Diagnosis and Therapeutic Approaches to Transthyretin Amyloidosis
The liver transplantation for hereditary ATTR amyloidosis has become a well-established treatment, because the main source of serum variant TTR is shut out. However, this treatment has several problems, such as expensive medical costs, lifelong administration of immunosuppressants, non-indication for the mutated-TTR gene carriers without clinical symptoms, shortage of liver donors, and further development of cardiac and ocular disorders. Therefore, we and other ATTR amyloidosis research groups have been investigating the possibility of stabilization of variant TTR, gene therapy, and immunotherapy for ATTR amyloidosis on the basis of TTR amyloid formation mechanism.
We present here the current diagnostic procedure and therapeutic approaches for the disease.
Keywords: ATTR amyloidosis; Amyloidosis; Hereditary; amyloidogenic transthyretin; autosomal dominant; biochemical and molecular genetic; gene therapy; immunotherapy; senile systemic amyloidosis; transthyretin
Document Type: Research Article
Affiliations: Department of Diagnostic Medicine, Graduate School of Medical Sciences, Kumamoto University, 1-1-1 Honjo, Kumamoto 860-0811, Japan.
Publication date: 2012-05-01
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