news
Meet the hemifusome: a new organelle with big impact
Scientists have discovered a previously unknown organelle inside human cells - the hemifusome - that could change our understanding of rare genetic disorders.
List view / Grid view
Genetic diseases
news
New discovery shows how viral vectors release genes in therapy
Researchers at The University of Osaka have found how structural changes in the VP1 protein of AAV vectors trigger genome release - offering insights for improving the safety, efficiency and stability of gene therapy delivery systems.
article
Cracking the code to scalable cell and gene therapy
Cell and gene therapy is rapidly transforming the treatment of complex diseases, yet scaling production efficiently remains a challenge. Dr Tia Harmon from PHC Corporation of North America explores the innovations that are optimising cell expansion to enhance the discovery of advanced therapies.
news
CRISPR activation: quicker diagnosis of rare genetic diseases
Using CRISPRa to activate genes in readily accessible cells provides an effective and accurate diagnosis of genetic diseases.
news
Soft nickases could be the future of safe, effective gene editing
Researchers in the US have developed a new CRISPR method for treating genetic conditions using nickases that they say is safer and more effective.
news
Promising new RNA editing technology could be used to treat genetic diseases
New research by the University of California, San Diego could provide a much simpler way to repair disease-causing mutations in RNA.
news
First studies of human genetic variation released by the gnomAD Consortium
The Genome Aggregation Database (gnomAD) Consortium has released seven papers leveraging its database to study genetic variants and their potential for guiding discovery of safer drugs.
news
Scientists make improvements to AI pipeline used to diagnose genetic diseases
Building on previous research, scientists have made improvements to an artificial intelligence pipeline used to diagnose genetic diseases via blood samples obtained from gravely ill infants.
