13th International VHL Medical Symposium Selected Presentation - PDF document

13th International VHL Medical Symposium Selected Presentation Summaries previous 2 days, data from MyVHL was presented, The 13 th International VHL Medical Symposium and VHL Alliance programs, including support held in Houston, TX on October 4-6, 2018 brought groups, CCC Process Improvement and Wellness together VHL researchers, clinicians, and patients Coaching were discussed. from around the world. The symposium was a great way for researchers to have the opportunity Three major outcomes were identified: to meet and interact with physicians treating VHL, 1. Identify additional data and tools needed to and with VHL patients and families. Most assess the feasibility of genetic therapy for VHL importantly, it was a venue to discuss the retinal eye disease. tremendous advances that have been made in 2. Form Task Force to reevaluate VHL research that is bringing us closer to effective Surveillance Guidelines and determine any treatments for VHL. necessary changes. An entire day was devoted to presentations and 3. Publish meeting outcome summary in Clinical discussions directed to VHL patients, their families, Cancer Research (CCR) Journal. and friends. In addition to lay summaries from the CONTENTS 1. Novel Insights into VHL Biology ..................................................................................................................... 4 Signaling in ccRCC: Molecular Mechanisms and Targeted Therapy .................................................................................... 4 Erin Bruno Rankin, PhD ................................................................................................................................................................ 4 Identifying Novel Therapeutic Targets for VHL Disease Downstream of a Unique VHL-AURKA-HDAC6 Signaling Axis . 4 Ruhee Dere, PhD ........................................................................................................................................................................... 4 VHL Substrate Transcription Factor ZHX2 as an Oncogenic Driver in RCC ......................................................................... 4 Qing Zhang, PhD ........................................................................................................................................................................... 4 The HIFs in Kidney Cancer – New Insights and Targeting Potential .................................................................................... 5 Mei Koh, PhD ................................................................................................................................................................................. 5 Signaling in ccRCC: Molecular Mechanisms and Targeted Therapy .................................................................................... 5 Edward LaGory, PhD ..................................................................................................................................................................... 5 Multiple Tumor Suppressors Regulate a HIF-Dependent Negative Feedback Loop Through ISGF3 in Kidney Cancer ... 5 Haifang Yang, PhD......................................................................................................................................................................... 5 2. Gene Editing: Fact or Friction for VHL Disease ............................................................................................. 6 AAV Targeted Therapy for Inherited Retinal Dystrophy: Bench to Bedside ....................................................................... 6 Daniel Chung, DO, MA .................................................................................................................................................................. 6 Cas9 Mediated Therapy .......................................................................................................................................................... 6 Giannicola Genovese, MD ............................................................................................................................................................ 6 13 th International VHL Symposium, 2016 1 Houston, TX Presentation Summaries