ARVO 2024

My research focuses on the use of CRISPR/Cas9 gene editing in Best disease, a rare inherited form of macular degeneration. My specific research interests include stem cells in disease modelling, retinal pigment epithelium (RPE) function and dysfunction, and the influence of genes in disease. I was extremely grateful to be awarded a travel grant by the ARVO foundation, and this article documents my own experience in the beautiful Seattle, as a first-time ARVO attendee.

Sunday 5 May: My first day at ARVO: Genome editing, hydrogels and the immune system
Sunday morning marked the first day of the conference, and we arrived bright and early at Seattle Convention Centre, where crowds of researchers had already started to gather. I was blown away by the sheer size of the place, with two enormous buildings connected by a skybridge, reported to hold around 11,000 ARVO attendees. I was buzzing with anticipation to start the day, and had already selected the talks I would attend through the handy ARVO app.

My day began with a session on “Advancing therapeutic strategies for neurodegenerative blindness”, and the room was bustling with people, many even standing, as Kryzysztof Palczewski took the podium for a talk on genome editing in the eye. He emphasised the importance of transient expression of the CRISPR/Cas9 machinery in the eye, and presented the favourability of virus-like particles and cell penetrating peptides for genome editing. I left the talk feeling inspired, with a deepened understanding of the many potential therapeutic strategies for inherited retinal disease, as well as the pitfalls associated with each.
The afternoon involved frantic dashing across exhibit halls to catch talks in different buildings – all part of the authentic ARVO experience. In the “Current clinical trials and novel approaches for treatment of genetic disorders” session, I enjoyed an incredible talk from Brittni Scruggs on the ability of fibrin hydrogel-encapsulated adeno-associated virus to transduce RPE when placed on top of the retina in a pig model. I was excited by this concept, as our genome editing approach is dependent on the efficient delivery of CRISPR/Cas9 machinery to the RPE, so novel delivery strategies are crucial.
I then ventured to a session entitled “ImmunoQuest: Journey into the intricacies of immune responses in ocular diseases” to attend a talk from Evangeline Foster, a postdoctoral researcher in the Carr Lab. Evie discussed her work on the development of an in vitro platform to model the involvement of the immune system in age-related macular degeneration (AMD).  

Evangeline Foster presenting her talk.

Monday 6 May: My poster session, and diet in AMD
On the second day of the conference, I was scheduled to present my poster. For me, this was another milestone: it was my first time presenting a poster at an international conference. I arrived early to mount it, and the poster session on “Stem cells and therapeutic development” began at 8.30am. Positioned at the front of the vast poster hall, my poster was a popular one, and I enjoyed talking people through my research, answering their questions, and taking on board their suggestions. I love sharing my knowledge and expertise, so it delighted me that people working with similar techniques were even asking me for advice on their protocols.

Zaynab Butt presenting her poster at ARVO.

Feeling energised from talking science, I made my way over to the ballroom, where the Proctor Award Lecture was given by Emily Y Chew, MD, FARVO. The introduction shed light on her incredibly successful career, with 541 peer-reviewed papers, and 80,361 citations. As chair of the Age-Related Eye Disease Study 2 (AREDS2), a phase 3 study that investigates the use of lutein/zeaxanthin and omega-3 fatty acids as AMD treatment, Emily stressed the significance of diet in AMD risk. Eating fish was shown to reduce risk of AMD by 31%, as well as decrease the risk of drusen progression in those with early AMD. It was powerful to hear the profound consequences of what we eat from a leading expert in the field, and inspired by the talk, I headed off to lunch with a new-found craving for salmon.

Emily Chew giving the Proctor Award lecture.
 

In the afternoon, I attended a session on genes and their influence in disease, in which Freddie Braddock, a PhD student in Alison Hardcastle’s lab delivered a great talk describing a novel autosomal dominant Corneal Stromal Dystrophy associated with a SPARCL1 missense variant 

Freddie Braddock presenting his talk.

Tuesday 7 May: Stem cells and gene therapy
I had a later start on Tuesday, spending the morning perusing the exhibit halls and networking with people at their posters. My afternoon was spent in a session on stem cells and gene therapy, where talks centred around clinical trials of AAV gene therapies in various congenital eye diseases. Dominik Fischer presented the year 3 interim results on the safety and efficacy of voretigene neparvovec (VN), the first available gene therapy for patients with RPE65-associated retinal dystrophies. The data showed that VN is both safe and effective in patients. This was uplifting news and showed that gene therapies can be used to treat inherited ocular disease.
I then moved to a session on the use of patient-derived stem cells to model and treat inherited retinal disease. This session included a talk from Michael Whitehead, a postdoctoral researcher in Mike Cheetham’s lab, on the pathogenic OPA1 variants disrupting calcium homeostasis in iPSC-derived retinal ganglion cells, increasing their susceptibility to cell death. 

Michael Whitehead.

Wednesday 8 May: More genome editing approaches
My first session of the day was a series of talks on therapeutic approaches for inherited retinal disease. The session was a busy one, so I was quietly glad I had arrived early to find a seat near the front. It opened with an exciting talk from Bikash Pattnaik, who talked about the CRISPR VISION Program, an academic-industry partnership that utilises a non-viral genome editing approach to treat congenital blindness. This talk was followed by Ana Alonso-Carriazo Fernandez, another postdoctoral researcher in my lab, who delivered a fantastic talk on genome editing as a therapeutic for Late-Onset Retinal Degeneration. 

Ana Alonso-Carriazo Fernandez.
 

The ARVO social took place in the evening, with a fun-filled karaoke night at the Museum of Pop Culture. The museum was free to explore for the event, so it was a great opportunity to relax, take a break from a science, and have fun with colleagues.

Thursday 9 May: Closing remarks (and more posters)
After the previous night’s festivities, I arrived a little later at the conference centre for the morning poster session. I even managed to find a few posters on Best disease and spent some time chatting with the presenters about our shared experiences researching the same condition. I dropped in to visit my PI, Amanda Carr, who was presenting her poster on the effects of modifying culture conditions on ARPE-19 cells.

PI Amanda Carr presenting her poster.

The closing keynote was given by Sheril Kirshenbaum, an award-winning science writer, author, and scientist, who hosts the show “Serving Up Science” on PBS. Her inspirational talk drove home the need for effective science communication, with ideas on how to connect with different audiences and counter the rise of misinformation. As an aspiring science communicator, her mission to enhance public understanding of science is one that resonated with me, and I found it to be a perfect close to the conference

Sheril Kirshenbaum in the Closing Keynote lecture.

Reflecting on my experience, I recognised that science communication is integral to our work, not only to disseminate information to the public, but to promote the exchange of ideas between scientists. International conferences like ARVO are a hub for engaging with fellow researchers, sharing our expertise, starting new collaborations, celebrating each other’s successes, and acknowledging that we are all working towards the same goal. With such a positive first experience, I look forward to attending more ARVO meetings in the future.

Links

• UCL IoO at ARVO 2024
• Zaynab Butt
• Carr Lab
• ARVO