The Progress and Promise of Gene Editing (CRISPR-Cas9) - What's next for the field: epigenetic editing, the 'dark genome

[AussieNeil]

"New technologies such as the CRISPR-Cas9 offer the possibility of altering an individual's genome, or even a generation's genome.

:

Most of the research involved ex vivo experiments -- removing cells, editing them in a lab and then replacing them.

While this process is "relatively easy" for immune cells or blood stem cells, "this isn't possible with most bodily tissues," noted the New Scientist's Michael LePage.

:

Regarding other conditions studied through ex vivo experiments, Porteus said his lab developed a method for correcting mutations of sickle cells in patients' stem cells. If a cure is found, it might take only a few "tweaks" to then find a cure for other illnesses, such as severe combined immunodeficiency, he noted."

(My emphasis)

Full article in Medpage Today: medpagetoday.com/genetics/g...

Neil

[Hoffy]

Very promising..

[SeymourB]

It looks like scientists will need an alternative to Cas9. The problem? Cas9 comes from bacteria, and many people have antibodies to it:

​​biorxiv.org/content/early/2...

I'm surprised this wasn't tested earlier. I'm thinking they'll need to make an artificial equivalent, but I don't know enough about the molecular level chemistry do speculate more. People who know even less are selling CRISPR related stocks.

=seymour=

[Jm954 Administrator in reply to SeymourB]

Coincidentally another report about potential resistance in my inbox this morning

statnews.com/2018/01/08/imm...

[cllady01 Former Volunteer]

Research is the basis of breakthroughs. From our perspective as patients who want something now, it is difficult to discern if dogged continuation in what keeps having "road blocks" is good news or bad news.

Without the continual re-routing/re-visioning a research team does after a find that tells them they are on the wrong tack or need a tweak that will set their mission back for who knows what amount of time, there would be no breakthroughs of the type that at least give a remission.

They must have days of feeling they are beating a dead horse, and then have to take a deep breath and go back to the task, no matter how minor the finding, there is success in failure if the process of seeking continues.