TTX-381, a one-time, subretinal AAV gene therapy, is designed to deliver a functional TPP1 gene copy directly to retinal cells to produce the missing TPP1 enzyme and preserve patient vision. The post Andelyn and Tern Therapeutics Partner on Gene Therapy Manufacturing appeared first on GEN - Genetic Engineering and Biotechnology News.| GEN – Genetic Engineering and Biotechnology News
CRISPR-based gene editing technology Casgevy has been approved for treatment of sickle cell disease and beta-thalassaemia in the NHS| Genomics Education Programme
Gene therapies can take place either 'in vivo' or 'ex vivo'; we compare these two approaches and their importance in gene therapy The post In vivo and ex vivo gene therapies explained appeared first on Genomics Education Programme.| Genomics Education Programme