SciShow | The First CRISPR Gene Therapy Is Here @SciShow | Uploaded 6 months ago | Updated 1 day ago
CRISPR is a powerful gene editing tool, but its uses have been purely scientific until now. In 2023, US and UK drug regulators including the FDA approved Casgevy, a CRISPR/Cas9-based therapy for sickle cell disease and beta-thalassemia. The FDA also approved a similar treatment, Lyfgenia. Here's how they work.
Hosted by: Reid Reimers (he/him)
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Huge thanks go to the following Patreon supporters for helping us keep SciShow free for everyone forever: Adam Brainard, Alex Hackman, Ash, Benjamin Carleski, Bryan Cloer, charles george, Chris Mackey, Chris Peters, Christoph Schwanke, Christopher R Boucher, DrakoEsper, Eric Jensen, Friso, Garrett Galloway, Harrison Mills, J. Copen, Jaap Westera, Jason A Saslow, Jeffrey Mckishen, Jeremy Mattern, Kenny Wilson, Kevin Bealer, Kevin Knupp, Lyndsay Brown, Matt Curls, Michelle Dove, Piya Shedden, Rizwan Kassim, Sam Lutfi
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Sources:
https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease
https://www.gov.uk/government/news/mhra-authorises-world-first-gene-therapy-that-aims-to-cure-sickle-cell-disease-and-transfusion-dependent-thalassemia
https://www.ema.europa.eu/en/medicines/human/EPAR/casgevy
https://www.statnews.com/2023/12/15/europe-crispr-casgevy-approval-sickle-cell-thalassemia/
https://www.nhlbi.nih.gov/health/sickle-cell-disease
https://www.nhlbi.nih.gov/health/sickle-cell-disease/sickle-cell-trait
https://www.nhlbi.nih.gov/health/sickle-cell-disease/symptoms
https://www.nhlbi.nih.gov/health/sickle-cell-disease/health-effects
https://www.nhlbi.nih.gov/health/sickle-cell-disease/treatment
https://www.nhlbi.nih.gov/health/sickle-cell-disease/living-with
https://www.hopkinsmedicine.org/health/conditions-and-diseases/sickle-cell-disease#:~:text=The%20liver%2C%20heart%2C%20kidneys%2C,Leg%20ulcers
https://www.ncbi.nlm.nih.gov/books/NBK526064/#:~:text=History%20and%20Physical,the%20age%20of%206%20years.
https://www.nejm.org/doi/10.1056/NEJMoa2031054
https://www.nejm.org/doi/full/10.1056/NEJMoa2117175
https://www.nejm.org/doi/full/10.1056/NEJMoa2109167
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9069474/
https://www.npr.org/sections/health-shots/2019/12/25/784395525/a-young-mississippi-womans-journey-through-a-pioneering-gene-editing-experiment
https://www.npr.org/sections/health-shots/2020/06/23/877543610/a-year-in-1st-patient-to-get-gene-editing-for-sickle-cell-disease-is-thriving
https://www.npr.org/sections/health-shots/2023/12/25/1219342935/sickle-cell-patients-journey-leads-to-landmark-approval-of-gene-editing-treatmen
https://www.npr.org/sections/health-shots/2023/03/16/1163104822/crispr-gene-editing-sickle-cell-success-cost-ethics
https://www.yalemedicine.org/news/gene-therapies-sickle-cell-disease
https://www.casgevy.com/sickle-cell-disease/treatment-journey
https://www.broadinstitute.org/what-broad/areas-focus/project-spotlight/crispr-timeline
https://www.broadinstitute.org/what-broad/areas-focus/project-spotlight/questions-and-answers-about-crispr
https://www.ncbi.nlm.nih.gov/books/NBK500011/
https://bitesizebio.com/41748/a-beginners-guide-to-lentiviral-transduction/
https://www.casgevy.com/sickle-cell-disease/find-an-ATC
https://www.mybluebirdsupport.com/qualified-treatment-center-locator
Images:
https://www.gettyimages.com/
CRISPR is a powerful gene editing tool, but its uses have been purely scientific until now. In 2023, US and UK drug regulators including the FDA approved Casgevy, a CRISPR/Cas9-based therapy for sickle cell disease and beta-thalassemia. The FDA also approved a similar treatment, Lyfgenia. Here's how they work.
