Good afternoon everybody I’d like to welcome you to the third Retina Australia webinar for 2023 vision loss priority setting partnership and an introduction to stem cell and Gene therapies my name is Sally Turnbull and I am the administrative officer for Retina Australia and I will be the facilitator of this webinar as a participant your camera and microphone will be switched off throughout there will be an opportunity for you to ask questions once both our speakers have finished their presentations if you have any queries during the proceedings you can type a question to me in the Q&A section our first Speaker this afternoon is Dr Eden Robertson Dr Robertson is an experienced psychosocial researcher with a special interest in supporting children with a serious illness and their families she has completed a bachelor of psychology honors graduate certificate in Adolescent and young adult health and well-being and a PhD in Childhood Cancer in 2019 she is currently a post-doctoral research fellow at UNSW Sydney focused for the past decade on addressing the psychosocial impact and challenges of several rare diseases including Childhood Cancer Developmental and epileptic encephelopathy and inherited retinal diseases alongside this role she is also the national research evaluation and impact manager for red kite a national Childhood Cancer support charity providing access to free practical emotional and financial support to help families take on the challenge of Childhood Cancer in 2022 Dr Robertson undertook a prestigious Fullbright scholarship at St Jude Children’s Research Hospital to further her skills in palliative care and best practice in supporting families at their child’s end of life she is passionate about Empowering Families to be more involved in their child’s care as well as involving consumers in research in this webinar Dr Robinson will present on her vision loss priority set setting partnership and the process they will undertake to arrive at the top 10 research priorities for inherited retinal diseases so I’ll just hand over to Eden now thank you so much Sally um it’s always a bit uncomfortable when someone provides such a um lovely introduction to me it’s um it’s very humbling and I’m very pleased to be able to present for you today so let me just start sharing my screen
okay great so to start with thank you so much to Retina Australia for Julia Hall for inviting me to speak with you today um I’m calling in from the land of the Wallumedegal people um and I’d like to pay my respects to Elders past and present um so as Sally had mentioned I’m a behavioral scientist or a psychosocial researcher with UNSW Sydney and over the past 10 years I’ve very much been committed to how can we better understand families experiences of rare diseases and develop appropriate interventions and support to ensure that they um you know live a live a um the best possible lives as possible so earlier this year I was brought on to lead a project in inherited retinal diseases and I feel very privileged to be a part of this role as I’m able to partner with individuals with lived experience health professionals and advocacy organizations like Retina Australia to be able to identify research priorities for inherited retinal diseases um so I wanted to take this opportunity to share a little bit about what this project involves why it’s so important and then how you can support us if it is of interest to you so as some context and this may be um all very basic knowledge um for you we know that inherited retinal diseases are the leading cause of blindness in children and adults of working age and it affects around one in 3,000 people and this includes diagnoses such as Retinitis Pigmentosa Usher’s, Stargardt’s disease and Leber’s and there’ve been some really rapid medical advancements over the past few years and this includes improved diagnostic Technologies and access to genetic testing and at the moment there are over 250 currently known genes that cause inherited retinal diseases with the exception of a specific where gene therapy has proved successful we know that IRDs are currently incurable but it’s really great to see that there’s so much fantastic research out there at the moment Research into gene therapy and stem cell therapy and it’s showing promise for a future world without inherited retinal diseases and you’ll hear a little bit more about this from Vivian after my presentation so why is my project come into existence I think primarily it’s because money unfortunately doesn’t grow on trees we know that we’ve only got a limited number of working days and days in a year to get this work done and yet our list of what needs to be done feels quite infinite so how do we actually decide on what gets shifted to that very top of our list what is the most important area for us to focus on what requires the urgency to get done now and typically we’ve been driven by whatever the government the funding bodies pharmaceutical companies and researchers like myself decide what is most important and
