| Time | Session |
|---|---|
| 2:00pm – 2:10pm | Welcome Heather Gray-Edwards, DVM, PhD Co-chair, NTSAD Scientific Advisory Council |
| 2:10pm – 2:50pm | Keynote: Advances in the Therapeutic Application of CRISPR/Cas9 for In Vivo Genome Editing Sean Burns, MD Vice President, Disease Biology, Intellia Therapeutics |
| 2:50pm – 3:20pm | Preclinical programs with an eye to “compassionate use”. Experiences from N=1 INDs. Lauren Black, PhD Distinguished Scientist, Charles River |
| 3:20pm – 3:30pm | Break |
| 3:30pm – 4:00pm | In Vivo Base Editing of the Human Late-Onset Tay Sachs Disease MutationA change in the sequence of DNA. Many mutations are "silent" and do not cause disease. When mutations occur in genes and disrupt the production of a functional protein, they may lead to genetic disease. More Delays Symptom Onset and Substantially Prolongs Life-Span Rick Proia, PhD Senior Investigator, Genetics and Biochemistry Branch, NIDDK |
| 4:00pm – 4:30pm | Rewriting the Code: Pioneering Individualized GeneOften referred to as the "unit of heredity." A gene is composed of a sequence of DNA required to produce a functional protein. More Editing Techniques for Tay-Sachs Disease Treatment Zhenya Ivakine, PhD Scientist, Program in Genetics and Genome Biology, The Hospital for Sick Children, Toronto |
| 4:40pm – 5:00pm | Discussion: How do we move gene editing for Tay-Sachs and Sandhoff into the clinic? |
Translating preclinical CRISPR editing in Tay-Sachs and Sandhoff rodent models to human clinical trialsMichael2023-05-12T02:09:24+00:00
