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Lessons from the first-in-human in vivo CRISPR/Cas9 editing of the TTR gene by NTLA-2001 trial in patients with transthyretin amyloidosis with cardiomyopathy
Introduction: Transthyretin amyloidosis (ATTR amyloidosis) is a progressive fatal disease characterized by accumulation of amyloid fibrils composed of misfolded transthyretin (TTR) protein in tissues, resulting in cardiomyopathy and heart failure. Approximately 50,000 people have hereditary ATTR amy...
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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Magdi Yacoub Heart Foundation
2023
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10624232/ https://www.ncbi.nlm.nih.gov/pubmed/37928601 http://dx.doi.org/10.21542/gcsp.2023.4 |