gene editing Jak2

[Steve_Essex]

Hi,

just came across this article, apologies if previously been shared…

it outlines research at Stanford to use gene editing techniques to identify, cut out and replace mutant Jak 2 genes

techfinder.stanford.edu/tec....

[EPguy]

This is a big deal. It provides the "mutant sequence can be specifically targeted ". This is relatively easier in CALR for potential curative therapy, but has been the key missing step in Jak2. A long way before MPNs can use it but good news.

It's a 70 page patent which published just recently. This means they've been at it for years but were not free to tell anyone till the patent published.

worldwide.espacenet.com/pat...

It's all about exotic uses of RNA. It relies on SCT so it's not a magic shot, but it's autologous meaning the patient's own marrow. So rejection/immune suppression should not be a concern.

[Roxanne22 in reply to EPguy]

Hello, what is SCT pls? Thanks EPGuy - it certainly brings hope to many of us.

[EPguy in reply to Roxanne22]

SCT is marrow transplant.

[Roxanne22 in reply to EPguy]

oh so would rely on removing area of diseased marrow and replacing with our own healthy marrow, along those lines? (sorry for my ignorance, I need an 'idiot's guide' as I am no medic.

[Steve_Essex in reply to EPguy]

be interesting to see the published outputs of the invivo research.. which looks like is already well underway… however I’m sure it’ll still take quite a bit of time to get over the hurdles to bring into clinical trial… but I had no idea research had progressed as far as being able to specifically target the Jak2 mutant genes…

[EPguy in reply to Steve_Essex]

In the patent summary they are missing IFN as an option, see text here, surprising. If SCT is required, this will remain 2nd line therapy. For pts responding well to IFN, Rux, esp with reduced allele, I don't expect Drs will order this solution since any SCT has signif risks. Even those with good counts on HU may not qualify.

In general this will probably remain 2nd line unless/until they can find a way without SCT. The patent does mention other ways, (IV, etc) but does not offer details.

I think the coming CALR immune therapies don't need SCT.

sum

[Maisie10]

thank you for sharing .

[Fox756]

Oh my gosh, I hadn't heard about this. Thank you for sharing.

[SoledadBarcelona]

Thanks. Very interesting. This way has some problems to all king of cancer. Waiting more time to see what is there for us. Regards

[leefsu97]

what an exciting article! Thank you for sharing this is so hopeful

[Roxanne22]

Thanks Steve_Essex, this fills me with hope for myself and others with PV, can you imagine how joyous this would be to find a 'cure'.

It's really encouraging to see how the research is moving on in this and other applications to give us more palatable solutions to this complex condition. sending love and optimum health to us all!

[Bindi77]

Thank you for sharing! Love hearing the hopeful news

[ainslie]

interesting info, well found 👍

[Jazzman7]

many thanks. Ad not seen this.

[KLCTJC]

Thank you for sharing. It is amazing to see how much medicine has changed and the change in focus. I have been practicing for 15 years now and in the last 3-5, I have seen so much progress on targeting diseases at their specific molecular level. Autoimmune and cancer specifically. I have lots of faith for all of us. Everyone is in my prayers😊

[EPguy in reply to KLCTJC]

That's two of us on the MPN + autoimmune magic hopes.

[shiftzz]

My jak2 mutation is down to about 2%, effectively 'cured' but I still have PV symptoms. Is it possible that JAK 2 is not wholly responsible for PV.

[EPguy in reply to shiftzz]

I think you're right. Also some criteria for min residual disease have VAF well under 1%. In the Bes trials "undetectable" was about <0.01%. Maybe that would fix the symptoms. But at <2% you're on the high probability path to at least not have a later increase.

[russkatt in reply to shiftzz]

That is a great question. I have often wondered the same t

[EPguy in reply to russkatt]

In studies by the Dr. Silver group they found that lowest alleles did not necessarily fix the marrow condition. That was one argument they made for not ceasing IFN even if VAF was very low. So it could be this lingering aspect affects symptoms.

[Nrl303]

As someone who is in their mid 20’s, I’m hopeful there will be something that can correct this! 🙏🏻

[saltmarsh]

Many thanks for this post.

[mhos61]

Great you found this. It’s much appreciated.

[mother2britton]

Great post thank you!

[KyleeR]

This is so very exciting .. thank you for sharing

[Mirabellegage]

all very interesting, thanks for sharing! I’m sure I speak for us all that we’re all hoping that the speed of medical advances outpaces the progression of our disease.

[Iwillwin123]

This is interesting. And useful to follow how and when , it reaches implementation as treatment.