
Gene editing saves girl dying from leukaemia in world first - cedricr
https://www.newscientist.com/article/dn28454-gene-editing-saves-life-of-girl-dying-from-leukaemia-in-world-first/
======
fasteo
In this particular case, they have used TALEN, but we have two additional gene
editing techniques that show promise. Namely,

\- ZFN [2]

\- CRISPR [3]

I have great hopes in all of them and in fact, my genetic condition [4] has
already been fixed in-vitro using both mitoTALENs [5] and ZFN [6].

Exciting times for medicine. Too bad that we have a long, desperate
bureaucratic burden ahead.

[1] [https://en.wikipedia.org/wiki/Transcription_activator-
like_e...](https://en.wikipedia.org/wiki/Transcription_activator-
like_effector_nuclease)

[2]
[https://en.wikipedia.org/wiki/Zinc_finger_nuclease](https://en.wikipedia.org/wiki/Zinc_finger_nuclease)

[3]
[https://en.wikipedia.org/wiki/CRISPR](https://en.wikipedia.org/wiki/CRISPR)

[4]
[http://www.ninds.nih.gov/disorders/mitochondrial_myopathy/mi...](http://www.ninds.nih.gov/disorders/mitochondrial_myopathy/mitochondrial_myopathy.htm)

[5]
[http://www.nature.com/nm/journal/v19/n9/full/nm.3261.html](http://www.nature.com/nm/journal/v19/n9/full/nm.3261.html)

[6]
[http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3992073/](http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3992073/)

~~~
yarou
It truly is an exciting time to live in.

CRISPR in particular looks promising for editing human embryos, and we may be
on the cusp of a post-cancer world with CAR T-Cell Therapy.

One of my coworkers, who has the same cancer as my deceased father, is going
for an experimental treatment involving CAR T-Cell therapy.

Unfortunately, there will always be a bureaucratic burden, but I doubt it will
restrict the progress of these types of technologies and innovation.

------
drjackal
On Monday I got the worst phone call of my life. My sister, who is in her 40s
has been diagnosed with Leukemia. The variant she has is Acute Myeloid
Leukemia (AML) which has a 5 year survival rate of only about 30%. She seemed
healthy just a few days before diagnosis, she was just a bit tired and had low
appetite. Then she noticed some abnormality in her vision and that's when she
did a blood test and found extremely low hemoglobin count.

Shows how quickly your world can come crashing. Cancer is awful. Cancer and
global warming are where most of the worlds research dollars should be going.
This story brings hope for the future.

~~~
cema
Why global warming?

~~~
coob
Really? You're going to be that person?

~~~
cema
Oh, so it's political. Never mind then.

(If it is not political, I still would like to see the answer.)

------
medymed
Would be fascinating if they could harvest healthy host progenitor cells,
destroy the leukemic cells with another persons's engineered off-the-shelf
graft which has a couple of chemically (e.g. tetracycline) inducible kill-
switches, and wait until the graft destroys the cancer to turn on the kill
switch and replant the patient's stored cells. This could prevent lifelong
graft versus host disease, but the risk of death/complications from an
additional retransplant or the reemergence of dormant cancer cells may be too
great to make it worth the risk.

~~~
2skep
There are other platforms that do that. Take a look at Kiadis which can
identify activated T cells and kill them and then reinfuse rest of white cells
back into the patient.

------
NotSammyHagar
This is an incredible medical advance, the old idea from at least the 70s
actually working. It will take a long time until we can apply this to most
diseases or conditions. There are so many different causes and interactions
behind medical problems, even those that can be attacked by genetics +
specially designed org like this. I'm sad my mom's cancer couldn't be treated
by something like this.

Live long enough, and we'll keep seeing amazing things!

~~~
nonbel
They call this (also CRISPR) gene editing, but interestingly when I looked at
the literature whats going on is always this:

1) Cells are put in a dish

2) Gene "editing" tech is added to the dish

3) Most, if not the vast majority of the cells die. Or this information is
simply not reported, even if the authors did report it in other papers so they
know it is important.

4) The surviving cells consist largely of those with "edited" genes. Control
cells, who were not exposed to the "editing" conditions, also have a small
percentage of "edited" genes.

It sounds to me like they are selectively killing the cells that are not
already mutants. I read around 8 papers on CRISPR, none of them addressed this
possibility (or mention it and cite someone who did address it).

Of course, in terms of getting the desired mutants for the clinic it works
just as well. Misunderstanding why it works is not good though.

------
dang
Also
[https://news.ycombinator.com/item?id=10518432](https://news.ycombinator.com/item?id=10518432).
(Edit: not a duplicate, but an article with additional info.)

------
dnautics
I'm not sure how this is a 'world first'. Gene therapy has from time to time
been used to treat childhood leukemias for a long time, e.g.:

[http://www.uphs.upenn.edu/news/News_Releases/2012/12/tcell/](http://www.uphs.upenn.edu/news/News_Releases/2012/12/tcell/)

