Research Spotlight: Gene Therapy Improves Brain Function in Lab Models of Mucolipidosis IV
Mucolipidosis IV (MLIV) is a rare neurologic disease of childhood with extremely high unmet need.
Contact Information
Ziv Williams, MD
Wang Ambulatory Care Center
WAC 745K
15 Parkman Street
Boston,
MA
02114
Phone: 617-643-4102
Email: zwilliams@partners.org
Despite continued scrutiny into the single-neuronal correlates of motor and cognitive behavior, remarkably little remains known about the mechanisms by which information is communicated between neurons locally or across cortical areas. Such communication is central to neuronal functioning at a systems level, yet remains among the most poorly understood topics in neuroscience.
The focus of the Neuronal Communication and Restoration Lab is to interrogate multiple-neuronal ensembles across interconnected cortical areas, and examine the manner by which communication between neurons correlates with ongoing behavior.
We are specifically targeting tasks that engage the acquisition of dynamic motor responses, and are examining the underlying neuronal processing that allow such information to be "transferred" from one area within the CNS to the other. These and similar findings may answer a number of basic questions about the process by which multi-modal information is shared between cortical areas and how concerted neuronal activity may instruct downstream areas responsible for cognitive and executive control.
Despite intense scientific effort, there remains virtually no effective treatments for reconstituting the "line of communication" across areas damaged within the CNS. The second main aim of the lab is to develop new approaches for transforming recorded neuronal activity into signals that can be used to drive intact downstream areas within the CNS such as the spinal cord. Our group and others had previously demonstrated that ensemble neuronal activity may accurately predict movement intention, and that delivery of event-related electrical stimuli in areas responsible for motor production can reproducibly alter targeted limb movement. Our current goal is to exploit these properties in order to systematically dissociate and approximate movement production with motor intent. Such findings may offer alternate strategies leveraged to circumvent damaged areas within the CNS, and provide a unique perspective into the individual roles that motor cortical neurons and spinal efferent activity play in adaptive motor behavior.
Mass General is the #1 research hospital in America, with a large percentage of that dedicated to Neuro-related diseases.
Mucolipidosis IV (MLIV) is a rare neurologic disease of childhood with extremely high unmet need.
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