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Updated: Aug 11, 2026

Genetic Analysis of Hereditary Transthyretin Ala97Ser Related Amyloidosis
06:33

Genetic Analysis of Hereditary Transthyretin Ala97Ser Related Amyloidosis

Published on: June 9, 2018

Systemic amyloidosis

Helen J Lachmann1, Philip N Hawkins

  • 1National Amyloidosis Centre, Department of Medicine, Royal Free and University College Medical School, Royal Free Campus, London NW3 2PF, UK. h.lachmann@medsch.ucl.ac.uk

Current Opinion in Pharmacology
|February 18, 2006
PubMed

Insights

Amyloidosis involves abnormal protein deposits in organs, posing diagnostic challenges. Novel therapies target amyloid formation and deposits, potentially treating rare systemic forms and common diseases like Alzheimer's and type II diabetes.

Area of Science:

  • Biochemistry
  • Pathology
  • Medicine

Background:

  • Amyloidosis is a group of diseases characterized by abnormal extracellular deposition of insoluble protein fibrils.
  • These conditions affect multiple organ systems and present significant diagnostic and management difficulties.

Purpose of the Study:

  • To summarize the current understanding of amyloidosis.
  • To discuss emerging therapeutic strategies targeting amyloid formation and deposits.

Main Methods:

  • Review of current literature on amyloidosis pathogenesis and treatment.
  • Analysis of novel therapeutic approaches including fibrillogenesis inhibition and amyloid destabilization.

Main Results:

  • Current treatments focus on reducing precursor protein supply and supportive care.
  • Emerging strategies aim to inhibit amyloid formation or break down existing deposits.

Conclusions:

  • Advances in understanding amyloid formation are driving novel therapeutic development.
  • These novel strategies may offer effective treatments for systemic amyloidosis, Alzheimer's disease, and type II diabetes.

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