Systemic diseases of liver origin (SDLO) are complex diseases in multiple organ systems,
such as cardiovascular, musculoskeletal, endocrine, renal, respiratory, and sensory organ …

Oligonucleotide-based therapies are a promising approach for treating a wide range of hard-
to-treat diseases, particularly genetic and rare diseases. These therapies involve the use of …

Ion-pairing reversed-phase liquid chromatography (IP-RPLC) is the reference separation
technique for characterizing oligonucleotides (ONs) and their related impurities. The aim of …

Background Predictive biomarkers for oesophageal squamous cell carcinoma (ESCC)
immunotherapy are lacking, and immunotherapy resistance remains to be addressed. The …

Abstract mRNA therapeutics are set to revolutionize disease prevention and treatment,
inspiring the development of platforms for safe and effective mRNA delivery. However …

Recessive dystrophic epidermolysis bullosa (RDEB) is a rare and most often severe genetic
disease characterized by recurrent blistering and erosions of the skin and mucous …

Antisense oligonucleotide-based (ASO) therapeutics have emerged as a promising strategy
for the treatment of human disorders. Charge-neutral PMOs have promising biological and …

Although cationic liposomes are efficient carriers for nucleic acid delivery, their toxicity often
hampers the clinical translation. Polyethylene glycol (PEG) coating has been largely used to …

The present investigation showed that each of the three different liquid chromatography
modes may be successfully used for the qualitative analysis of nusinersen metabolites in a …

Oligonucleotide therapeutics (ONTs) represent a new modality with unique pharmacological
and chemical properties that modulate gene expression with a high degree of target …