Lab techniques in ALS research

Question 1

How much % of ALS is familial

Answer 1

10%

Question 2

Which other condtions does ALS show overlaps with and presents in 5% of patients together?

Answer 2

FTD

Question 3

What gene mutation has been identified to cause ALS

Answer 3

TDP-43

Question 4

What does APO stand for in the context of gene therapies?

Answer 4

APO likely refers to antisense oligonucleotide (ASO), which are short, synthetic nucleic acids used to selectively bind to complementary RNA targets in gene therapy.

Question 5

How can antisense oligonucleotides (ASOs) be used in gene therapy?

Answer 5

ASOs can be designed to inhibit the expression of disease-causing genes by targeting specific RNA molecules involved in disease processes. Alternatively, they can be used to enhance the expression of therapeutic genes, offering a versatile approach in gene therapy applications.