The main goal of the molecular diagnosis of hereditary diseases is the identification of disease-causing mutations with a view to detecting carrier individuals who may benefit from early diagnosis, ...

Antisense oligonucleotides (ASOs) are small, single-stranded molecules that can suppress the expression of targeted genes and thereby achieve therapeutic effects. However, many ASO-based drug ...

Heteroduplex oligonucleotide (HDO) is a brand new oligonucleotide drug, pioneered by researchers of Tokyo Medical and Dental University, Osaka University and ISIS. HDO is found to be significantly ...

Researchers have developed a drug delivery platform wherein heteroduplex oligonucleotide drugs conjugated with cholesterol are able to cross the blood-brain barrier and achieve therapeutic ...

The development of CRISPR/Cas9 revolutionized researchers’ ability to edit the genome at will. However, the nature of CRISPR edits is not entirely controllable or predictable. This uncertainty is due ...

In the few past years, engineered endonucleases (Meganucleases, ZFN, TALENS) and CRISPR/Cas9 technology have revolutionized genome editing possibilities. Genome editing can now be used in a wide range ...

Caption This study revealed that, by binding to lipoproteins, cholesterol-conjugated heteroduplex oligonucleotides (Chol-HDOs) can remain in the bloodstream for longer and reach brain tissue much more ...

Heteroduplex oligonucleotide is a brand new oligonucleotide drug. It is found to be significantly potent at reducing expression of the target RNA, and also improves the phenotype in disease models ...