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Sep 13

Research Bit: Therapeutic correction of nuclear transport dysfunction in ALS

Research Bits
The Packard Center welcomed Tom Lloyd from Johns Hopkins University to a recent Investigator's Meeting.

Meeting Date: 13 September 2019

Presenter: Tom Lloyd

Talk Title: Therapeutic correction of nuclear transport dysfunction in ALS


What was the question being asked?

Neurons have two main compartments: the nucleus, which houses the cell’s DNA and produces RNA, and the cytoplasm, where all of the cell’s protein is made by decoding RNA. It has recently been shown by many groups that the transport of RNA and protein between these two compartments is altered by mutations associated with ALS. The work presented here was aimed toward identifying potential therapies that can be used to restore the normal transport function within motor neurons, in an attempt to repair some of the damage caused by the ALS mutations.

Why is this important for ALS research?

Since the end goal of all ALS research is to understand and develop treatment for patients with ALS, this work is important as one of the many steps that it takes to produce a drug for ALS patients.

What was the take-home message?

Using a drug called KPT-350, the presented experiments showed promising results that the defects in transport between the nucleus and cytoplasm can be reversed, which ultimately reduces the risk of death of motor neurons.

How do you think the results of this study might impact future approaches to the treatment of ALS?

Based on the experiments that have been performed in many model systems, KPT-350 has already made it to clinical trials. Soon, we will understand how effective the treatment is in slowing or stopping the progression of ALS.

Ben Zaepfel
Ph.D. Candidate | Rothstein Lab
Biochemistry, Cellular and Molecular Biology Ph.D. Program
The Johns Hopkins School of Medicine

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