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Introduction from Chair - Michael E. Goldberg, MD
Critical Periods and Brain Development - Takao K. Hensch, PhD
Mature brain function is potently shaped by the surrounding environment during infancy and early childhood. Biological constraints carefully orchestrate such windows of brain plasticity that are now being unraveled at the interface of molecular and systems neuroscience. Understanding when and how complex neural circuits are sculpted by experience carries an impact far beyond the lab, including education policy, therapeutic approaches to mental illness or recovery from brain injury in adulthood.
Seeing is Believing: A Gene Therapy Success - Jean Bennett, MD, PhD
Gene therapy has restored sight in patients with Leberís Congenital Amaurosis (LCA), an inherited blindness that begins at birth. This presentation will describe the safety and efficacy results after gene therapy in 12 subjects born with LCA. The subjects were treated at The Children’s Hospital of Philadelphia. The subjects include five children and seven adults who have been followed now for more than 3.6 years post treatment. All of the children enrolled in the study are now able to navigate independently, read and write, take part in normal classroom activities, and participate in sports. Adults in the study also have more independence due to improvements in their vision. This the first pediatric human gene therapy study involving a non-lethal disease, and it is also the first gene therapy study of treatment of inherited retinal degeneration in which pediatric subjects have been enrolled. The success of this gene therapy study provides the foundation for gene-based approaches for restoring vision in other, more common forms of blindness.
Project Prakash: Sight and Insight - Pawan Sinha, PhD
The translation from basic science to societal impact often takes many years. However, in some instances, even the conduct of basic research can yield tangible benefits. I shall describe an effort that perhaps fits in this category. Named ‘Project Prakash’, this initiative provides sight to blind children on the one hand and helps address several fundamental questions regarding brain plasticity and learning on the other.
Michael E. Goldberg is the David Mahoney Professor of Brain and Behavior in the Developments of Neuroscience, Neurology, Psychiatry, and Opthalmology at Columbia University College of Physicians and Scientists.
Takao K. Hensch is Professor of Molecular Cellular Biology and Neurology at Harvard University and Children's Hospital Boston.
Jean Bennett is Professor of Ophthalmology and Cell and Developmental Biology. She is the F.M. Kirby Center for Molecular Ophthalmology at the Scheie Eye Institute, University of Pennsylvania.
Pawan Sinha is Professor of Vision and Computational Neuroscience at MIT.