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First CRISPR Treatment Poised for Historic Approval
PLUS: Complexity Challenges Progress in Autism
Kris Tatiossian, PhD
April 09, 2023
Hi friends 👋🏼,
Big news this week as the first-ever CRISPR therapy seeks approval. Plus, more stories for Autism Awareness Month:
First CRISPR Treatment Poised for Historic Approval
Complexity Challenges Progress in Autism READER REQUEST
Current & Emerging Treatments for Tuberous Sclerosis Complex READER REQUEST
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🧬✂️ First CRISPR Treatment Poised for Historic Approval
1/ Vertex and CRISPR Therapeutics have submitted the first-ever CRISPR-based cell therapy for approval 🥳
@crisprclassroom Exciting news for patients with #sicklecell disease: the first #CRISPR #genetherapy is seeking approval for clinical use. This groundbreak... See more
2/ The therapy, called Exa-cel (previously CTX001), stopped painful crises in sickle cell and led to 90% of beta-thalassemia patients no longer needing regular blood transfusions!
3/ Patients will continue to undergo a 15-year safety follow-up, but we will likely see the FDA’s final decision in the next 8-12 months.
♾️ Complexity Challenges Progress in Autism
Image courtesy of Dr. Alysson Muotri, Ph.D. presented at the Autism Tree Global Neuroscience Conference, 2022
April is Autism Awareness Month, so let’s dive in to understand this complex and nuanced condition.👇🏼
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