Retinitis Pigmentosa & Luxturna Gene Therapy Webinar
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- Опубликовано: 28 авг 2024
- Masters students in the Medical Genomics Program from the University of Toronto present this webinar in hopes of providing an in-depth explanation on the genetic underpinnings of retinal diseases and highlight exciting breakthroughs in gene therapy, specifically the recently approved Luxturna, that could have extraordinary outcomes for patients battling these diseases.
Test Yourself!
Link to pre-webinar quiz: www.surveymonk...
Link to post-webinar quiz: www.surveymonk...
More resources to learn about Luxturna treatment and access:
Drug:
luxturna.com/
Insurance:
mysparkgenerat...
British Columbia- coverage under consideration
www2.gov.bc.ca...
Book: www.amazon.com...
Interviews/patient success stories:
Jake Hogan: • Jack Sees a Different ...
Cori Haas:
dnascience.plo...
Organizations:
Foundation fighting blindness: www.fightingbl...
Curing retinal blindness foundation: crb1.org/
Canadian Agency for Drugs and Technologies in Health (CADTH) Patient Input on Luxturna: cadth.ca/sites...
Video generated using VideoScribe
Music by: www.bensound.com
CC-BY-SA
License Creative Commons Attribution license (reuse allowed)
Content References:
NIH National Human Genome Research Institute. About retinitis pigmentosa. (2013). at www.genome.gov....
NIH US National Library of Medicine. Genetics home reference: retinitis pigmentosa. (2020). at medlineplus.go....
NIH National Centre for Advancing Translational Sciences. Retinitis pigmentosa. (2016). at rarediseases.i....
Online Mendelian Inheritance in Man, OMIM. Johns Hopkin University, Baltimore, MD. MIM Number: 268000: 2020: . World Wide Web URL: omim.org/entry....
The Retina Foundation of Canada. Retinitis pigmentosa. (n.d.) at retinacanada.c....
NIH National Eye Institute. Retinitis Pigmentosa. (2019). at www.nei.nih.go....
Online Mendelian Inheritance in Man, OMIM. Johns Hopkin University, Baltimore, MD. MIM Number: 189969: 2020: . World Wide Web URL: omim.org/entry...
Fighting Blindness Canada. Luxturna treatment: restoring vision becomes a reality. (2020). at www.fightingbl....
Russell, S., Bennett, J., Wellman, J. A. & Chung, D. C. Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial. Lancet 390, 849-860 (2017).
Maguire, A. M. et al. Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation-Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials. Ophthalmology 126, 1273-1285 (2019).
Acland, G. M. et al. Gene therapy restores vision in a canine model of childhood blindness. Nat. Genet.28, 92-95 (2001).
Maguire, A. M. et al. Age-dependent effects of RPE65 gene therapy for Leber’s congenital amaurosis: a phase 1 dose-escalation trial. Lancet 374, 1597-1605 (2009).
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@sssproductions I love videos like this, how can I make one like this with the animations? Is there a certain software that does it?
The Important Bits:
1.) When the video was made it cost $425,000 per eye but they hoped to reduce it
2.) It improves light sensitivity and the size of your vision field but doesn't make your vision clearer, so you will be able to navigate in the dark but not clearly see what you are navigating around.
3.) It's safe, tested in 21 patients with no problems. 1 Injection per eye.
4.) It takes 30 days to reach full effect and tests show that the effects are still there after 4 years.
So basically, it's not the miracle cure we all want but it is a step in the right direction. It has been approved by the FDA and Canada's Health.
Is there anybody who has enough grace's to share for my gene therapy. Thank you in advance and God bless you and your family.
Why so expensive???only few can afford this amount half million for both eyes😢😢😢😢
The cost will come down significantly, sadly with things like this the people who funded the drugs aim to get back their money as quickly as possible incase another drug comes out leaving them out of pocket. It means the early adopters tend to be wealthy.
Is it available for a common man outside USA & CANADA?
I don't know how genes work but is there any hope of qualifying for this therapy when your RP gene is RLBP1? Do you know of any other interventions?
The visual acuity part made me kind of sad, I was hoping I would get back sharpness :(
How’s the procedure to have the disease cured?
Buenas tardes vivo España tengo una hija tiene Retinose pimentaria Sidroma huster por favor ay cura para isto gracias
does this therapy work with RP1 mutation
Is possible eye optic nerve damaged regeneration.. please reply
Is it or will available in India?
I am a filipino interested to avail this gene therapy but unfortunately i don't have a money to pay it.
Kabayan may RP din ako kamusta naman pamumuhay mo at ilang taon ka na? Pano ka nakakapag trabaho ng normal padin ba?
@sss productions @luxturna its a question for both. will this gene therapy still work after covid infection or vaccination? i m asking because after infection body develops some immunity against viruses and covid vaccines are to stop virus infection and hence viruses are not that effective. gene therapy is also based on viruses. please provide some knowledge.
The virus contains no viral parts; it's essentially an empty shell that carries the RPE65 gene into retina cells. So this treatment will not be affected in any way by any vaccines.
Can it cure optic nerve damage from glaucoma.??
If gene therapy is a thing, what about addressing Sticklers disease which also affects the eyes and can cause retina detachment?
It's not that easy my guy
It's actually "Stickler syndrome" if you want to be a stickler for correct nomenclature 😅
I'm from Pakistan and how can I get treatment for Retinitis Pigmentosa
my daughter has been diagnosed with RP due to RP1 mutation is there anyway to have gene therapy
This gene therapy works only for patients with RPE65 mutation since this delivers its functional copy into the retinal cells of the patients and LUXTRUNA is the only FDA-approved drug for Retinal dystrophy, however, you can search for any clinical trials for the patients with mutation as your child.
Please treatment rp
Please help me retinitis pigmentosa ag 20 years
Don't worry brother all will be ok..
There are treatments to stop degeneration don't worry
In future there will be defenantly cure to this disease
Don't worry !
@@nagender.anagender.a9904 me sosaid kar longa bhai please help me
@@abhisonu9711 even I don't know much about this but I can say there will be cure to this for next 2-3 years
@@nagender.anagender.a9904 sir we are suffering from this disease that many people have gone into depression thinking of our illness sir ham apse samprak krenge please contect number