3 ms·

Question for the Bio people, from what I've seen gene therapies so far target very narrow scope sometimes rare diseases. Is there any 'broad spectrum' type gene

Question for the Bio people, from what I've seen gene therapies so far target very narrow scope sometimes rare diseases. Is there any 'broad spectrum' type gene therapy currently in the pipeline of any company out there? (not specific to audiology) And is the narrow scope because it is easy to control in these early days or is that we simple don't know enough to make more complex gene therapeutics without understanding collateral damage, side-effects, for example?

kozinc 3y ago
Being a layman too, as I understand it, it's because the deafness is caused by a single (mutated) gene which makes it (comparatively) easy to change. In this case, because the altered gene causes a necessary protein to be faulty or missing, gene transfer therapy can introduce a normal copy of the gene to recover the function of the protein. For other diseases caused like this you're mostly right on the money - sometimes because while a single gene might be the culprit, the mechanism is unknown, sometimes mutations in some ‘single-gene disorders’ may not be in a single-gene at all, not to even mention other possible interactions or the risks inherent to gene therapy.
salubrioustoxin 3y ago
Yes. Also, even within rare disease this application has desirable features. Obvious bio marker (hearing), relatively isolated organ system with obvious delivery method (inject into cochlea). main downside is that there is an existing alternative (implant) so risk/benefit question is less obvious compared to eg neurodegenerative diseases.
- DoingIsLearning 3y ago
  Is delivery critical or just get it close enough to tissue in ROI? Are we aiming to modify specific cells/layers? And what happens to adjacent tissue that is 'accidentally' modified? Do you have any literature recommendations on the state of the art in Gene Therapy 'delivery' mechanisms/apparatus?
huytersd 3y ago
EBT-101 is an experimental procedure where they try to use viruses to excise the HIV retrovirus DNA from every cell in your body.

kozinc 3y ago

Being a layman too, as I understand it, it's because the deafness is caused by a single (mutated) gene which makes it (comparatively) easy to change. In this case, because the altered gene causes a necessary protein to be faulty or missing, gene transfer therapy can introduce a normal copy of the gene to recover the function of the protein. For other diseases caused like this you're mostly right on the money - sometimes because while a single gene might be the culprit, the mechanism is unknown, sometimes mutations in some ‘single-gene disorders’ may not be in a single-gene at all, not to even mention other possible interactions or the risks inherent to gene therapy.

salubrioustoxin 3y ago

Yes. Also, even within rare disease this application has desirable features. Obvious bio marker (hearing), relatively isolated organ system with obvious delivery method (inject into cochlea). main downside is that there is an existing alternative (implant) so risk/benefit question is less obvious compared to eg neurodegenerative diseases.

DoingIsLearning 3y ago
Is delivery critical or just get it close enough to tissue in ROI? Are we aiming to modify specific cells/layers? And what happens to adjacent tissue that is 'accidentally' modified? Do you have any literature recommendations on the state of the art in Gene Therapy 'delivery' mechanisms/apparatus?

huytersd 3y ago

EBT-101 is an experimental procedure where they try to use viruses to excise the HIV retrovirus DNA from every cell in your body.