The Translational Neuroscience Center strives to accelerate the development of therapeutic options for children with brain disorders by fostering timely and efficient translational research.
The Translational Neuroscience Center strives to accelerate the development of therapeutic options for children with brain disorders by fostering timely and efficient translational research.
Our Advocacy and Research Partners
The Translational Neuroscience Center’s alliances with patient advocacy groups, community partners and other research organizations are fundamental to our mission. Combining our knowledge, commitment and resources, we accelerate translational research, convene scientific and family meetings, and reflect patient and caregiver priorities in our program design.
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Featured News & Events
Boston Children’s receives $35 million donation to accelerate development of therapeutic options for children with brain disorders
The Translational Neuroscience Center is thrilled to share the exciting news that our Center has received a $35 million donation from Hansjoerg Wyss, through the Wyss Medical Foundation, to accelerate the development of new therapeutic options for children with neurodevelopmental disorders.
Development of a Patient and Caregiver-Centered Pediatric Disease Concept Model for Kleefstra Syndrome
In collaboration with RSZ TNC researchers and support from IDefine – The Kleefstra Syndrome Foundation, a graduate student from the Boston University Genetic Counseling Program, Kristen Connors, developed a disease concept model for Kleefstra syndrome (KS). This patient and caregiver-centered research identified unique aspects of the condition that most greatly impact the patient population and their families. This data may be used to generate educational materials for families and clinicians, and direct the eventual development of KS-specific interventional treatments that target features that are most important to the KS community.
Base editing of trinucleotide repeats that cause Huntington’s disease and Friedreich’s ataxia reduces somatic repeat expansions in patient cells and in mice
RSZ TNC and Broad institute researchers utilize base editing to reduce the repetitiveness of trinucleotide repeats that cause Huntington’s Disease and Friedeich’s Ataxia.