2019 Scientific Symposium

2019 SLC6A1 Connect International Conference
Schedule (.pdf)

SLC6A1 Connect Update
Speaker: Amber N. Freed, Organization: SLC6A1 Connect

The Clinical Presentation of SLC6A1
Speaker:
Katherine L. Helbig, MC, LCGC, Institution: Children’s Hospital of Philadelphia
Slides (.pdf)

Simons Searchlight Registry Findings for SLC6A1
Speaker:
Jennifer Bain, MD, PhD, Institution: Columbia University Medical Center
Slides (.pdf)

Characterization of Variant Tolerant and Intolerant Sites in SLC6A1 Dysfunction
Speaker:
Dennis Lal, PhD, Institution: Cleveland Clinic & Broad Institute
Slides (.pdf)

Celluar In Vitro Modeling for SLC6A1
Speaker:
Julieth Sierra Delgado, D, MD, M.Sc., Institution: Nationwide Children’s Hospital
Slides (.pdf)

Mouse Models for SLC6A1
Speaker:
Frances Shaffo, PhD, Institution: University of Texas Southwestern
Slides (.pdf)

GABAergic Systems and the Role of GAT1
Speaker:
Amy Brooks-Kayal, MD, Institution: Children’s Hospital of Colorado
Slides (.pdf)

Model SLC6A1 Mutation Associated Disorders from Stem Cells to Mice
Speaker:
Katty Kang, MD, PhD, Institution: Vanderbilt University
Slides (.pdf)

Pharmacological Thoughts for SLC6A1
Speaker:
Michael Rogawski, MD, PhD, Institution: University of California, Davis
Slides (.pdf)

Potential Drug Targets for SLC6A1
Speaker:
Ana C. Puhl, PhD, Institution: Collaborations Pharmaceutical
Slides (.pdf)

Flourescent Sensors for Neuroscience
Speaker:
Jonathan Marvin, PhD, Institution: Howard Hughes Medical Institute
Slides (.pdf)

Anti-Sense Oligonucleotide Treatment
Speaker: Athma Pai, PhD, Institution: University of Massachusetts
Slides (.pdf)

Stoke’s TANGO Upregulation Update
Speaker:
Gene Liau, PhD, Institution: Stoke Therapeutics
Slides (.pdf)

Gene Replacement Therapy
Speaker: 
Steven Gray, PhD, Institution: University of Texas Southwestern
Slides (.pdf)

Considerations and Approaches for GMP Manufacturing of Viral Vectors for Gene Therapy
Speaker:
Nick Conley, PhD, Institution: Locus Biosciences
Slides (.pdf)