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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
Chronic hiccup chronicles: Causes, treatments, and patient solutions
Discover causes, treatments, and patient-driven solutions for chronic hiccups, a rare and mysterious condition.
Articles
| 8 min read
by
Aparna Nathan, PhD
Gene therapy for Huntington's disease: A new hope to halt progression
A one-time gene therapy aims to stop Huntington's disease at its source. But first, it has to survive clinical trials.
Articles
| 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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Partnering to translate population-scale signals into mechanistic insights for faster drug discovery
New collaborative initiatives are bringing pharmaceutical R&D together around large-scale datasets to accelerate therapeutic discovery.
Articles
Modeling neurotropic viral infections using human cerebral organoids
Using fetal-stage brain organoids, researchers are uncovering how Zika virus impacts neurodevelopment and contributes to microcephaly.
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The bioprocess trifecta: completing the real-time data picture with at-line titer measurement
Explore approaches to integrating timely protein titer measurements with cell health data to improve bioprocess visibility and decision-making.
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Volume 21 • Issue 4 • December 2025
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