Please help Why did we use biodegradable nanoparticles? Please use The worksheet below and don’t copy and paste from Google thank you

Human Anatomy & Physiology (11th Edition)
11th Edition
ISBN:9780134580999
Author:Elaine N. Marieb, Katja N. Hoehn
Publisher:Elaine N. Marieb, Katja N. Hoehn
Chapter1: The Human Body: An Orientation
Section: Chapter Questions
Problem 1RQ: The correct sequence of levels forming the structural hierarchy is A. (a) organ, organ system,...
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Please help Why did we use biodegradable nanoparticles? Please use The worksheet below and don’t copy and paste from Google thank you
Abstract
Did you know that the cell copies 50 nucleotides (letters of
DNA code) per second when it is dividing? And it only makes
one mistake per 100 million nucleotides! That's like copying
the full 32 volumes of Encyclopedia Britannica twelve times
and only making one typo!
Most times even these mistakes are caught and fixed. But
sometimes a mutation (mistake in the code) gets passed
on. In eggs and sperm that means an unborn baby will get
one bad copy of that gene.
Introduction
Imagine you misspelled one letter of one word on an
assignment, and the teacher gave you a 0% for the whole
thing. Not fair!
But this is what happens in diseases like B-thalassemia. Just
one of the nucleotides in the DNA code for a gene is copied
wrong, and yet the molecule that gene is supposed to make
can't do what it needs to.
B-thalassemia is a blood disorder. People with this disease
cannot produce hemoglobin, the molecule in red blood cells
that carries oxygen through the blood.
We would like to fix the section of their DNA. Then they
could produce the oxygen-carrying molecule on their own!
This is called genetic editing. If we correct the bad letter of
the DNA code we can cure a person for life!
That's very difficult, though: bodies are very good at
destroying the molecules we use to do genetic editing. Most
of the time this is a very good thing. Outside of the lab,
In most cases, even this is okay. The baby is a carrier of
a bad copy of the gene, but often the good copy from the
other parent will work well enough. In rare cases, though,
a baby may receive a bad copy from both parents. This
means they will have a genetic disease.
There are several diseases that are caused by a single
nucleotide mutation. Scientists have always wanted to use
genetic editing to correct the bad part of the gene. We
found a way to do it in real, live mice!
Fig. 1. A dose-up of our nanoparticles inside a baby
mouse's blood vessels. Here they carry a fluorescent
molecule. They're so bright because they are loaded with
lots of molecules, but they're still so small that they can
travel through the tiniest blood vessels!
(See the full video on our website!)
most unfamiliar molecules are from a virus or infection.
But it makes our job very hard.
We thought we could solve this problem using nanoparticles.
Transcribed Image Text:Abstract Did you know that the cell copies 50 nucleotides (letters of DNA code) per second when it is dividing? And it only makes one mistake per 100 million nucleotides! That's like copying the full 32 volumes of Encyclopedia Britannica twelve times and only making one typo! Most times even these mistakes are caught and fixed. But sometimes a mutation (mistake in the code) gets passed on. In eggs and sperm that means an unborn baby will get one bad copy of that gene. Introduction Imagine you misspelled one letter of one word on an assignment, and the teacher gave you a 0% for the whole thing. Not fair! But this is what happens in diseases like B-thalassemia. Just one of the nucleotides in the DNA code for a gene is copied wrong, and yet the molecule that gene is supposed to make can't do what it needs to. B-thalassemia is a blood disorder. People with this disease cannot produce hemoglobin, the molecule in red blood cells that carries oxygen through the blood. We would like to fix the section of their DNA. Then they could produce the oxygen-carrying molecule on their own! This is called genetic editing. If we correct the bad letter of the DNA code we can cure a person for life! That's very difficult, though: bodies are very good at destroying the molecules we use to do genetic editing. Most of the time this is a very good thing. Outside of the lab, In most cases, even this is okay. The baby is a carrier of a bad copy of the gene, but often the good copy from the other parent will work well enough. In rare cases, though, a baby may receive a bad copy from both parents. This means they will have a genetic disease. There are several diseases that are caused by a single nucleotide mutation. Scientists have always wanted to use genetic editing to correct the bad part of the gene. We found a way to do it in real, live mice! Fig. 1. A dose-up of our nanoparticles inside a baby mouse's blood vessels. Here they carry a fluorescent molecule. They're so bright because they are loaded with lots of molecules, but they're still so small that they can travel through the tiniest blood vessels! (See the full video on our website!) most unfamiliar molecules are from a virus or infection. But it makes our job very hard. We thought we could solve this problem using nanoparticles.
