Towards Targeted Therapies for Neurodevelopmental Disorders

July 31, 2020 via Zoom

Co-hosted by:

Ethan M. Goldberg, MD, PhD, The Children’s Hospital of Philadelphia Epilepsy NeuroGenetics Initiative
& Anastasios Tzingounis, PhD, The University of Connecticut Department of Physiology and Neurobiology

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About the Symposium

Towards Targeted Therapies for Neurodevelopmental Disorders 2020 is a one-day online/virtual symposium highlighting cutting-edge advances in neuroscience, neurology, genetics, and development related to epilepsy, autism, intellectual disability, psychiatric disorders, and related conditions. Please join us to hear from a spectacular panel of speakers using a range of approaches from genetics, to molecules, circuits, and systems. The goal of the symposium is to bring the community together at a time when many scientific conferences have been cancelled.
 
The symposium will be organized into two Talks (40 min +10 min for moderated Q&A), four themed Minisymposia with three speakers per session (25 +10), followed by themed breakout sessions for short talks by students and postdocs (10 +2). Those interested in signing up for a short talk can indicate this in the registration.
 

Schedule

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10:00am – 10:05am

Welcome / Introduction

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10:05am – 10:45am

Followed by 10-minute Q&A 

Speaker #1

Guoping Feng, PhD

James W. and Patricia T. Poitras Professor of Brain and Cognitive Sciences at MIT
Director, Model Systems and Neurobiology, Stanley Center for Psychiatric Research
Institute Member, Broad Institute of Harvard and MIT

“Exploring therapeutic strategies for ASD: from genes to circuits”

Guoping Fend

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10:55am – 12:10pm

Followed by 10-minute moderated panel discussion

Minisymposium #1 – Human Genetics to Inform Treatment Targets

Heather Mefford MD, PhD, University of Washington

Christel Depienne, PhD, Institut für Humangenetik, Universitätsklinikum Essen, Germany

Gemma Carvill, PhD, Northwestern University Feinberg School of Medicine
“Poison exons in epilepsy” 

Moderator: Katherine Helbig, MS, LCGC, CHOP

Heather MeffordChristel DepienneGemma CarvillKatherine Helbig

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12:20pm – 1:35pm

Followed by 10-minute moderated panel discussion

Minisymposium #2 – Targeted Therapy for Neurodevelopmental Disorders

Michelle Gray, PhD, University of Alabama at Birmingham
“Using Preclinical Models of Huntington’s Disease in Pursuit of Therapeutics”

Gaia Colasante, PhD, Ospedale San Raffaele, Milan, Italy
”CRISPR/dCas9 mediated Scn1a gene upregulation as a therapeutic approach in Dravet Syndrome”

Elaine Hsiao, PhD, UCLA
“Microbiome-diet interactions for refractory epilepsy”

Moderator: Ethan Goldberg, MD, PhD

Michelle GrayGaia ColasanteElaine HsaioEthan Goldberg

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1:45pm – 2:15pm

Break

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2:15pm – 3:00pm

Followed by 10-minute Q&A 

Speaker #2

Arnold Kriegstein, MD, PhD

John G. Bowes Distinguished Professor in Stem Cell and Tissue Biology
Director, The Eli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research, UCSF School of Medicine

“Cell type vulnerabilities in early human cortical development”

Arnold Kriegstein

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3:10pm – 4:25pm

Followed by 10-minute moderated panel discussion

Minisymposium #3 – Mechanisms of Neurodevelopmental Disorders

Helen Bateup, PhD, UC Berkeley
“Synaptic changes in the basal ganglia drive autism-related behavioral alterations”

Brady Maher, PhD, Lieber Institute for Brain Development and Johns Hopkins University

Jess Cardin, PhD, Yale University

Moderator: Ethan Goldberg, MD, PhD, CHOP/UPenn

Helen BateupBrady MaherJess Cardin

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4:35pm – 5:50pm

Followed by 10-minute moderated panel discussion

Minisymposium #4 – Mechanisms of/Treatment for Ion channelopathies

Kevin Bender, PhD, UCSF
“Towards therapeutics for loss-of-function in SCN2A”

Matt Weston, PhD, University of Vermont

Jeanne Paz, PhD, UCSF

Moderator: Tasso Tzingounis, PhD, University of Connecticut

Kevin BenderMatt WestonJeanne PazTasso Tzingounis

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6:00pm – 6:15pm

Break

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6:15pm – 7:15pm

Parallel Virtual Poster Sessions

Poster Session #1 – Experimental models of neurodevelopmental disorders
Poster Session #2 – Human genetics
Poster Session #3 – Gene therapy
Poster Session #4 – Ion channelopathies

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7:15pm – 8:15pm

Virtual Happy Hour in Breakout Rooms

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Towards Targeted Therapies for Neurodevelopmental Disorders 2020 will be organized into two Talks (40 min +10 min for moderated Q&A), four themed Minisymposia with three speakers per session (25 +10), followed by themed breakout sessions for short talks by students and postdocs (10 +2). Those interested in signing up for a short talk can indicate this in the registration. Signup for the short talks will close at 5 pm on 7/15.
 

Registered Attendees as of 6/27

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