Agenda

Gene Therapy for Sensory Disorders

Wednesday, April 11, 2018

8:30

Coffee and Pastries

   

9:00

Opening Remarks
George Q. Daley (Dean, Harvard Medical School) and David P. Corey (Bertarelli Professor of Translational Medical Science, Harvard Medical School)

   

9:15

Restoration of Hearing in the Otoferlin KO Mouse Using Dual AAV Vectors
Omar Akil (University of California San Francisco)

   
9:45

Keynote Lecture - Treatment of Autosomal Dominant Hearing Loss by In Vivo Delivery of Genome Editing Agents
David Liu (Professor of Chemistry and Chemical Biology, Harvard University and Vice-Chair of the Faculty, Broad Institute)

10:45 Break

11:15

New AAV Vectors for Gene Addition Therapy
David P. Corey (Bertarelli Professor of Translational Medical Science, Harvard Medical School)

   

11:45

Novel Gene Therapy Approaches for Hereditary Hearing Loss
Jeffrey R. Holt (Boston Children's Hospital and Harvard Medical School)

12:15 Lunch

13:15

Keynote Lecture - Restoring Vision
Botond Roska (Co-Director, Institute of Molecular and Clinical Ophthalmology, Basel)

   

14:15

Optical Imaging of the Inner Ear for Diagnosis and Therapy of Hearing Loss
Konstantina M. Stankovic (Massachusetts Eye and Ear and Harvard Medical School)

Next Generation Auditory Brainstem Implants (ABIs): Translation to Clinical
Implementation

Daniel J. Lee (Massachusetts Eye and Ear and Harvard Medical School) and Stéphanie P. Lacour (École polytechnique fédérale de Lausanne)

Imaging Brain Networks in Children with Autism
Dara S. Manaoch (Massachusetts General Hospital and Harvard Medical School) and Dimitri Van De Ville (École polytechnique fédérale de Lausanne)

15:15 Break

15:45

Using Gene Modified Hematopoietic Stem Cells to Treat Cerebral Adrenoleukodystrophy
David A. Williams (Boston Children's Hospital and Harvard Medical School)

   

16:15

Stem Cell and Gene Therapy for Retinal Degeneration
Yvan Arsenijevic (Jules-Gonin Eye Hospital)

16:45

Keynote Lecture - Overview of the Development Program for Voretigene Neparvovec: Challenges and Solutions
Katherine A. High (President and Chief Scientific Officer, Spark Therapeutics)

   
17:45 Closing Remarks
   
18:15 Reception