Recombinant AAV (rAAV) vectors are a key component of an emergent therapeutic paradigm with a demonstrated definitive benefit for genetic diseases. Immune responses are among the most challenging barriers to human gene therapy. Viral vectors are highly advantageous because of their evolved ability to negotiate complex intracellular pathways to efficiently deliver a DNA payload; however, the human immune system has evolved multiple pathways, including innate pathogen-associated molecular pattern (PAMP) sensors that trigger adaptive effector functions to eliminate infected cells.
No takes yet. Share an insight, caveat, or question.
J. Fraser Wright (2020) studied this question.
Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context: