Says capsid is shed inside the nucleus but video shows the capsid docked at a nuclear pore complex and "injecting" its genome into the nucleoplasm. It's also a double stranded molecule but the dsDNA synthesis occurs during / after capsid disassembly using the single stranded DNA viral genome (though, it's possible to package AAV with dsDNA).
This video acts like non-integration into the host genome is a benefit. But if you're trying to correct a faulty gene, why WOULDN'T you want the theraputic gene integrated into the host genome? Without that you're only giving someone a temporary treatment. Good for revenue maybe, but bad for the patient.
Because otherwise how is this a long term solution to a disease? How can you ensure "stable and long term expression" of the targeted protein if the episomes aren't passed on when the cell divides? Would it no only stay in cells non-dividing cells? Either this would require routine follow up therapies or there is some mechanism not explained in the video that causes long term expression of the proteins? Could still be useful but the video states "stable and long term expression" of the molecule@@mol_biologist30
it's because AAV are small vector with a packaging capacity of 5kb and the problem is the immune system gradually neutralize AAV with antibodies. however, the good thing is that even though they are not permanent, AAV have a long-term expression of the target gene.
in some cases we are inducing for example pancreatic acinar cells into becoming beta cells, and in that case we wouldn't want every single pancreatic acinar cell to transcribe those genes and become beta cells now wouldn't we?
In the case of permanent or stable correction, another vector *LENTIVIRUS* or retroviruses can be used as they are capable of integrating into the host genome. However, human genome integration (patterns, sites and its effects) is still not yet fully understood... Therefore it is safe to first go easy "temporary" until some crucial questions are answered satisfactorily.
