Searching for Small Molecules that Can Break Down Protein Aggregates Involved in Neurodegenerative Disease
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27

Sep

2018

27

Sep

2018

Searching for Small Molecules that Can Break Down Protein Aggregates Involved in Neurodegenerative Disease

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Considerable effort in the research community is devoted to the seawrch for small molecule drugs that can break down or inhibit formation of the protein aggregates associated with various forms of neurodegenerative disease. One of these is α-synuclein, a prominent feature of Parkinson’s disease. Potential treatments based on clearance of α-synuclein are at varying points in the development and regulatory approval pipeline. The materials here provide one of many examples of continued efforts to produce new drug candidates that can enter that pipeline. This is an uncertain process: scanning the compound libraries for new possibilities has unknown (but certainly low) odds of success in any given case. It is expensive and slow besides, and few sources of funding are willing to roll the dice given those points.

Parkinson’s disease (PD) is characterized by a progressive loss of dopaminergic neurons, a process that current therapeutic approaches cannot prevent. In PD, the typical pathological hallmark is the accumulation of intracellular protein inclusions, known as Lewy bodies and Lewy neurites, which are mainly composed of α-synuclein. Recently, we have developed an

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