AHA 22: Results of the CRISPR-Cas9 First-in-Human Trial
Автор: Radcliffe Cardiology
Загружено: 2022-11-05
Просмотров: 6999
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AHA 2022 — Dr Julian Gillmore (University College London, London, UK) joins us to share the key findings from the CRISPR trial (NCT04601051).
This Phase 1 study was conducted to assess the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of NTLA-2001 in participants with hereditary transthyretin amyloidosis with cardiomyopathy (ATTRv-CM) or wild type cardiomyopathy (ATTRwt-CM).
Questions
1. What are the unmet needs of patients with ATTR amyloidosis?
2. What is NTLA-2001 and how does it work?
3. What is the trial design, outcome measures and eligibility criteria?
4. What are the key findings?
5. How should this trial and the findings impact medical research?
6. What are the next steps?
Recorded remotely from London, AHA 2022.
Interviewer: Mirjam Boros
Video Editors: Thomas Green and Dan Brent
Radcliffe brings medical knowledge, insight and innovation to life for CV clinicians around the world, using our communications & creative expertise, our platforms and connections across the community to help transform theory into practice faster.
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