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Delivery of Proteins, Peptides or Cell-impermeable Small Molecules into Live Cells by Incubation with the Endosomolytic Reagent dfTAT
Published on: September 2, 2015
Folding proteins in fatal ways
1Center for Neurologic Diseases, Harvard Medical School, Brigham and Women's Hospital, Boston, Massachusetts 02115, USA. dselkoe@rics.bwh.harvard.edu
Nature
|December 20, 2003
Summary
Protein misfolding causes insoluble deposits leading to neurodegenerative diseases like Alzheimer's and Parkinson's. Understanding protein folding offers new therapeutic strategies for these challenging amyloidosis conditions.
Area of Science:
- Neuroscience
- Biochemistry
- Pathology
Background:
- Human diseases involving insoluble protein deposits (amyloidoses) have been known for nearly 200 years.
- These conditions were previously considered secondary and of minor importance.
- Emerging evidence highlights their significant pathogenic role.
Purpose of the Study:
- To explore the link between protein misfolding and the development of human diseases.
- To understand the mechanisms underlying protein aggregation in neurodegenerative disorders.
- To identify potential therapeutic avenues based on protein folding principles.
Main Methods:
- Review of existing literature on protein folding and amyloidosis.
- Analysis of the role of protein aggregates in cellular dysfunction.
- Examination of specific examples in neurological diseases.
Main Results:
- Misfolded proteins can form extracellular or intracellular aggregates.
- These aggregates cause significant cellular dysfunction.
- Neurodegenerative diseases like Alzheimer's and Parkinson's are prime examples.
Conclusions:
- Protein misfolding is a central mechanism in various human diseases.
- Understanding protein folding provides critical insights into disease pathogenesis.
- This knowledge facilitates the development of novel therapeutic strategies for amyloidosis and related disorders.
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