, it ...

CYSTARAN (cysteamine) 0.44% ophthalmic solution Cystaran (cysteamine ophthalmic solution; Sigma-Tau) is the first FDA-approved therapy for the treatment of corneal cystine crystal accumulation in ...

Peptides as therapeutics are advantageous since they are generally safe and well tolerated, with a short half-life limiting risk for off-target toxicities. However, because of their small size, most ...