Hosted by: Reid Reimers (he/him)
----------
Support SciShow by becoming a patron on Patreon: https://www.patreon.com/scishow
----------
Huge thanks go to the following Patreon supporters for helping us keep SciShow free for everyone forever: Adam Brainard, Alex Hackman, Ash, Benjamin Carleski, Bryan Cloer, charles george, Chris Mackey, Chris Peters, Christoph Schwanke, Christopher R Boucher, DrakoEsper, Eric Jensen, Friso, Garrett Galloway, Harrison Mills, J. Copen, Jaap Westera, Jason A Saslow, Jeffrey Mckishen, Jeremy Mattern, Kenny Wilson, Kevin Bealer, Kevin Knupp, Lyndsay Brown, Matt Curls, Michelle Dove, Piya Shedden, Rizwan Kassim, Sam Lutfi
----------
Looking for SciShow elsewhere on the internet?
SciShow Tangents Podcast: https://scishow-tangents.simplecast.com/
TikTok: https://www.tiktok.com/@scishow
Twitter: http://www.twitter.com/scishow
Instagram: http://instagram.com/thescishow
Facebook: http://www.facebook.com/scishow
#SciShow #science #education #learning #complexly
----------
Sources:
https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease
https://www.gov.uk/government/news/mhra-authorises-world-first-gene-therapy-that-aims-to-cure-sickle-cell-disease-and-transfusion-dependent-thalassemia
https://www.ema.europa.eu/en/medicines/human/EPAR/casgevy
https://www.statnews.com/2023/12/15/europe-crispr-casgevy-approval-sickle-cell-thalassemia/
https://www.nhlbi.nih.gov/health/sickle-cell-disease
https://www.nhlbi.nih.gov/health/sickle-cell-disease/sickle-cell-trait
https://www.nhlbi.nih.gov/health/sickle-cell-disease/symptoms
https://www.nhlbi.nih.gov/health/sickle-cell-disease/health-effects
https://www.nhlbi.nih.gov/health/sickle-cell-disease/treatment
https://www.nhlbi.nih.gov/health/sickle-cell-disease/living-with
https://www.hopkinsmedicine.org/health/conditions-and-diseases/sickle-cell-disease#:~:text=The%20liver%2C%20heart%2C%20kidneys%2C,Leg%20ulcers
https://www.ncbi.nlm.nih.gov/books/NBK526064/#:~:text=History%20and%20Physical,the%20age%20of%206%20years.
https://www.nejm.org/doi/10.1056/NEJMoa2031054
https://www.nejm.org/doi/full/10.1056/NEJMoa2117175
https://www.nejm.org/doi/full/10.1056/NEJMoa2109167
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9069474/
https://www.npr.org/sections/health-shots/2019/12/25/784395525/a-young-mississippi-womans-journey-through-a-pioneering-gene-editing-experiment
https://www.npr.org/sections/health-shots/2020/06/23/877543610/a-year-in-1st-patient-to-get-gene-editing-for-sickle-cell-disease-is-thriving
https://www.npr.org/sections/health-shots/2023/12/25/1219342935/sickle-cell-patients-journey-leads-to-landmark-approval-of-gene-editing-treatmen
https://www.npr.org/sections/health-shots/2023/03/16/1163104822/crispr-gene-editing-sickle-cell-success-cost-ethics
https://www.yalemedicine.org/news/gene-therapies-sickle-cell-disease
https://www.casgevy.com/sickle-cell-disease/treatment-journey
https://www.broadinstitute.org/what-broad/areas-focus/project-spotlight/crispr-timeline
https://www.broadinstitute.org/what-broad/areas-focus/project-spotlight/questions-and-answers-about-crispr
https://www.ncbi.nlm.nih.gov/books/NBK500011/
https://bitesizebio.com/41748/a-beginners-guide-to-lentiviral-transduction/
https://www.casgevy.com/sickle-cell-disease/find-an-ATC
https://www.mybluebirdsupport.com/qualified-treatment-center-locator
Images:
https://www.gettyimages.com/
