However, it is important to note that NDD therapeutics may face more obstacles in clinic than the retinal delivery of Luxturna as the brain is less accessible than the eye and requires the delivery vector to cross the blood-brain barrier (BBB)
For the 3.2 mg dose used here, 2.5 mL of BAC water in an 80 mg vial produces 25 draws of 0.1 mL, or 10 units
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The chemiluminescent readout was obtained using SuperSignal West Femto maximum sensitive substrate (Thermo Scientific GmbH, Schwerte, Germany) with a c400 Azure imaging system (lowest sensitivity, 10 s exposure time)