Nanocarriers enhance peptide and oligonucleotide therapy by improving stability, bioavailability, and targeted delivery for effective clinical applications.

An antisense therapy restores production of the protein FMRP in cell samples taken from patients with fragile X syndrome. This breakthrough was possible because of the novel findings, also presented ...

Peptide nucleic acids (PNAs) represent a revolutionary class of synthetic nucleic acid analogues, in which the traditional sugar–phosphate backbone is replaced by a neutral N-(2-aminoethyl) glycine ...

Therapeutic oligonucleotides act on different stages of pathological gene expression. Standard schematic of oligonucleotide activities in pathogenesis disease progression. Decoys bind to transcription ...

So far, 2013 has been a good year for antisense technology. In fact, it’s been great. In January the FDA OK’d Isis Pharmaceuticals’ Kynamro™ (mipomersen sodium), the only currently marketed antisense ...

TMDU researchers demonstrate proof of concept of antisense nucleic acid therapy to prevent the spread of α-synuclein pathologies in synucleinopathies. Parkinson's disease (PD), as well as many other ...

Cure Rare Disease has been awarded a $5.69 million grant from the California Institute for Regenerative Medicine (CIRM) to advance the development of an antisense oligonucleotide therapy for ...

“Together, these findings support a beneficial role for CD47 knockdown in alleviating age-associated metabolic dysfunction through coordinated effects across multiple organs” “Together, these findings ...

The Nature Index 2025 Research Leaders — previously known as Annual Tables — reveal the leading institutions and countries/territories in the natural and health sciences, according to their output in ...

An antisense therapy developed by Joel D. Richter, PhD, Sneha Shah, PhD, and Jonathan K. Watts, PhD, at UMass Chan Medical School and Elizabeth Berry-Kravis, MD, PhD, at RUSH University Medical Center ...