Tim W. Yu, M.D., Ph.D.
|Title||Assistant Professor of Pediatrics|
|Institution||Boston Children's Hospital|
300 Longwood Ave
Boston MA 02115
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|1990 - 1994||John Harvard Scholar|
|1990||Robert C. Byrd Scholar|
|1993 - 1994||Barry M. Goldwater Scholar|
|1994 - 2003||Medical Scientist Training Program|
|1999 - 2001||MIND Institute Fellow|
|2000||Achievement Rewards for College Scientists|
|2008 - 2010||Fellow, Clinical Investigator Training Program|
|2010 - 2012||Nancy Lurie Marks MeRiT Fellow|
|2013||Children's Hospital Pediatric Associates Award|
Available: 07/05/17, Expires: 07/01/18
Rapid advances in human genetics are allowing us for the first time to identify genes responsible for autism spectrum disorders. Our lab has identified a set of genes subject to biallelic mutation -- if you will, "human knockouts" -- that lead to autism. The medical student will have an opportunity to investigate the molecular and neurobiological mechanisms responsible for these effects, using cellular and animal models.
Available: 07/01/17, Expires: 07/01/18
Recent advances in genomic and transcriptomic engineering allow one to alter DNA, RNA and splicing with remarkable precision. These techniques open the door to new genetically specific therapies and are revolutionizing the management of previously untreatable conditions like Duchenne Muscular Dystrophy. We are developing an approach to personalizing treatments for *individual* patients with genetic disease, using antisense oligonucleotides customized to each case (ie, true "N of 1" therapy). The project involves a detection component (deploying sequencing strategies to identify suitable patients with mutations amenable to this approach) and a therapeutic component (designing and testing the therapeutic approach in cellular models). Previous experience in genomics and molecular biology will be helpful but not required.
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