....i would be interested to know what the difference is with AAV approach to delivering drugs.
I’ve recently done some reading around this subject. Here are some of the views I found on viral vector delivery:
Viral vectors and existing lipid vector technology can cause liver damage and activate an adverse immune response in human patients. Viral vectors may cause accidental mutations in host DNA, which can lead to cancer. Patients treated with viral vectors can also develop antibodies against these vectors that make the treatment less effective over time.
Viral vectors have a high affinity for target cells and can be loaded with sufficient genetic material for eliciting a therapeutic effect. However the use of virus particles in therapy has its own limitations such as:
Limited loading capacity
Difficulty in their production
Safety associated with their oncogenic, inflammatory, and immunogenic potential. The latter limits their repetitive administration and use.
Unexpected or undesired genetic expression following their random integration within the host genome.
…drawbacks, such as the bloodstream’s rapid clearance of viral vectors (when injected systemically), their immunogenic and inflammatory potential, together with certain safety concerns, have driven the development of new synthetic gene-delivery vectors.
Viral vectors are particularly good at targeting cells of the retina, so this methodology is the delivery system of choice for gene-editing approaches to inherited retinal diseases. But in practice, viral vectors are difficult to manufacture and hard to scale up for commercial production. We need more science to make them a practical delivery vehicle.
Delivery technologies for Gene disruption therapeutics include antibodies, aptamers, cholesterol, synthetic nanoparticles and, in theory, viral and non-viral vectors. Each delivery method has its advantages and disadvantages, however none are currently sufficient to efficiently delivery therapy to specific tissues and organ systems, with the exception of the liver. Therefore most applications using Gene disruption therapies must rely on local delivery methods…
Examples of viral delivery technologies include retrovirus, recombinant adeno-associated viruses (rAAV), and lentiviruses, which permanently deliver a payload to cells. Each one of these viral technologies has distinct features which make them more applicable for some applications but not others. A major drawback of viral delivery technologies is the complex manufacturing that currently does not support efficient scale up at reasonable costs and at high reproducibility.
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