Methods
What do we need to genetically edit DNA?
Essentially we need just three components:
A donor DNA with the correct sequence - it serves as a
template. We want the cell to use this correct sequence
to replace the mutated section of the gene.
(2) An editor molecule. This molecule physically breaks the
DNA backbone of the mutated gene and inserts the
donor DNA in its place.
3. A specificity molecule. This molecule tells the editor
molecule exactly where the DNA strand needs to be cut
for our donor DNA to get added.
The editor molecule makes permanent changes to DNA. We need
a very accurate one, or we might create new mutations! So we
decided to use the safest editor molecule out there: the cell's
own DNA repair molecules. The native DNA repair molecules can
detect unusual structures, and swap in the donor DNA to fix them.
But we also had to pick the right specificity molecule. Peptide
nudeic acids (PNAS) bind to double-stranded DNA to make a
strange-looking triple-stranded PNA-DNA segment, which is
recognized by the native DNA repair molecules.
Lastly, we needed to pick the right kind of vehicle to deliver our
editing molecules. We decided to use a biodegradable polymer
nanoparticle that is known to break down to safe natural
molecules after its job is done.
DNA replication is the fastest in fetal (unborn) animals, so we
decided to genetically edit mice in utero (before their birth). We
then injected the donor DNA and PNA-loaded nanopartides into
fetal mice with the ß-thalassemia mutation and waited for the
results!
Fig. 2. Hemoglobin levels of mice
with B-thalassemia. Untreated
mice (black boxes) have low
levels, while mice treated with our
PNA-nanopartides (blue boxes)
have levels in the normal range.
(The shaded area marks the
normal range of hemoglobin for
healthy mice.)
16
14
12
00
6
Legend:
No treatment
PNA/Nanoparticle treatment
Hemoglobin concentration (grams/deciliter)
Low hemoglobin Normal range
HOH
6 weeks
10 weeks
Did you know that this type of plot is called "box
and whisker"? It is a graphical representation of a range
of data points. The box represents the middle half of the
values (if you were to line all the values up in order, from 14
to 34 of the way through), and the whiskers show the full
range of the data values.
Transcribed Image Text:Methods What do we need to genetically edit DNA? Essentially we need just three components: A donor DNA with the correct sequence - it serves as a template. We want the cell to use this correct sequence to replace the mutated section of the gene. (2) An editor molecule. This molecule physically breaks the DNA backbone of the mutated gene and inserts the donor DNA in its place. 3. A specificity molecule. This molecule tells the editor molecule exactly where the DNA strand needs to be cut for our donor DNA to get added. The editor molecule makes permanent changes to DNA. We need a very accurate one, or we might create new mutations! So we decided to use the safest editor molecule out there: the cell's own DNA repair molecules. The native DNA repair molecules can detect unusual structures, and swap in the donor DNA to fix them. But we also had to pick the right specificity molecule. Peptide nudeic acids (PNAS) bind to double-stranded DNA to make a strange-looking triple-stranded PNA-DNA segment, which is recognized by the native DNA repair molecules. Lastly, we needed to pick the right kind of vehicle to deliver our editing molecules. We decided to use a biodegradable polymer nanoparticle that is known to break down to safe natural molecules after its job is done. DNA replication is the fastest in fetal (unborn) animals, so we decided to genetically edit mice in utero (before their birth). We then injected the donor DNA and PNA-loaded nanopartides into fetal mice with the ß-thalassemia mutation and waited for the results! Fig. 2. Hemoglobin levels of mice with B-thalassemia. Untreated mice (black boxes) have low levels, while mice treated with our PNA-nanopartides (blue boxes) have levels in the normal range. (The shaded area marks the normal range of hemoglobin for healthy mice.) 16 14 12 00 6 Legend: No treatment PNA/Nanoparticle treatment Hemoglobin concentration (grams/deciliter) Low hemoglobin Normal range HOH 6 weeks 10 weeks Did you know that this type of plot is called "box and whisker"? It is a graphical representation of a range of data points. The box represents the middle half of the values (if you were to line all the values up in order, from 14 to 34 of the way through), and the whiskers show the full range of the data values.
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