Rare & Genetic Diseases
Biobanking gives scientists access to thousands of biological samples, moving
precision medicine one step closer to reality.
| 8 min read
Alnylam Pharmaceuticals develops therapeutics based on the RNA interference (RNAi) pathway discovered in 1998.
| 5 min read
Paolo Martini leads the biotech company’s rare disease research program with the hope of developing the very first treatments for genetic metabolic disorders.
Gene therapy has a complicated history, marked by many ups and downs and crafted by ever-advancing technologies.
| 10+ min read
Scientists identified specific genes that confer protection or vulnerability to various infections, pointing the way to new treatments.
| 6 min read
Experts discussed the major hurdles of AAV gene therapy, including immunogenicity, vector design, and AAV variability.
| 10 min read
Researchers discovered a new gene required for pancreas development, but much to their surprise, they found that it was absent in all animals except primates.
| 4 min read
A new porcine model for the rare neurological disorder CLN3 Batten disease more faithfully recreates human symptoms and could accelerate drug development.
| 1 min read
Transgenic mice play pivotal roles in scientific and therapeutic discovery, but how did they become a staple model system?
| 10 min read
Injecting muscle-derived cells into the tongue could help alleviate swallowing difficulties caused by head and neck cancer treatment.
| 4 min read
Microscopy images reveal the structural change that enlarges an ion channel pore, opening new directions in drug discovery and delivery.
| 10+ min read
Discover causes, treatments, and patient-driven solutions for chronic hiccups, a rare and mysterious condition.
