Some rare skin diseases not only reduce the quality of life of patients, but also can be devastating conditions, leading to amputations or death. At the 31st annual congress of the European Society of Gene and Cell Therapy (ESGCT), held last week in Rome, different laboratories showcased their approaches to editing mutations related to this group of diseases.

Using whole genome sequencing, scientists at Boston Children’s Hospital have studied the genes and mutations of ataxia-telangiectasia (A-T) that would respond to treatments with splice-switching antisense oligonucleotides (ASOs). Their work, published on July 12, 2023, in Nature, determined the appropriate individualized genetic therapy for these patients and identified a new drug.

Vanda Pharmaceuticals Inc. and Olipass Corp. have entered into a research and development collaboration agreement to jointly develop a set of antisense oligonucleotide (ASO) molecules based on Olipass' proprietary modified peptide nucleic acids.

"RNA was long thought to be an 'undruggable' target for small molecules, because most cellular RNAs have extensive secondary structure, but only limited tertiary structure," Matthew Disney told BioWorld Science.

Two separate groups have recently shown that in mouse models, inactivation of a single gene was enough to directly convert other cell types in the brain into neurons.