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[ 1 , 2 ] Since its theoretical inception in the 1972s by Friedmann & Roblin, [ 3 ] gene therapy has evolved significantly, with early clinical trials conducted in the 1990s and major significant breakthroughs occurring throughout the 21st century
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Given the population allelic frequencies of common mutations, the incidence of CUD in Hong Kong is estimated to be ~1 in 60,000.26 This estimate matches with early results from our NBS data published in this issue and reported recently.27,28 However, NBS is not 100% sensitive
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