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.

Question

Want to see the full answer?

Answer 1

Question

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.

Expert Solution & Answer

Want to see the full answer?

Check out a sample textbook solution

See solution

Students have asked these similar questions

Complete the clean-pushing mechanism for the given ether synthesia from propanol in concentrated sulfurica140°C by adding any mining aloms, bands, charges, nonbonding electron pairs, and curved arrows. Draw hydrogen bonded to cayan, when applicable. ore 11,0 HPC Step 1: Draw curved arrows Step 2: Complete the intend carved Q2Q 56 QQQ Step 3: Complete the intermediate and add curved Step 4: Modify the structures to draw the QQQ QQQ

6. In an experiment the following replicate set of volume measurements (cm3) was recorded: (25.35, 25.80, 25.28, 25.50, 25.45, 25.43) A. Calculate the mean of the raw data. B. Using the rejection quotient (Q-test) reject any questionable results. C. Recalculate the mean and compare it with the value obtained in 2(a).

A student proposes the transformation below in one step of an organic synthesis. There may be one or more reactants missing from the left-hand side, but there are no products missing from the right-hand side. There may also be catalysts, small inorganic reagents, and other important reaction conditions missing from the arrow. • Is the student's transformation possible? If not, check the box under the drawing area. • If the student's transformation is possible, then complete the reaction by adding any missing reactants to the left-hand side, and adding required catalysts, inorganic reagents, or other important reaction conditions above and below the arrow. • You do not need to balance the reaction, but be sure every important organic reactant or product is shown. + T G OH де OH This transformation can't be done in one step.