Central Dogma and Genetic Medicine Click & Learn

Student Worksheet

OVERVIEW
This worksheet complements the Central Dogma and Genetic Medicine Click & Learn.

PROCEDURE
As you proceed through the Click & Learn, follow the instructions below and answer the questions in the spaces
provided.

1. Let’s review! The central dogma of molecular biology refers to the process of gene expression. Write the
definition of gene expression in your own words.
It is the translation of information from DNA sequences into actual synthesis of molecules.

2. Click on the “Central Dogma” menu tab at the top of the screen.

The table below outlines the steps in eukaryotic gene expression. Click on each tab or scroll through the page

and briefly summarize each step below.

Gene Molecules Involved

Summary
Expression What molecules and proteins
What happens during this step?
Steps are involved in this step?
RNA polymerase The enzyme transcribes a gene’s DNA into RNA
transcript with a complimentary sequence.
Transcription

Spliceosome The RNA transcript is edited through splicing, the

enzymes trims out introns and splices together
RNA Splicing exons.

tRNA mRNA is transported from the nucleus to the

mRNA cytoplasm.
Transport

Ribosome The ribosome translates mRNA code into amino

acids.
Translation

Cytoplasm, ER, and golgi The poplypeptide chaine is folded and assembled
Protein system. into a 3D functional protein.
Processing

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 Click & Learn
Central Dogma and Genetic Medicine Student Worksheet

3. Mutations in the DNA can affect the structure and function of proteins. Some mutations may even cause
genetic diseases. Scientists and doctors can intervene at different points during gene expression to develop
treatments for such genetic diseases—or genetic medicine. Let’s learn about the genetic medicines that are
being developed.

Select the “Genetic Medicine” tab located on the top right of the screen. Click on the tab corresponding to the
genetic medicine(s) that your instructor assigns to you, or scroll through the interactive and click on the pink
“+” sign labeled with that genetic medicine. Read the “Genetic Medicine” tab material, watch the video, and
read the information in the “Learn more” link. Then, complete the appropriate row(s) below.

Detailed Description
Genetic Short Summary
Describe how this genetic medicine would be used to treat a genetic
Write a one-sentence summary of
Medicine disease. (For example, mention how it would fix the disease-causing
how this genetic medicine works.
mutation and/or result in a functioning protein.)
a technological tool that Technology can be used to alter a gene to correct a
can change DNA. disease-causing mutation as well as block a gene from
CRISPR- expressing itself.
Cas9

a technological tool that As a viral vector enters the body and infects specific
delivers genes inside the cells, it releases its genome. It makes its genome
Gene cell. public. The cells then produce a protein.
Therapy

turning genes on and off. Genes that cause sickness can be disabled.

Gene
Switches

a single strand of RNA that With the resulting single-stranded RNA, one or more
that the splicosome splits exons, including those with the disease-causing
Exon over. mutation, were spliced out.
Skipping

a tool that can remove can be used to turn off undesirable genes.
segments of RNA.
RNA
Interference

a tool that can remove These drugs target or interact with small molecules to
Small segments of RNA. directly counteract the detrimental effects of disease-
Molecule causing proteins.
Drug

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 Click & Learn
Central Dogma and Genetic Medicine Student Worksheet

4. Now let’s learn about some of the diseases that may be treated using these genetic medicines.

Scroll through the interactive and click on the pink “+” sign that is labeled with the disease(s) that your
instructor has assigned to you. Next, click on the “Case Study” tab at the top to reveal information about the
disease. Read the “Case Study” material, watch the video, and read the information in the “Learn more” link.
Then fill in the appropriate row(s) in the table below.

Name of What are the key characteristics of the disease and whom does it affect?
Disease How can the featured genetic medicine be used to treat the disease?
One of the key signs of the illness is extreme farsightedness. It has an impact on
the retina. Gene therapy can be used to treat this condition by designing a
Leber therapeutic gene.
Congenital
Amaurosis

One of the key characteristics of this condition is HBB gene mutations. As a

result, red blood cells take on the sickle shape, which increases the body's need
for oxygen. Genetic treatment can treat this problem by turning on a gene that is
Sickle Cell normally inactive after birth.
Disease

The primary characteristics of this condition are mutations in the gene that
creates the protein dystrophin in muscle cells. Impacted muscles and muscular
Duchenne fibers. Exon skipping genetic medicine can be used to cure this issue by requiring
Muscular the faulty exons to be spliced out.
Dystrophy

The HTT gene, which is essential for the proper functioning of nerve cells, is one
of this disease's primary hallmarks. It damages the brain. RNA interference
genetic therapy can treat this problem by removing the offending HTT mRNA.
Huntington’s
Disease

The main symptoms of this condition are CFTR gene mutations. Affected are the
linings and the airways. Small-molecule medicines with genetic medicine can be
used to treat this illness by concentrating on each molecule and improving the
Cystic Fibrosis processing of the mutant CFTR protein.

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Central Dogma and Genetic Medicine Student Worksheet

APPLY WHAT YOU HAVE LEARNED

5. You are a researcher working on a treatment for Hutchinson-Gilford progeria syndrome, an extremely rare
genetic disorder that causes accelerated aging in children. Children with progeria generally appear healthy
at birth but soon start growing more slowly than other children and lose their hair. Additional symptoms
include stiffness of joints, heart problems, and stroke. These children typically die of heart disease at an
average age of 13 years.

Progeria is caused by a mutation in a single gene, called lamin A. Scientists have identified over 1,400
mutations in the lamin A gene that result in changes in transcription, RNA splicing, and/or protein
production. Lamin A codes for a protein required for the structural support of the nuclear envelope in cells.
Without a functional protein, the nuclear envelope becomes unstable, eventually damaging the nucleus and
causing cells to die.

Based on what you learned in this Click & Learn, propose a genetic medicine strategy you could develop to
treat patients with progeria. Describe which step in gene expression you might target and why you would
target that step, the intervention tool you would use, and explain how this strategy would treat the disease.
It has been demonstrated that bacteria have CRISPR DNA sequences, which are repetitive DNA
sequences, and "spacer" DNA sequences, which perfectly match virus sequences in between
the repeats. It was later found that bacteria turn these DNA components into RNA in response to
viral infection. The nuclease protein, which also serves as a virus defense, is targeted by the
RNA for cleavage by the viral DNA section.

When the so-called guide RNA is created particularly for that one region, there is a higher
likelihood that the DNA will be cut there and nowhere else in the genome.

How CRISPR can be used to treat progeria:

The typical approach involves using the Cas9 protein to cut DNA, which can silence the mutant
gene.

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