Trusted Resources: Education
Scientific literature and patient education texts
Lay Summary of “TTR Gene Silencing Therapy in Post Liver Transplant Hereditary ATTR Amyloidosis Patients”
source: Amyloid: The International Journal of Experimental and Clinical Investigation
year: 2020
authors: Orly Moshe-Lilie, Diana Dimitrova, Stephen B. Heitner, Thomas H. Brannagan III, Sasha Zivkovic, Mazen Hanna, Ahmad Masri, Michael Polydefkis, John L. Berk, Morie A. Gertz, Chafic Karam
summary/abstract:The rationale is as follows:
1) some people with hATTR who undergo liver transplant continue to have disease progression, despite the fact that they have a “new” liver which does not have a mutated TTR gene;
2) One of the explanations behind the progression of disease after liver transplant is that wild type TTR (non-mutated TTR) continues to misfold and attach to the amyloid that was deposited prior to the transplant;
3) Decreasing the production of TTR protein should, in theory, slow down the deposition of misfolded TTR protein and the progression of amyloid.
Based on these observations, patients with hATTR who underwent liver transplant but continue to have disease progression were started on “TTR knock down” therapy.
DOI: 10.1080/13506129.2020.1784134
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