2 ms·
> The genetic instructions for making the antibody were packaged up inside a harmless virus, which was then used to infect the noses of mice. That's interestin
by darkerside 8y ago
> The genetic instructions for making the antibody were packaged up inside a harmless virus, which was then used to infect the noses of mice.
That's interesting. I didn't realize gene therapy could be so localized.
- Nasrudith 8y agoWell every copy of the DNA doesn't make it into the germ-line and viruses generally infect differing types of cells in the first place.
- rubatuga 8y agoYep, they usually use a pseudo virus that doesn't replicate, called an adeno associated virus. Check out this guys video on his own gene therapy for lactose intolerance : https://m.youtube.com/watch?v=J3FcbFqSoQY https://m.youtube.com/watch?v=J3FcbFqSoQY
- rotexo 8y agoYep, adeno-associated virus (AAV) vectors seem very promising. Depending on the type of vector used, you can target the eye, joints, liver, lungs, etc. [1]. Relatively recently, the FDA approved the first AAV-based therapy for a rare retinal disorder [2]. Also worth checking out are some promising trials for treatment of severe hemophilia using AAV vectors [3]. These might end up being great, because the current gold standard treatment involves giving people regular injections of recombinant clotting factor protein, which is even more expensive than gene therapy. Plus, with gene therapy, you have one treatment that lasts a long time, versus having to get an infusion of the treatment regularly. [1] (https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5138125/ https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5138125/) [2] (https://www.labroots.com/trending/drug-discovery/7533/adeno-associated-virus-gene-therapy-fda-approved-rare-retinal-disease https://www.labroots.com/trending/drug-discovery/7533/adeno-...) [3] https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5728634/ https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5728634/