When researchers study rare diseases, breakthroughs can be made that help patients. Revisit studies that are putting genetic puzzle pieces together to tackle these conditions.
Groundbreaking new research marrying high-intensity focused ultrasound with genetically modified cells may spur bone and soft tissue regeneration.
Advances in gene therapy are yielding new options for treating inherited retinal degenerations, giving specialists new tools — and new hope for patients and families.
A Cas9 protein discovered in meningitis bacteria can act as precise “scissors” for both types of genetic material, cutting at a desired spot guided by CRISPR RNAs.