When I was 20 weeks pregnant, my doctor recommended an amniocentesis so we could find out what was causing concerns during my pregnancy. They took amniotic fluid for testing, and that's when my husband and I learned our daughter had a rare genetic disorder called Fanconi anemia (FA). We found out that we were both carriers, something we had never known.
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David Liu
PhD
David R. Liu is the Richard Merkin Professor and director of the Merkin Institute of Transformative Technologies in Healthcare, vice-chair of the faculty at the Broad Institute of MIT and Harvard, the Thomas Dudley Cabot Professor of the Natural Sciences at Harvard University, and a Howard Hughes Medical Institute (HHMI) investigator. Liu’s research integrates chemistry and evolution to illuminate biology and enable next-generation therapeutics. His major research interests include the engineering, evolution, and in vivo delivery of genome editing proteins such as base editors and prime editors to study and treat genetic diseases; the evolution of proteins with novel therapeutic potential using phage-assisted continuous evolution (PACE); and the discovery of bioactive synthetic small molecules and synthetic polymers using DNA-templated organic synthesis and DNA-encoded libraries.
Base editing—the first general method to perform precision gene editing without double-stranded breaks, and a Science 2017 Breakthrough of the Year finalist—as well as prime editing, PACE, and DNA-templated synthesis are four examples of technologies pioneered in his laboratory. These technologies are used by thousands of labs around the world and have enabled the study and treatment of genetic diseases. Five base editing clinical trials are already underway to treat leukemia, hypercholesterolemia, sickle-cell disease, and beta-thalassemia, and the first clinical benefit of a base edited therapeutic in a T-cell leukemia trial has been reported.
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David Liu
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The Family Retreat offers families a break from appointments, treatment decisions and everyday responsibilities. Children can try new activities and grow in confidence while caregivers learn, share practical support and connect with one another. The relationships formed at camp often continue long after everyone goes home.
By Cecilia Córdoba, mother of Agustina Milagros Kaucic When my daughter Agustina was 2 and a half years old, our lives changed forever. It was very difficult to reach a diagnosis. And when we finally received it, it was devastating....