Urgent and how I like to describe this is is using a bike path analogy so imagine a square Park and we want to build a bike or walking path from one side side a to the other side of the park side B the opposite side but you don’t get any input from people that will be using that path what you ultimately get is two very different looking paths one is what the governments the funding bodies the pharmaceutical companies and researchers want and then the other is what the patients the carers the family members and health professionals who care for those individuals wants and so these are really different paths and it doesn’t it doesn’t match up often a lot so how can we scientifically capture what are the priorities for us as researchers what should we actually be focusing on to ensure that the path that we’re creating is the right path and the one that gets used so the method or the approach that I’m using is called a priority setting partnership and these projects identify and prioritize questions that have yet to been answered by research that key stakeholders considered the most important and the reason being is we want to drive research that is going to be the most meaningful to those impacted by we want to ensure that we’re walking along the path and creating the path that people want so when we Define our stakeholders for this priority setting partnership we have individuals who are living with an IRD and that’s including syndromic IRDs such as Ushers we’ve got guardians and family members such as parents Partners grandparents siblings as well as the health and supportive Care Professionals such as ophthalmologists GPs Educators clinical geneticists as examples and we want to capture the voice of all of these individuals to end up with these top 10 research priorities and it’s it’s a bit of a mouthful of what we’re including but kind of broken down we want to have a look at the priorities regarding anything to do with the prevention of inherited retinal diseases the diagnosis of diseases, disease progression and control, treatment as well as the management of IRDs um so it may be that the treatment of focus research may not be the priority for a lot of stakeholders and that managing living well with an inherited retinal disease is been more of an important focus or we could find that research into a better diagnosis pathway is considered appropriate than Research into disease progression so this is all what we’re trying to find out from this project and so how we actually get to those top 10 priorities is this um kind of roughly put a five-step process the first step is to create a Steering group of individuals to guide how this project is completed so rather than being directed by me someone who has no lived experience of an IRD I am guided by the people who understand these conditions and can advocate for the community um on their behalf so our steering group includes some fantastic individuals who have a child with an IRD like Holly Feller and Emily Shepard who are the co-founders of Usher Kids Australia um and parents um of a child who have who has ushers we also have individuals in our group who themselves are living with an IRD such as the wonderful Leighton Boyd as well as organizations who can meaningfully represent individuals impacted by IRDs such as Retina Australia and we’ve also got some researchers and health professionals um involved in this project I think I saw Lauren Ayton was online and we’re really grateful for their involvement to be able to drive through this project we’ve also connected with some key organizations and this is going to be really critical to allow us to not only promote this project and get participants or individuals to participate but it’s really to also disseminate the priorities to make sure that whatever we identify as the top 10 priorities is actually taken forward and some examples of the key organizations that we’re working with include Retina Australia is one of our key um Partners Blind Citizens Australia, Deaf Blind Australia um in the Royal Australian New Zealand College of ophthalmologists so I’ve got this fantastic group of people who are really committed to progressing the right research in inherited retinal diseases the second step is now to actually undertake a survey with individuals impacted by an IRD um as well as parents and guardians and health professionals about what questions they have about the prevention, diagnosis, progression and control treatment or potential treatments, epidemiology as well as the management of inherited rental diseases so it’s it’s it seems quite complicated and quite broad but really what we’re asking people is to submit any questions they have about IRDs that they have unanswered that they just haven’t had that answer and they’re still a bit unsure and we really take it from there so this survey is anonymous and expected to take about 5 to 10 minutes to complete it can take a little bit longer if you been unsure of what questions um you want to submit and there’s multiple formats for people to participate including over the line o online and the QR code on screen can take you to the online survey we can do it over the phone on paper on Braille or in Ausland Via a video call with the Deaf Blind Australia team so basically once we get all of these submissions people are telling us what they have unanswered what are a bit concerned or wondering about we collect all of these questions and organize them and work out which ones do actually still need research to answer and what we’re hoping to do once we get more responses we will have a really representative idea from the Australian population about what questions are unanswered so so far we’ve had 46 individuals respond about 100 submitted questions and we’ve got questions like will I go completely blind how can I slow down vision loss do lifestyle factors like diet affect the progression and what clinical trials are currently available so they can be quite broad questions but this is the type of questions we’re really wanting to find out um what people with an IRD are wondering they don’t feel like has been answered by research the third step of our process is to take the most common questions that are submitted in that survey the ones that are unanswered and then ask individuals to rank these of of