Advancements in Genetic Editing Technology

So this is a precision tool

that now allows us to take this protein RNA complex

and introduce it into cells or tissues to correct mutations

at sites where we know there's a deleterious change in the genetic code.

So I wanted to show you an example

of how this can be utilized.

This protein complex can actually be injected directly

into fertilized eggs of a mouse,

and in the experiment you'll see we're targeting a gene

that is responsible for the black coat color in mice.

And so normally these mice have beautiful, glossy black coats,

and once we make this targeted change,

we then implant these edited eggs back into a female mouse,

and when she gives birth to pups,

you'll see that the pups are now mostly white.

And the remarkable thing about this experiment

is that when these mice grow up,

you can test them and show that every cell

in the body has this single genetic change

that gives rise to the white coat color,

but otherwise they're absolutely normal.

So they are normal mice, no mutations elsewhere in the genome.

This is a type of experiment that

used to take at least a year to create a mouse

like this, and now it can be done in a few weeks

and by people that don't have to have special expertise.

So this is a technology that's been very exciting

over the last two and a half years

to see this technology taking off.

These are publications in the scientific literature.

It's been sort of exponential growth of publications,

people using this technology for all sorts of applications.

And so some of these include making changes,

targeted genetic changes in plants,

in fungi, in animals that are important agriculturally,

in animals that are important to us as pets,

and also in thinking about human health,

also to do things like make changes in stem cells,

which are cells that can give rise to new organs.

Also to make changes in animals that are important as models of human disease,

such as mice and monkeys,

and we think within the not too distant future

it will be possible to actually use this technology

to make changes in humans.

End of transcript · 372 words

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