LETI-101 has demonstrated therapeutically relevant reductions of mutant huntingtin (HTT) protein, exceeding 80%, while preserving essential ...

RNA-based therapeutics have rapidly emerged over the past decade, offering a new class of medicines that differ significantly from conventional drugs. These therapies can be programmed to target or ...

Aptamers are single-stranded DNA or RNA molecules with unique 3D structures that allow for specific binding to a wide variety of ions and molecules. Due to their unique properties, aptamers have been ...

CRISPR protein AcrVIB1, a promising inhibitor whose precise role has not yet been determined. In cooperation with the Helmholtz Centre for Infection Research (HZI) in Braunschweig, a research team ...

The CRISPR-Cas gene scissors offer a wide range of potential applications, from the treatment of genetic diseases to antiviral therapies and diagnostics. However, to safely harness their powers, ...

Wulf Blankenfeldt at HZI. “The nuclease Cas13b can recognize and cut RNA. It is currently used to silence genes, whether to study their function, clear viruses, or counteract genetic diseases li ...

The CRISPR-Cas gene scissors offer a wide range of potential applications, from the treatment of genetic diseases to antiviral therapies and diagnostics. However, to safely harness their powers, ...

Guided by RNA, the Cas9 endonuclease breaks DNA at a target sequence. Imprecise repair of the double strand break can result in insertion or deletion mutations, while repair pathways can be ...

Newco Akribion Therapeutics GmbH has raised €8 million (US$8.3 million) in a seed round to develop a new and potent class of RNA-targeted CRISPR nucleases, which, rather than cleaving specific nucleic ...

ZWINGENBERG, Germany, Feb. 4, 2025 /PRNewswire/ -- Akribion Therapeutics ("Akribion"), the early-stage biotech developing a unique, RNA-guided, nuclease-based technology for programmable cell ...