these what they think is the most important so imagine we’ve got a list of 40 questions and now we want people to tell us what are the most important of those what top 10 do they think are most important and that survey is expected to go um live around March next year we’ll take the top ranked questions then to a workshop and these workshops will be a fantastic opportunity for individuals with an IRD, family members and health professionals to come together to discuss why they think certain priorities should be at the top and otherwise other ones perhaps not to be able to finalize whatever we put forward as our top 10 so I think this is a really unique process um to Priority setting Partnerships and it’s different to typical research is that it’s it’s not up to me as the as the lead researcher to make that final call it’s up to the individuals who represent the community who have the lived experience who will decide what is in the top 10 and then we’ll be opening up expressions of interest for those workshops around May May June next year then the final step is then to disseminate our top 10 so whatever our top 10 is is what we all push forward um and we need to be taking that to funding bodies to researchers to organizations to our partner organizations um to really advocate for this research to go ahead and that’s this is perhaps the most important part as we want to make sure that those priorities are actually taken forward and actually undertaken by the research community so we will be talking to funding bodies we’ll be trying to establish grants that address those priorities and advocate to researchers to undertake that those projects so to wrap up the vision loss priority setting partnership it seems lot like lots of steps but really it’s just the way for indviduals who represent inherited retinal diseases who are impacted by inherited retinal diseases to drive forward what actually gets done so we will advocate for the top 10 research priorities irrespective of what they are whether they align with my beliefs whether they align with a steering groups beliefs we will advocate for those to the government to funding bodies and to other researchers and our hope is that the researchers will undertake these projects that address what is most important to those impacted by inherited rental diseases which will lead to more me meaningful research so if you do have any questions about this project I do um ask for you to reach out to me um you can email me at vision loss PSP unsw.edu.au or you can call me on 0293480708 um and we can walk um talk through it the survey one where you can submit your questions is currently open and we would love to have as many of you participate as possible um and then potentially participate as well in the in the second follow-up ranking survey and the workshops so I’ll finish up there and obviously putting some questions um through to Sally if if um you have any questions now and we can answer them after the next presentation so thank you thank you so much Eden that’s fantastic no worries thank you so we’ll have some questions after we’ve heard Vivian speak so we’ll do all the questions at the end of the session if that’s okay with people and so now we’ll move on to our second speaker who is Vivienne Kaiser a PhD candidate from the Children’s Medical Research Institute in Sydney Vivan is a PhD student um and uh she’s working under the supervision of Dr Anai Gonzalez Cordero who’s one of our currently funded researchers she completed a Bachelor of Science Bachelor of Advanced Studies at the University of Sydney with first class honors in 2021 she currently works on Usher syndrome an inherited disease that causes deafness and blindness in children her work aims to develop AAV Gene therapies for this disease using stem cell organoids derived directly from patients with Usher syndrome this afternoon she will provide us with an introduction to stem cells and gene therapies touching also on her own project focusing on Usher syndrome so I’ll hand over to you Vivienne thank you so much thank you Sally um let me just share my
screen all right right so hello everybody um I’m Vivian as s mentioned um a PhD student so I’ve just started I’m in my first year in um Anai Gonzalez Cordero’s lab um at the Children’s Medical Research Institute so I’m just going to be just telling you about what we do in the lab pretty much um and so a stem cell story we want to try and treat blindness with stem cells and so just just a brief background about our lab we have 16 people um five PhD students all under the supervision of Anai and we are experts in stem cells um so Anai got her PhD from the University College London um where she was able to establish um a stem cell facility um when she came back to Australia and it was the first of its kind in in Australia so it’s really exciting research which I’ll tell you about so first of all what is a stem cell so if you’re unaware um stem cells are basically cells that have not been differentiated yet into an adult cell so they don’t know what they want to be when they grow up they could be a gardener or a barista or personal trainer or a nurse or they have no idea what they want to be and so once they um differentiate in what they want to be that’s where we get the adult cells from um so yeah Yeah so basically you’ve got your stem cells and they can differentiate into whatever cell type and so blood cells muscle cells cardiac cells any single cell type and it’s quite amazing because it comes from the same cell everything in your body comes from the same cell and so yeah our bodies are made up of 30 trillion cells which is a huge amount with over 200 different types