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Testing preexisting antibodies prior to AAV gene transfer therapy: rationale, lessons and future considerations

Medication options : For constipation, over-the-counter polyethylene glycol may help

Its proposed mechanism is interference with assembly of the SNARE complex necessary for synaptic exocytosis, particularly at the neuromuscular junction

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These challenges include degradation by cellular nucleases, high renal clearance, and low penetration in biological membranes due to their negative charge