Hereditary Transthyretin-Related Amyloidosis: Genetic Heterogeneity and Early Personalized Gene Therapy

Point mutations of the transthyretin (TTR) gene are related with hereditary amyloidosis (hATTR). The number of people affected by this rare disease is only partially estimated. The real impact of somatic mosaicism and other genetic factors on expressivity, complexity, progression, and transmission o...

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Detalles Bibliográficos
Autores principales: Dugo, Ketty, Bruno, Francesca, Sturiale, Valentina, Brancato, Desiree, Saccone, Salvatore, Federico, Concetta
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9598525/
https://www.ncbi.nlm.nih.gov/pubmed/36289657
http://dx.doi.org/10.3390/biomedicines10102394

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9598525/
https://www.ncbi.nlm.nih.gov/pubmed/36289657
http://dx.doi.org/10.3390/biomedicines10102394

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