of cells and they all come from the exact same cell and so but where do they come from well they do come from embryos so after um an egg is fertilized uh in the this this stage here which is called the blastocyst stage um your these stem cells start to form and become um and can go on to become whatever cell they want okay so we can take them from embryos and we can grow them in the lab um in and differentiate them into what diff whatever cell type we want but it is obviously not very ideal to be taking stem cells from embryos and so this amazing amazing man in 2012 called shinya yamanaka um invented a way to turn adult cells back into stem cells which bypasses any embryo work at all and so basically we get these adult cells and we put them back into stem cells where they can go on to differentiate into another another type of cell and so and this just requires four genes that can untrain our cells from being specialized and so this means that we can take cells from a patient um so we can use blood cells or skin cells and turn those cells into stem cells um and so the advantages to this again is we don’t need to use embryos for this we can take cells from any any patient um such as patients with diseases children and patients with different genetic makeups and so what our lab is focusing on is using these stem cells derived from adult cells to cure genetic blindness and so the most common causes of blindness in children as I’m sure you’re very aware of this um inherited retinal diseases in children and then after 40 years age related macular degeneration um and so how can we um so basically um we will give these cells instructions these stem cells to become an eye cell okay because as I said before these stem cells can become whatever you want so when we give them instructions they can become an eye cell okay and we can culture these eye cells and this is a really great advantage because it is like the eye is a very very complicated organ and it is you can’t just easily take eye cells from a patient or anything so but if we can differentiate them from stem cells we can grow them in dishes and flasks and use them to study but again eyes are very very complex and just studying one different cell type in the eye won’t actually um give you an idea of the entire disease in in as a whole so what we want to do is we can group these stem cells together and add different things into them and they together they can form mini organs or mini retinas so they instruct each other and they for start to begin to form groups of eye cells which then go on to instruct each other more and we’re left with a 3D mini human eye structure okay um and so we have we’ve got in our lab we make mini human retinas and so yeah this this is the back of the eye which I’m sure you’re aware that contains all the um light sensing um receptors in there and so this is the key to Vision so we can form the cells necessary for vision um in the mini human eyes and these are functional um retinas as well with neural activity and all of that um and so yeah we can use these these um use these to study disease such as age related macular degeneration or inherited retinal disease and so one of the things our lab is doing me specifically as well is gene therapy and basically what I do for this is we grow up the organs the mini retinas from patients with inherited retinal diseases and so they have this mutation we then can deliver a healthy Gene to these cells or to these organs um with a viral mechanism okay and so for this virus it’s a safe virus because we have ripped out all the guts of the virus that is bad um and replaced it with the healthy Gene and so this can go into the cells and the retinal cells will be restored in the organoids and so I’m focusing I’m doing this on Usher syndrome uh where I’m creating some different gene therapies for that so I’ve created three gene therapies for that and I’m in the process of testing them out on retinas or on three different cell lines um so three different patients um and their retinas but I’m also using these stem cells um to grow inner ear organoids as well so I’m doing that in parallel so another thing that our lab does is cell therapy and basically with this we get um a healthy patient this time we get the blood cells and the stem cells from that healthy patient we grow up these organs and then we isolate these photo receptor cells from those mini organs and Transplant them back into a patient with AMD or something like that and then that restores Vision so we have done that um already in mice we’ve been able to get stem cells and remove the eye cells from it and inject them into um Blind Mice and vision has been restored in these mice so it is a really promising approach so yeah so we’re one step closer to curing blindness with these exciting exciting mini organs so now we can grow mini organs outside of the body and use them to create Gene and cell therapies and so and this is much better than um using animal models or anything like that because it is precise to that person and to that patient and it is it recapitulates the human State very very well um and so in the near future hopefully we are going to use the stem cell technology to restore Vision um so yeah thank you for listening um and I would love to hear some questions or any comments and just just a a thank you to everything that you guys do as well for for our research I we really appreciate it yes thank you so much Vivian that’s such an exciting uh presentation um we now have time for some questions um so if you’d like to ask a question you can type it into the Q&A section at the bottom of your screen or if you raise your hand um uh we can we can uh say your name and turn your microphone on so that you can ask your question directly and then we’ll turn your microphone off afterwards um so our first question is from Julia Hall
