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and a homozygous pathogenic mutation (4 points)

Total protein was extracted with a combination of RIPA buffer (Sigma Aldrich, US) and a protease inhibitor cocktail (Roche, Shanghai, CN) per the manufacturers instructions

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Australias TGA Medical Devices The Therapeutic Goods Administration (TGA) is the unit of the Australian governments Department of Health and Aging that oversees medical device regulation in Australia