Retinitis Pigmentosa & Luxturna Gene Therapy Webinar

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: https://www.surveymonkey.com/r/GH8SRGP
Link to post-webinar quiz: https://www.surveymonkey.com/r/GPRHP6X


More resources to learn about Luxturna treatment and access:

Drug:
https://luxturna.com/

Insurance:
https://mysparkgeneration.com/pdf/Pat...

British Columbia- coverage under consideration
https://www2.gov.bc.ca/assets/gov/hea...

Book: https://www.amazon.com/Forever-Fix-Ge...

Interviews/patient success stories:
Jake Hogan:    • Jack Sees a Different Life after LUXT...  
Cori Haas:
https://dnascience.plos.org/2017/07/2...

Organizations:
Foundation fighting blindness: https://www.fightingblindness.ca/
Curing retinal blindness foundation: http://crb1.org/
Canadian Agency for Drugs and Technologies in Health (CADTH) Patient Input on Luxturna: https://cadth.ca/sites/default/files/...


Video generated using VideoScribe

Music by: https://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 https://www.genome.gov/Genetic-Disord....
NIH US National Library of Medicine. Genetics home reference: retinitis pigmentosa. (2020). at https://medlineplus.gov/genetics/cond....
NIH National Centre for Advancing Translational Sciences. Retinitis pigmentosa. (2016). at https://rarediseases.info.nih.gov/dis....
Online Mendelian Inheritance in Man, OMIM. Johns Hopkin University, Baltimore, MD. MIM Number: 268000: 2020: . World Wide Web URL: https://omim.org/entry/268000.
The Retina Foundation of Canada. Retinitis pigmentosa. (n.d.) at https://retinacanada.com/en/learn-mor....
NIH National Eye Institute. Retinitis Pigmentosa. (2019). at https://www.nei.nih.gov/learn-about-e....
Online Mendelian Inheritance in Man, OMIM. Johns Hopkin University, Baltimore, MD. MIM Number: 189969: 2020: . World Wide Web URL: https://omim.org/entry/180069#0003
Fighting Blindness Canada. Luxturna treatment: restoring vision becomes a reality. (2020). at https://www.fightingblindness.ca/news....
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).