you should be good now Julia hi sorry I can’t turn the video on but um that’s it I’ve got I guess we’ve got B in here first vivian thank you for a very very interesting presentation I wanted to ask if somebody wanted to participate in the trial and have their adult cells converted to stem cells to participate in clinical research how would they go about seeking out to participate in if they work yeah so a great question um you would probably contact um my supervisor so Anai Gonzales Cordero um you can email her directly um but it’s not like a a straight process there will have to be there’s a lot of ethical or ethic regulations and all of that that um have to be undergone before before your blood can be um given but it is definitely possible we’re always wanting more more cell lines because it just it it allows our study to be a lot more um holistic and we can get an idea of different patients and and the differences between them as well so yeah we we do welcome welcome people at all so just yeah email Anai Gonzalez Cordero thank you Julia um I had a question actually for Vivian as well um you mentioned that you can treat the cells when you have them as stem cells um you do things to them to make them develop in a particular way it’s probably very complicated but how do you make them become eye cells as opposed to muscle cell what is the what is the process that you go through to specify that yeah no that’s a great question and it’s still one that we’re still try like because it’s very new this this this field so it’s still we’re still having a bit of difficulties with that but basically for the we mimic a baby growing in the womb pretty much um and there are certain genes that are expressed um to allow these stem cells to differentiate different ways um and so we just like give in a in a lab setting give them those exact same factors and its exact same concentrations like it’s a very very fine balance to get them to go to retinas um but basically for this one what we do is so we grow these cells um and then um they grow like on a like a monolayer of cells and basically after a month when we add um all the different media that contains the factors specific for retinol differentiation they just spontaneously Just Produce and then we have to sit there and pick them out of the plate and transfer them to different um um wells but yeah yeah it’s really exciting that we can do it and yeah I’ve recently um been able to create the inner ear now which has hasn’t been done yet so that’s very exciting um wow so there’s a very specific sort of um recipe I suppose of what you need to put in and how do you find out what that is like is that is that just because you’ve studied the embryonic cells so much that you know what the what those factors are yeah exactly yeah and it and I mean this research has been going on for a while so I mean I’m new here the protocols were already established lucky for me um but yeah you start off by looking at how um the embryo was formed like in the womb um and that’s already been very very well characterized like from years and years ago so we know that the factors that are used um but we don’t really know the the balance of the different factors and like the concentration so it’s just a lot of trial and error and like I say we those retinas spontaneously pop up we also get brains popping up and and we don’t want the brains but yeah so it can be you just have to choose the the ones that are doing what you want them to do exactly exactly wild isn’t it fantastic okay we have a question now from Leighton Boyd no thanks Eden and Vivienne and I just can’t I know did a great presentation and it’s as a member of that group is it’s just amazing work that I think all of us and I think as patients we need to pass that survey on to as many people as we can we need to know all those questions there’s no question that’s a silly question it’s all important information so I just want to really emphasize to people to pass that survey on give us input that’s important stuff and Vivian your work I’ve been fortunate enough to go to to the lab in Sydney and it’s it’s mindblowing you know the the the work that Anai and the team are doing can you just tell us a little bit we keep saying we’re growing we’re doing this and doing that what do we growing them in are we growing them in the garden are we growing them in the sorter how do we put them here store them into a special how does that work thank you yeah yeah so we we don’t grow them in the garden we grow them in a in our tissue culture lab where we have to um like properly scrub up we have a mask a hair net gloves everything it is going to be extremely extremely sterile we can’t have any bacteria contaminating these organs because they they take 30 weeks to grow um or to mature and so if if like bacterial contamination comes in like 28 weeks it’s like no um but anyway so what we do we keep them in incubators so 37 degrees um which is the same as our bodies because that’s that’s where how they like to grow so they we have I think we have like 12 incubators and like six fumehoods where we do our um culture and yeah it’s it’s great a lot of work these these cells take to grow but yeah very exciting um thanks very much Leighton um I have a question for Eden it’s I mean obviously you’ve done you’ve had some responses from people so far and obviously still are have you been surprised by any have you had any that you really weren’t expecting it it’s it’s been really interesting to see