Steven Gray

Company: University of Texas Southwestern Medical Center

Job title: Associate Professor

Seminars:

Rett Syndrome as a Case Example of Gene Dosage Sensitivity: miRARE-mediated Feedback Regulation as a Possible Solution 9:10 am

Overview of AAV9 gene therapy Is AAV9 good enough? When building your AAV, how will you control dosage? Evaluating dose sensitivity strategies and efficacy data using Rett syndrome as a case study What is the best formulation to yield better biodistribution?Read more

day: Day Two

Panel Discussion – Diving into Specific Therapeutic Modalities 10:40 am

Advantages and disadvantages across small molecules, gene therapy, antisense oligonucleotides, gene replacement, stem cell therapy and hybrid strategies With each modality, what needs to happen before the trial, concurrently with the trial and after? What’s the longevity of these approaches? What are the knock on effects of taking these genes out? What is a good…Read more

day: Day Two