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genetic disease
genetic disease
| 5 min read
by
Stephanie DeMarco, PhD
Moderna’s path to mRNA therapies for rare metabolic disorders
Paolo Martini leads the biotech company’s rare disease research program with the hope of developing the very first treatments for genetic metabolic disorders.
Articles
| 10+ min read
by
Tiffany Garbutt, PhD
The chronic hiccup chronicles
Researchers and patients developed unexpected and innovative solutions to a mysterious disease that appears as little more than a hiccup.
Articles
| 7 min read
by
Aparna Nathan, PhD
A gene therapy hope for halting Huntington’s disease
A one-time gene therapy aims to stop Huntington's disease at its source. But first, it has to survive clinical trials.
News
| 4 min read
by
Dan Samorodnitsky, PhD
The intricate design of a fundamental filter protein
A collaboration of nephrologists and biochemists created the first structure of “megalin,” a critical protein in kidney and brain disorders.
News
Available on Demand
Cell Therapy Advances: From Benchtop to Bedside
LIVE May 12th, 2022 at 2:30 PM
Discover the most current cell therapy research and the challenges of translating them into the clinic.
Webinars
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