the questions come through I think for me as psychosocial researcher I’m always kind of front of mind is how can we improve the well-being of families and and you know the mental health and and adjustment to life with vision impairment that seems to be a question that’s coming through quite a lot how can we actually manage mental health especially when we know that our vision may be kind our visual loss can be progressing that seems to be a question that’s coming through a lot that I wasn’t necessarily expecting I think the the way that the survey is set up is that you can submit five questions and so people it’s quite broad because inherited retinal diseases even you can hear from what Vivian’s talked about we kind of don’t there’s not enough information out there yet to really specify I want to know this specific stem cell therapy and how it works for this specific disease people are giving really broad questions and that’s really allowing them to provide five questions across five very broad topics so we’ve got questions about cure which we knew was going to be a question that’s come up how can we you know Advance stem cell and gene therapy for a cure but then because that’s one question in itself and that’s encompassing so many for us as researchers questions it’s allowed people to explore additional things like mental health which probably always sit off to the side as a secondary um research aim to to cure um we’ve had some questions about how can we actually better support health professionals to support us as patients to support the patients say how can we better train health professionals to understand experiences of individual individuals with IRDs and how to better support them to navigate and to adjust well to life so they’re kind of the three main questions that seem to be coming out that are quite a clear theme but as Leighton said no question is a silly question I think we’re really wanting to get as many questions as possible so that it can be representative so that whatever we get from the top 10 is truly representative of what the community actually wants yeah I was just going to say it’s it’s a wonderful opportunity for people to uh to speak thank you so much Julia um Hall has another
question hi just a quick one for Eden uh Eden we have a lot of members who don’t have access to online or digital facilities to answer the survey I think you mentioned it in your presentation that there’s a phone number perhaps people could call we get a lot of calls to the office where that’s the easiest option is that your phone number on your presentation yes so that’s my phone my personal phone number um and if you call it on either a Monday or a Tuesday I will be able to answer it at that point in time or if you call outside of those hours I will always give I will give a call back and you can complete the survey we could just have a chat and just you can just tell me you know what’s kind of coming into your mind and then I can kind of take your feedback and put it into the survey kind of structure if that would be valuable we can also do it via video call if that’s easier as well as having the deaf blind Australia consultant Provide support through Auslan and I can also provide it mail out a hard copy paper version if that would be easier with screen readers that’s terrific thanks so much thanks okay so we’ve got now got a question from Mark Boyd vivian talked a lot about ethics and I it’s just a quick one on those ethical things um when you’re dealing with creating life in different parts what controls are there of how much you can create in virtually having a life and death sort of situation thank
you yeah that it is a very good question um yeah well I mean science is not at the stage yet where we can actually create life with these these organs um they can’t like these are so for the retinas it’s just the retinas it’s not the entire eye or anything um but no it is it is a really good good ethical point that you made there and I I mean I can’t really answer it I have to say um yeah it is tricky but there is a lot like a lot of paperwork to get ethics approval to get someone’s blood um and working with human samples um but yeah no it is a it is it is a good point absolutely yeah thank you is there anybody else that has a
question Sally if it’s all right I might just comment um in addition to what Vivian just um mentioned for Mark that question that you just asked Mark about how can we ensure that you know any samples provided and it’s ethically used and not created life that’s the type of questions as well that our my project we’re wanting to hear from people because we if we know that this is something that’s really important important for individuals we will prioritize that and say we need to have protocols and information resources that patients actually do understand about how these processes work and and you know how their samples will be used and what it means for the future and you know educating about what the ethics process actually requires so Vivian and Anai would have had to go through a really strict process where the project gets kind of written up in a really structured um kind of document then gets sent to external peer reviewers to look over it and say actually I’m concerned about the well-being of this these patients you need to provide more information here or it needs to be explained more simply this is the process that goes through research um at the back back end that’s often not talked about um to Consumers and so if that’s an area of research that we need to be ensuring that we’re dedicating more time to then that’s the type of questions we’re hoping to get within our priority