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The article: Association of Messenger RNA Level With Phenotype in Patients With Choroideremia: Potential Implications for Gene Therapy Dose Background The central dogma of biology is simply that DNA makes RNA makes protein. Messenger RNA (mRNA) transfers the information from DNA and is used to create protein. The question this study ultimately seeks to answer is “How much mRNA is needed to have healthy retinal function in CHM patients?” It is important to know this information when conducting gene therapy clinical trials so an effective “dose” can be delivered....

Washington, DC - Nov. 6, 2019 The American Society of Gene and Cell Therapy held a 3 day summit to discuss policy and other issues around gene editing and gene therapy. Day 1 addressed regulatory issues in gene therapy development Day 2 dealt with payment policies around gene therapy Day 3 (the day I attended) was focused on the ethical, societal, and policy issues around germline gene editing Links to some of the presentations can be found on ASGCT’s website....

Panorama view from Whistler Mountain. These notes from Thursday May 2, the last day of ARVO 2019. The conference had thinned out a bit as some people had to leave, but there were still some presentations of interest. You can read a summary or my notes from the other days here: Friday, Sunday, Monday, Tuesday, and Wednesday. Papers Subfoveal gene augmentation therapy for choroideremia: One-year results from a Phase I/II trial of AAV2-hCHM - Rachel M....

“Digital Orca” sculpture outside of the Vancouver Convention Center. These notes from May 1 are the continuation of a few articles about my time at ARVO. You can read a summary or my notes from the other days here: Friday, Sunday, Monday, Tuesday. Papers AAV2-hCHM Subretinal Delivery to the Macula in Choroideremia: 2 year Results of an Ongoing Phase I/II Gene Therapy Trial - Thomas Aleman Aleman presented more follow-up data from this phase I/II clinical trial that was performed on 10 CHM patients....

ARVO 2019 took place at the Vancouver Convention Center from April 28 to May 2 and featured novel work, continued efforts, and updates on clinical trials. On the Friday before ARVO the Foundation Fighting Blindness and the Casey Eye Institute hosted a summit featuring gene therapy, stem cell therapies, and non-viral therapies. Randy Wheelock had been organizing a special interest group on Sunday to discuss combination therapies before his passing....

ARVO poster area B. Roughly 1,000 new posters are displayed every day of ARVO. See my notes from Sunday and Monday. Patient Engagement in Ophthalmology Research This special interest group discussed current methods of patient involvement in research and explored new ways this could be improved. A special emphasis was put on international policies, specifically in Europe and Canada. BC SUPPORT is one of the branches of the SPOR initiative across Canada meant to include patients and caregivers in research design....

The Vancouver Convention Center’s East Building These are my notes from the second day at ARVO 2019. Check out Sunday’s notes if you missed them. Changing What it Means to be Blind: We’re All in This Together This session took place late in the evening and was put together by Kristin Smedley from the Curing Retinal Blindness Foundation. Smedley began the session by playing a TEDx talk she gave in 2017 at Lincoln Square....

The Vancouver Convention Center’s West Building A few words Similar to my article about the FFB Innovation Summit this post is a gathering of my notes from ARVO 2019. I will not be explaining topics, instead this is meant to be a reference for myself and others within the CRF. However I want these notes to be available to everyone since I think it is vitally important for those affected by disease to be invited into the discussion about research....