Would it ever be possible to repair the genes of someone who has a genetic defect, but is already an adult?

[Elite Engineer]

It pains me to see people suffering from debilitating genetic diseases, where (if their lucky) the only treatment is to mildly reduce the symptoms. 

Maybe not now or even in 20 years, but is there any possibility that an individuals genetic errors could be fixed? I really can't think of any mechanism

that could do this, even in a far-reaching theory.

 

Your thoughts?

~ee

[StringJunky]

You might find this Science Mag article interesting: https://www.sciencemag.org/news/2016/05/gene-editor-crispr-won-t-fully-fix-sick-people-anytime-soon-here-s-why

[Elite Engineer]

Thank you!

[Itoero]

11 hours ago, Elite Engineer said:

It pains me to see people suffering from debilitating genetic diseases, where (if their lucky) the only treatment is to mildly reduce the symptoms. 

Maybe not now or even in 20 years, but is there any possibility that an individuals genetic errors could be fixed? I really can't think of any mechanism

that could do this, even in a far-reaching theory.

 

Your thoughts?

~ee

The antisense technique is a way of blocking/silencing a particular gene which is known to be causative of a particular disease, by adding correct nucleotides which bind with mRNA to prevent mRNA from  binding with a ribosome. There are many  therapies being developed to treat certain diseases. There is a trial going on to test the AON(Antisense OligoNucleotide)  technique on people with Huntington. It's a very promising therapy but it doesn't cure a disease, it prevents diseases from manifesting or it can slow or block the progress. https://en.wikipedia.org/wiki/Antisense_therapy#Cancer

CIRPSR is also a very promising technique but it has to develop/evolve a lot to be safe and effective. For more information about the state of basic research and the potential medical applications of CRISPR-Cas9 technology: http://innovativegenomics.org/

Edited July 29, 2017 by Itoero

[Elite Engineer]

On 7/29/2017 at 9:43 AM, Itoero said:

The antisense technique is a way of blocking/silencing a particular gene which is known to be causative of a particular disease, by adding correct nucleotides which bind with mRNA to prevent mRNA from  binding with a ribosome. There are many  therapies being developed to treat certain diseases. There is a trial going on to test the AON(Antisense OligoNucleotide)  technique on people with Huntington. It's a very promising therapy but it doesn't cure a disease, it prevents diseases from manifesting or it can slow or block the progress. https://en.wikipedia.org/wiki/Antisense_therapy#Cancer

CIRPSR is also a very promising technique but it has to develop/evolve a lot to be safe and effective. For more information about the state of basic research and the potential medical applications of CRISPR-Cas9 technology: http://innovativegenomics.org/

But is this done while the individual is still an embryo? What about grown people who already have the the full genetic disease?

[Itoero]

16 hours ago, Elite Engineer said:

But is this done while the individual is still an embryo? What about grown people who already have the the full genetic disease?

The antisense technique is applicable on grown humans. I suppose they intravenous inject nucleotides that bind to mRNA to silence 'bad' genes or alleles. If via DNA analysis you know you will get a genetic disease then with the antisense technique, you can prevent a disease from manifesting. When you already have the genetic disease then you can slow down or block the progress with an antisense therapy.

The CIRPSR  method changes the DNA so I think it's ' future music' before this is applicable on fully grown humans. The method is atm in development to treat embryo's. https://www.newscientist.com/article/2123973-first-results-of-crispr-gene-editing-of-normal-embryos-released/