setting partnership to actually dedicate funding to to um develop information resources to explain those things that’s great thank you so much um I’d also like now to ask um Rosalie O’Neal has a question I I have the advantage of being able to um be in Perth and Victoria and so I my brother passed away four weeks ago with Stargardt’s disease and um it took a lot of uh paperwork and a lot of ethical things so that his eyes have been donated to associate professor Fred Chen at the Lions Eye Institute is is that an advantage to have um tissue that does have the mutation yes of course yeah it would definitely be an advantage I’m trying to think of like if if we can study exactly um what he had and like the specific genetic mutations and all of that and then understand more about this disease then that it is definitely beneficial for future people with Stargardt’s um we are doing lot of work on Stargardt’s as well um but yeah it is definitely helpful okay because they said that they’ll keep the tissue for 10 years and I thought that’s pretty exciting you know to know that um it it’s going to be researched and looked at and you know maybe we can find a cure from them yeah for sure for sure so thank you for that that yeah it’s it’s very exciting to know that but yeah thank you thanks Rosalie. Leighton Boyd is has another question thank you um I just want to tell the the membership ethics from Retina Australia’s point of view we’re very very very conscious we we like to support research projects we’d like to be the the One-Stop shop where since Julia started with with as Julia said earlier we’ve reviewed our research protocol later on today and tomorrow we’ll be looking at some new funding um applications but at the prime um from now board point of view we’re always wanting projects and ethics approval and I think it’s important that that’s the case I think we do have a grants assessment committee we have a lot of scientists and researchers on board and I think that um people and members should be rest assured that um any project that we support as an organization is well equipped to supported and it’s a very very tedious and as Vivan said it’s a very painstaking process to get ethics approval so I think that’s great I think for us as a as a patient point of view we we would be wanting to be um you know assured that the samples and and the work that we’re involved in as patients is is all above board and and ticks all the boxes so that’s just a comment yeah I think that’s a really good point to make as well Leighton that any research you participate in you have the right to re revoke your consent from participation as well so say if you consent um blood to Vivian’s project and she uses that if in a year’s time you go actually I don’t feel comfortable with that I don’t want to include my sample anymore if you contact those researchers you can say remove all of the data that you’ve obtain from my from my sample I don’t want to participate any further and that’s totally okay there’s no um you know there’s not a black mark against your name for researchers or for hospitals and so I think that’s something that we really need to ensure that families do understand if you do participate that’s completely up to you and you can always change your mind at any
point thank you very much for that Eden if are there any more questions I can’t see any more but you’ve got a final opportunity to to ask a question before we thank our guests no more all right well um what I’d like to do is just thank both our speakers today for such um interesting and exciting presentations it’s so it’s always so fantastic to hear about these new developments and um we can have so much optimism about the future so I hope everybody has found the S the session informative if you have any questions if you think of any further questions don’t hesitate to contact us and we’ll make sure we’ll make contact with Eden and Vivien um so that they can they can answer any queries that you might have so that’s the end of the webinar thank you both very much for your time and expertise this afternoon and uh thank everybody else for joining us and I wish you well for the rest of your day thank you very much
Summary of the Webinar
A summary of this webinar is available to view here.
Retina Australia is delighted to welcome Dr Eden Robertson to present on the Vision Loss Priority Setting Partnership, and Vivienne Kaiser to provide an introduction to Stem Cell and Gene Therapies.
Vision Loss Priority Setting Partnership
Presenter: Dr Eden Robertson, University of NSW, Sydney
It is important that research about Inherited Retinal Diseases is guided by what matters most to those who will be impacted by it. That is why researchers from UNSW Sydney and the Children’s Medical Research Institute are undertaking a James Lind Alliance Priority Setting Partnership to identify the top 10 research priorities for Inherited Retinal Diseases. This project is guided by a steering committee that includes individuals with lived experience of an Inherited Retinal Disease, patient advocacy groups like Retina Australia, health professionals and researchers.
In this webinar, Dr Eden Robertson will present on their Vision Loss Priority Setting Partnership, and the process they will undertake to arrive at the top 10 research priorities for Inherited Retinal Diseases.
They will present the importance of engaging with individuals with lived experience, challenges in doing so, and the ultimate benefit of Priority Setting Partnership in driving drive the future research. It is important that research about Inherited Retinal Diseases is guided by what matters most to those who will be impacted by it.