Antisense Oligonucleotide Therapy

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Amyotrophic lateral sclerosis (ALS) is a rare neurodegenerative disease which targets motor neurons cells responsible for controlling involuntary muscles functions. The disease is also called “Motor Neuron Disease (MND)” and “Lou Gehrig’s disease”. This is a fatal neurological disease caused due to the death of motor neurons cells leading to paralysis and premature death.

The disease has varied symptoms in varied individuals, some have trouble in speaking, eating, chewing while another may face trouble in writing or picking up things Although the mean survival time for ALS patients is 5-10 years, the rate at which ALS progress varies from person to person. The early symptoms of the disease begin to appear in muscles which include stiff muscles, muscle twitches, muscle weakness, slurred and nasal speech, uncontrolled voice pitch, muscular cramps.

Amyotrophic lateral sclerosis (ALS) is a rare neurodegenerative disease which targets motor neurons cells responsible for controlling involuntary muscles functions. The disease is also called “Motor Neuron Disease (MND)” and “Lou Gehrig’s disease”. This is a fatal neurological disease caused due to the death of motor neurons cells leading to paralysis and premature death. The disease has varied symptoms in varied individuals, some have trouble in speaking, eating, chewing while another may face trouble in writing or picking up things Although the mean survival time for ALS patients is 5-10 years, the rate at which ALS progress varies from person to person. The early symptoms of the disease begin to appear in muscles which include stiff muscles, muscle twitches, muscle weakness, slurred and nasal speech, uncontrolled voice pitch, muscular cramps.

The exact causes of the disease are unknown but studies suggest in most of the cases it is genetics and mutation. Although mutations in dozens of genes are responsible but defect in chromosome 21 which encodes SOD1 (Superoxide Dismutase) and “chromosome 9 open reading frame 72,” or C9ORF72 is found to be mainly associated with ALS.

The patients of ALS are put on medication and therapies to prolong life but there has been no permanent cure for this disease. Recent research by ALS Association –USA has found the road to treat ALS targetingSOD1 and C9orf72 with Antisense Oligonucleotide (ASO) therapy. The antisense therapy prevents the production of proteins like SOD1 and C9orf72 responsible for ALS. ASOs are singe stranded short oligonucleotide sequences once delivered to the cell( motor neurons) targets to bind with mRNA of SOD1 inhibiting the formation of SOD1 protein which causes ALS. Scientist believes the antisense therapy may have much impact on C90rf72 mediated ALS. The research is underway to optimize c9orf72 drugs to clinical trials. We expect in future times ASO be a promising approach in treating ALS and other neurological disorders such as Spinal Muscular Atrophy(SMA), Huntington’s Disease, Myotonic dystrophy and many other diseases.

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