Amyotrophic lateral sclerosis (ALS)

Definition

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord. It leads to muscle weakness, paralysis, and eventually respiratory failure.

5 Must Know Facts For Your Next Test

1. ALS is linked to mutations in several genes, including SOD1, TARDBP, and FUS.
2. RNA sequencing has been used to identify biomarkers and understand gene expression changes in ALS patients.
3. CRISPR-Cas9 technology is being researched for potential therapeutic interventions by correcting genetic mutations associated with ALS.
4. Protein misfolding and aggregation are hallmark features of ALS pathology, often involving proteins like TDP-43.
5. ALS research utilizes model organisms such as mice and zebrafish to study disease mechanisms and test potential treatments.

Review Questions

• What are some of the key genes implicated in ALS?
• How is RNA sequencing useful in studying ALS?
• What role does CRISPR-Cas9 play in potential ALS treatments?

Related terms

RNA Sequencing: A technique used to analyze the quantity and sequences of RNA in a sample.

CRISPR-Cas9:

A genome editing tool that allows for precise modifications to DNA.

Protein Misfolding:

The process where proteins fail to fold into their functional three-dimensional structures.