Browsing Tag
gene therapy
22 posts
Placenta-tropic VEGF mRNA lipid nanoparticles ameliorate murine pre-eclampsia
Chappell, L. C., Cluver, C. A., Kingdom, J. & Tong, S. Pre-eclampsia. Lancet 398, 341–354 (2021). Article CAS …
Doctors cured her sickle-cell disease. So why is she still in pain?
For most of her life, Genesis Jones’s daily routine revolved around her illness, the painful blood disorder known…
Hope, despair and CRISPR — the race to save one woman’s life
When researcher Arkasubhra Ghosh finally met Uditi Saraf, he hoped that there was still a chance to save…
The immune system can sabotage gene therapies — can scientists rein it in?
A child receives a gene therapy for Duchenne muscular dystrophy.Credit: Elisabeth Schneider Charpentier/Look At Sciences via Science Photo…
How to kill the ‘zombie’ cells that make you age
Lurking throughout your body, from your liver to your brain, are zombie-like entities known as senescent cells. They…
CRISPR therapy restores some vision to people with blindness
A CRISPR-based gene-editing therapy led to improved vision in people with an inherited condition that causes blindness1. Access…
HEROS 2.0 trial results published
Researchers at Texas Children’s Cancer Center and the Center for Cell and Gene Therapy at Baylor College of…
Targeting RNA opens therapeutic avenues for Timothy syndrome
Nature, Published online: 24 April 2024; doi:10.1038/d41586-024-00911-1 A therapeutic strategy that alters gene expression in a rare and…
Top pharmaceutical company evaluates Optimer for precision liver medicine
Aptamer Group plc, the developer of novel Optimer® binders to enable innovation in the life sciences industry, today announces…
Gene therapy and glycoside drugs offer new hope for polycystic kidney disease treatment
Researchers have shown that dangerous cysts, which form over time in polycystic kidney disease (PKD), can be prevented…