Will genomic discoveries will lead to curative therapies involving genomic modifications for dozens of genetic diseases by 2030? Tim discussed this bold prediction at an NHGRI seminar last month.
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CZI’s Neurodegeneration Challenge Network brings together scientists to understand the fundamental biology of neurodegenerative diseases and develop new strategies for treatment and prevention, this year Tim gave the keynote presentation at their annual meeting. Watch his presentation here!
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Tim had the opportunity to speak on a panel at the Harvard Medical School Division of Biomedical Informatics Annual Precision Medical Conference. The focus of the conference this year was hyperindividualized treatments and the keynote speaker was Julia Vitarello, our patient Mila’s mother.
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Tim will be speaking at the upcoming Harvard Medical School Center for Bioethics annual bioethics conference, “Biotechnology and the Future of Medicine,” on March 9th at 2:40PM during the “N=1 Trials” and the Future of Hyper-Personalized Medicine session.
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In the spirit of Rare Disease Day, we are both reflecting on the past — in particular, the work we started with milasen two and a half years ago — and are looking toward the future…
Read MoreTim and Julia Vitarello shared Mila’s story at the GoldLab Symposium.
Read MoreThank you to the American Academy of Neurology for giving us a platform to share our research.
Read MoreTim had the opportunity to share Mila’s story with her mother Julia at the Broad Institute Rare Disease Day Symposium — Focus on Rare Disease: Decoding genomes, unlocking therapies.
Read MoreTim tells the story of Mila, a young girl with a rare, fatal, neurodegenerative condition called Batten disease, for whom the Yu Lab developed an experimental ASO therapy personalized to her specific genetic variant.
Read MoreTim presented our work on patient-customized oligonucleotide therapy in the Late Breaking Abstract Plenary Session. Cynthia presented a poster (and winner of a Readers’ Choice Award!) on her Rapid Exome NICU Sequencing project.
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