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12/22/2024 07:30:58 am

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"Disease in a Dish" Model Offers Hope for Better Huntington's Disease Treatments

Huntington's disease


A man afflicted with Huntington's disease

A new study shows a "diseases in a dish" model can be used to find out more about Huntington's disease.

Huntington's disease is a neurodegenerative disorder that leads to uncontrolled movements and cognitive impairments in adulthood.

It's caused by an autosomal dominant mutation in either of an person's two copies of a gene called Huntingtin. This means a child of an affected person has a 50 percent chance of inheriting the disease.

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Anthony Chan from Emory University School Medicine said their "diseases in a dish" model showed the development of cellular features of the disease like intranuclear inclusion of the Huntingtin protein that other cell models do not present.

Chan believes that they could use the features for drug therapy and genetic manipulation.

To generate these findings, researchers developed iPS cells coming from transgenic with reprogrammed cells derived from their skin or dental pulp.

They said the technique reprogrammed factors into somatic cells that became pluripotent stem cells able to differentiate any type of cell in the body.

They also induced the iPS cells to become neural progenitors able to differentiate neurons that developed intracellular and intranuclear aggregates on the huntingtin protein.

The Huntingtin protein is a classic sign of Huntington's that can increase the sensitivity of oxidative stress, researchers said.

Researchers noted oxidative stress sensitivity is a useful indicator that could ameliorate in cell culture either by an RNA-based gene knockdown approach or memantine drug.

Chan is confident their findings are proof of principle that this system could be a valuable tool that can lead to other therapies.

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