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On genetic engineering and CRISPRS: If a gene is
modified in an individual cell how does that
translate to all the other billions of genes in an
organism? Given that the changes you make in one
cell will replicate in new cells, how many cells
need to be modified to repair whatever you want
fixed, and how long does it take? |
Question Date: 2017-03-16 | | Answer 1:
Using CRISPR on zygotes is easier for this
reason than trying to cure diseases in adulthood.
Obviously, CRISPR in adults is a lot trickier,
since the daughter cells of one transgenic cell is
a much smaller proportion of the entire organism
(or to put it another way, most of an adult's
tissues have already differentiated). Rather than
trying to replace all the non-functional/diseased
cells with cells from a CRISPR lineage (which
would be really hard to do), researchers have to
get more creative.
The first clinical trial using CRISPR in humans
started only a few months ago (our current date is
March 2017) (check out this article:
gene editing ). In this case, the scientists
took some of the patient's immune cells out of
their body, cultured them in a dish, used CRISPR
to change the genes they wanted, and then injected
the cells back into the patient.
This allowed them to create cells that would be
good at targeting cancer, but would not be
degraded by the host's own immune system. The
scientists did not have to worry about changing
all of the patient's immune cells because they
were able to generate enough in the lab to fight
the disease.
Hereditary diseases in adults (such as
Huntington's or cystic fibrosis) will be harder to
fight with this technology because thousands
of cells in the body would probably have to be
reprogrammed. There might be other ways of
delivering a gene to cells, such as using a
genetically engineered virus (known as
gene vector therapy). This could allow a
greater number of cells to incorporate the gene of
interest. However, there are challenges there as
well, such as generating a virus that will target
very specific cell types.
| | Answer 2:
The reason CRISPR has been so successful is
because it specifically edits only a single gene,
not any unrelated ones. Also, no known
organism has billions of genes. Humans have about
20,000 genes though their DNA has billions of
nucleotide base pairs. So, to clarify,
technically CRISPR modifies a small number of base
pairs. In fact, what makes a sequence of DNA a
gene or not is not that straight forward. But
it is true that modifying a single gene, while
not modifying the DNA of other genes, could affect
the function of the other genes. If you use
CRISPR to modify a gene which ultimately activates
other genes, then it could have an effect. To
actually use CRISPR, unless you’re modifying a
fertilized egg, you are probably going to modify
more than 1 cell. How many cells are needed for
repair a function will depend on the
application. A single human cell takes around
12-24 hours to divide so if you don’t start with
many cells, it would likely take weeks to months
before the faulty function is repaired.
| | Answer 3:
The cell that you modify will be different, but no
other cell will be affected. If the cell then
divides, then the cells that come from it will
also be different, but not the cells that come
from other cells. To "repair" a human, say, you
would need to alter the genome of every cell in
the human's body. This would take so long that
you can't effectively do it, which is why genetic
engineering is almost always done on the
reproductive cells: if you alter the genome of an
animal while that animal is a single cell, then
that will affect the entire animal because every
cell in the animal's body is descended from that
single cell.
Now, if the problem you're trying to correct is
the lack of a hormone or some other chemical that
a cell produces and then secretes, then you don't
have to affect every cell, just the cells of the
organ producing the chemical, and even then only
enough of them to produce enough of the needed
chemical.
| | Answer 4:
CRISPR is a really powerful tool for editing
DNA but it is extremely difficult to use on
humans for a variety of reasons, both scientific
and philosophical. Let’s start with the science
because, surprisingly enough, it’s the easier part
to answer.When you edit with CRISPR, you change
a specific portion of the DNA in a single
cell. All edits that are made with CRISPR are
exclusive to the cell the edits were made. Now,
that one cell can go onto proliferate and make
many daughter cells that will all carry the CRISPR
mutation, but it won’t pass the mutation onto
cells near it. For this reason, you cannot just
use CRISPR for a person with blue eyes to make
them have brown eyes. You may now be thinking,
“couldn’t I just CRISPR ever eye cell so that they
all have the brown mutation”, and the answer is …
maybe? There’s nothing that says that you
couldn’t do this besides the fact that CRISPR as
an experimental technique isn’t perfect enough to
do this. So if you wanted to apply CRISPR to fix
genetic diseases, you would need to target every
cell that expresses the disease. Realistically,
this can only be done when the embryo is only a
few cells large. So there’s good news and bad
news. We can use CRISPR to fix genetic diseases
but only if the disease is found very early in
development.
Now that we’ve considered the
scientific hurdles of using CRISPR on humans,
let’s think about the moral implications. For one,
CRISPR is fairly new technique and we do not fully
understand it yet. We are pretty sure we
understand what happens when we CRISPR single
cells, but doing the same to a multicellular
organism like a human is a totally different
story. What if CRISPR had some devastating
effect that impaired or killed the fetus? We
don’t know what could happen and it is morally
wrong to test. Furthermore many people argue that
physically editing human DNA is going too far.
They would argue humans shouldn’t have that sort
of power to design life. On the other hand, others
would argue that if we have a way to eliminate
debilitating genetic diseases then we should. I
don’t think there is an objective correct answer,
but it is something to consider. Thank you for the
question, and I hope you take the time to think
about the ethical and scientific effects
that using CRISPR on people can have. Click Here to return to the search form.
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