High Investment and Government Initiatives to Boost Rare Diseases Treatment ModalitiesRockville, MD, Nov. 13, 2024 (GLOBE NEWSWIRE) -- According to Fact.MR, a market research and competitive ...
KRRO-110 is designed to co-opt an endogenous enzyme, Adenosine Deaminase Acting on RNA’s (ADAR), to edit the “A” variant on SERPINA1 RNA, repair an amino acid codon, and restore secretion of normal ...
The London-based company is developing the gene therapy for adenosine deaminase severe combined immunodeficiency, commonly known as ADA-SCID or “bubble baby” disease. OTL-101 is similar to GSK ...
Multiomics joint analyses based on a structural variant (SV) map from 16 genome assemblies and 2,105 resequenced accession genomes shed light on the regulatory effect of SVs on gene expression and ...