Rare diseases
A new kind of stem cell is revolutionizing regenerative medicine
Induced pluripotent stem cells are paving the way for personalized treatments to diabetes, vision loss and more. However, scientists still face hurdles such as strict regulations, scalability, cell longevity and immune rejection.
Richard Silverman to speak at 91亚色传媒 2025
Richard Silverman and Melissa Moore are the featured speakers at the 91亚色传媒 annual meeting to be held April 12-15 in Chicago.
Becoming a scientific honey bee
At the World Science Forum, a speaker鈥檚 call for scientists to go out and 鈥渕ake honey鈥� felt like the answer to a question Katy Brewer had been considering for a long time.
Study sheds light on treatment for rare genetic disorder
Aaron Hoskins鈥� lab partnered with a drug company to understand how RNA-targeting drugs work on spinal muscular atrophy, a disorder resulting from errors in production of a protein related to muscle movement.
New gene new strides in gangliosidosis
A gene that decreases disease progression in mice provides a new direction for human therapy.
How a gene spurs tooth development
University of Iowa researchers find a clue in a rare genetic disorder鈥檚 missing chromosome.
A tiny genetic tweak with big heart health implications
Researchers have discovered that a gene mutation found among members of the Amish community could inform heart disease treatment.
CRISPR and other new technologies open doors, but which diseases get prioritized?
Drug development comes down to money and science. Profits are a factor, but advocacy and incentives can tilt the scale.
Understanding the cause of childhood Alzheimer's
Today is World Sanfilippo Awareness Day.