Chapter 20, Problem 2MCP

Interpretation Introduction

Interpretation:

A hypothesis that explains the inability of antisense oligonucleotides to cross the blood-brain barrier has to be developed.

Concept Introduction:

The blood-brain barrier is the semipermeable and highly selective barrier that allows the movement of only some molecules such as small antibiotics via passive diffusion.

In the antisense gene therapy for Huntington’s disease, single-stranded short oligonucleotides are engineered so that they are complementary to the mRNA sequence of the harmful huntingtin protein. They are then inserted so that they can bind with the target mRNA and thus cause inhibition of translation. This prevents the formation of any mutated form of enzyme.