Uploaded July 2021 | Updated September 2026, 1 week ago

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Recently Intellia Therapeutics in collaboration with Regeneron announced some promising results for their phase I trial using their lead product NTLA-2001, for an Investigational CRISPR Therapy for Transthyretin (ATTR) Amyloidosis. In this video I discuss the science behind in vivo CRISPR gene editing and what this means more generally for the CRISPR field or as Intellia phrase it the #NewEraOfMedicine.

TIMESTAMPS
Intro - 00:00
Intellia & CRISPR-Cas9 genome editing - 00:35
NTLA-2001 - 02:30
#NewEraOfMedicine? - 08:00

REFERENCES
CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis DOI: 10.1056/NEJMoa2107454 nejm.org/doi/full/10.1056/NEJMoa2107454

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