Understanding autism and other neurodevelopmental disorders: From the embryo to brain organoids

  • Autism Research
Speaker Paola Arlotta, Ph.D.
Harvard University
Date & Time


Location

Gerald D. Fischbach Auditorium
160 5th Avenue
New York, NY 10010 United States

Autism Research

Autism Research lectures bring together scientists and scholars to discuss diverse and important topics related to autism.

Video replay of this event will be available shortly. Please check back at a later date.

On 1 November 2017, Paola Arlotta focused on the cerebral cortex and presented the challenges and opportunities of modeling human brain development using pluripotent stem cells within 3D human brain organoids. Building on developmental work in mice, such organoids promise a better understanding of complex neurodevelopmental conditions such as autism spectrum disorder.

Her talk was part of the Simons Foundation Autism Research lecture series.

About the Lecture

The cellular and molecular processes that control brain development in mammals, as well as how neurodevelopmental disorders affect such processes, remain poorly understood. The recent advent of brain organoids (lab-grown clusters of living neurons) offers new ways to study the relationships between neurodevelopmental disorders and brain development.

In this lecture, Paola Arlotta focused on the cerebral cortex and presented the challenges and opportunities of modeling human brain development using pluripotent stem cells within 3D human brain organoids. Building on developmental work in mice, such organoids promise a better understanding of complex neurodevelopmental conditions such as autism spectrum disorder. She discussed her recent work on the generation and long-term development of human brain organoids and the study of their developmental trajectories, cellular diversities and neuronal network features.

Arlotta then showed that phenotypic abnormalities observed in patients carrying genetic mutations linked to autism spectrum disorder can be reproduced within brain organoids. This work serves as a proof-of-principle demonstration that selected aspects of human neurodevelopmental conditions can be modeled in vitro with the goal of better understanding disease genetics and pathology.

About the Speaker

Paola Arlotta is a professor of stem cell and regenerative biology at Harvard University. She is a principal faculty member at the Harvard Stem Cell Institute, where she is co-director of the neuroscience program, an institute member at the Broad Institute, and an associate member of the Stanley Center for Psychiatric Research at the Broad Institute. She is interested in understanding the molecular laws that govern the birth, differentiation and assembly of the human brain’s cerebral cortex. The complexity of the brain fascinates her and she is driven to integrate developmental and evolutionary knowledge to investigate therapies for brain repair and for modeling neuropsychiatric disease. Arlotta received her M.S. in biochemistry from the University of Trieste, Italy, and her Ph.D. in molecular biology from the University of Portsmouth in the UK. She subsequently completed her postdoctoral training in neuroscience at Harvard Medical School.

Past Lectures

How emotions shape our memories

Kelsey C. Martin, M.D., Ph.D.Director, SFARI
Director, Simons Foundation Neuroscience Collaborations
Leonard Mlodinow, Ph.D.Physicist and Author

Have you ever contemplated the difference between a feeling, a thought and a memory? And how do all these things fit together in making us who we are?

Leonard Mlodinow is a theoretical physicist and best-selling author. In his latest book, “Emotional: How Feelings Shape Our Thinking,” he unpacks the role emotions play in our thinking and mental well-being.

Kelsey Martin, director of the Simons Foundation Autism Research Initiative (SFARI) and the foundation’s neuroscience collaborations, has spent much of her career as a neuroscientist seeking to understand better how experiences change brain connectivity to store long-term memories.

What do we mean by ‘autism risk genes’?

David Ledbetter, Ph.D.
Chief Clinical Officer, Dascena

Joseph Buxbaum, Ph.D.
Director, Seaver Autism Center
Professor, Psychiatry, Neuroscience, Genetics and Genomic Sciences
Vice Chair for Research and Vice Chair for Mentoring, Psychiatry, Icahn School of Medicine at Mount Sinai

Heather Mefford, M.D., Ph.D.
Full Member, St. Jude Children’s Research Hospital

David Ledbetter and Joseph Buxbaum discussed whether there are genes for which mutations confer risk specific to autism or whether these genes are really conferring general risk of disrupted brain development. The discussion was moderated by Heather Mefford.

Small molecules, genes and antisense oligonucleotides: Industry perspectives on treatment development for ASD

Federico Bolognani, M.D., Ph.D.
Vice President, Head of Clinical Science, Axial Therapeutics

Stuart Cobb, Ph.D.
Chief Scientific Officer, Neurogene; Research Fellow, University of Edinburgh

Yael Weiss, M.D., Ph.D.
Vice President, Business Development, Ultragenyx

Randy Carpenter, M.D.
Chief Medical Officer, Rett Syndrome Research Trust; Co-Founder, Allos Pharma

Federico Bolognani, Stuart Cobb, and Yael Weiss joined a panel to discuss new industry developments on the use of small molecules, gene therapy and antisense oligonucleotides as treatment approaches for autism spectrum disorders (ASD). The panel discussion was moderated by Randall Carpenter.

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