Personalized gene therapy for ALS patient set to proceed for the second time ever.

Amyotrophic Lateral Sclerosis(ALS) is a progressive neurodegenerative disease that affects nerve cells (motor neurons) of the brain and the Spinal cord. Motor neurons connect the brain to the spinal cord and from the spinal cord to the muscles of the body. Through motor neurons, the brain controls the movement of our bodies. In a patient suffering from ALS, motor neurons degenerate. Then the brain lost the ability to control muscle movement, and the patient started to loses the ability to speak, eat, move and breathe.

There are two types of ALS: Familial and sporadic. Sporadic is the most common form of ALS which accounts for 90-95 % of the cases. It may affect anyone, anywhere. Familial ALS is inherited. In those families, there is a 50% chance of each offspring will inherit a gene mutation and may develop the disease.

Jaci Hermstad (center) and her parents. Harvest Moon Photography by Hilary

A young woman who has developed an aggressive, early-onset form of ALS which killed her twin sister is set to receive a cutting-edge form of personalized medicine, gene therapy is yet to be approved for use. It involves designing a molecule that binds to the mutated strands of the gene and prevents the production of mutated protein that causes diseases.

The woman will become the second ever to try the approach. The first use was on another neurodegenerative disease, Batten disease.

References: Tailored treatment for ALS poised to move ahead. NATURE. 30th May 2019.
doi: 10.1038/d41591-019-00013-w

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