Paper Details

  1. Home
  2. Paper Details

Targeting transthyretin - Mechanism-based treatment approaches and future perspectives in hereditary amyloidosis.

Author: CoelhoTeresa, DohrnMaike F, HahnKatrin, HegenbartUte, IhneSandra, MedinaJessica, ZüchnerStephan L

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Overview

Transthyretin (TTR) amyloidosis is a lethal disease caused by the liver-derived transport protein TTR. This systematic review summarizes the current literature on TTR pathophysiology and therapeutic implications, including approved therapies like tafamidis, patisiran, and inotersen, and discusses future directions in research.
Paper Details　
Original Abstract of the Article :
The liver-derived, circulating transport protein transthyretin (TTR) is the cause of systemic hereditary (ATTRv) and wild-type (ATTRwt) amyloidosis. TTR stabilization and knockdown are approved therapies to mitigate the otherwise lethal disease course. To date, the variety in phenotypic penetrance i...See full text at original site
Dr.Camel IconDr.Camel's Paper Summary Blogラクダ博士について

ラクダ博士は、Health Journal が論文の内容を分かりやすく解説するために作成した架空のキャラクターです。
難解な医学論文を、専門知識のない方にも理解しやすいように、噛み砕いて説明することを目指しています。

* ラクダ博士による解説は、あくまで論文の要点をまとめたものであり、原論文の完全な代替となるものではありません。詳細な内容については、必ず原論文をご参照ください。
* ラクダ博士は架空のキャラクターであり、実際の医学研究者や医療従事者とは一切関係がありません。
* 解説の内容は Health Journal が独自に解釈・作成したものであり、原論文の著者または出版社の見解を反映するものではありません。


引用元：
https://doi.org/10.1111/jnc.15233

データ提供：米国国立医学図書館（NLM）

Navigating the Shifting Sands of Amyloidosis: Exploring Therapeutic Strategies Targeting Transthyretin

Imagine a vast desert, where the sands of time relentlessly erode the landscape, leaving behind remnants of a forgotten past. This research explores the complex world of transthyretin (TTR) amyloidosis, a debilitating disease characterized by the accumulation of misfolded TTR protein in various organs. The study examines the latest therapeutic approaches targeting TTR, seeking to develop effective strategies to mitigate the devastating effects of this disease.

A New Oasis of Hope: Exploring TTR Stabilization and Knockdown Therapies

The researchers delve into the intricate mechanisms underlying TTR amyloidosis, exploring the role of TTR mutations and other genetic and environmental factors that contribute to disease progression. The study examines the effectiveness of approved therapies, including TTR stabilizers and knockdown approaches, highlighting their potential to slow down disease progression and improve patient outcomes.

A Road to Recovery: Exploring Emerging Therapeutic Strategies for TTR Amyloidosis

This research, like a caravan navigating a treacherous desert, offers a glimpse into the future of TTR amyloidosis treatment. The researchers explore the potential of novel therapeutic approaches, including bivalent TTR stabilizers, amyloid-directed antibodies, and other promising strategies. The study highlights the need for continued research and development to find more effective treatments for this challenging disease.

Dr. Camel's Conclusion

This research offers a glimmer of hope for individuals living with TTR amyloidosis, highlighting the progress made in understanding and treating this complex disease. It's a reminder that with persistence and innovation, we can find new solutions to even the most daunting medical challenges. Like a desert traveler seeking a hidden oasis, we must continue our relentless pursuit of a cure for this debilitating condition.

Date :
  1. Date Completed 2021-06-18
  2. Date Revised 2021-06-18
Further Info :

Pubmed ID

33155274

DOI: Digital Object Identifier

10.1111/jnc.15233

SNS
PICO Info
in preparation
Languages

English

Positive IndicatorAn AI analysis index that serves as a benchmark for how positive the results of the study are. Note that it is a benchmark and requires careful interpretation and consideration of different perspectives.

This site uses cookies. Visit our privacy policy page or click the link in any footer for more information and to change your preferences.