Nearly half of all babies born with Down syndrome face congenital heart defects, often involving serious malformations that require surgery in the first months of life.

Using stem cells, gene editing, and AI, a team of scientists at Gladstone Institutes identified one gene that plays a key role in the heart defects seen in Down syndrome. Then, by reducing the levels of this gene in mice, they were able to fix the problem and restore normal heart development.

In this video, Deepak Srivastava, Katie Pollard, and Sanjeev Ranade explain what they found and how their work could pave the way for treatments to help prevent heart malformations in people with Down syndrome and related heart defects.

Read more about this study, which was published in the journal Nature.

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Katie Pollard, PhD
Senior Investigator and Director, Gladstone Institute of Data Science and Biotechnology

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