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Development and Validation of a Quantitative LCMS/MS Method for Measuring CYP4V2 Enzyme Activity in rAAV-hCYP4V2 Gene Therapy Products

2026-03-24

Abstract excerpt

Bietti crystalline dystrophy (BCD) is a hereditary retinal disease caused by loss-of-function mutations in the CYP4V2 gene. Gene replacement therapy using rAAV-hCYP4V2 represents a promising therapeutic strategy, requiring robust bioassays for product quality control. This study developed and validated a sensitive LC-MS/MS method for quantifying CYP4V2 enzyme activity. Lysates from HeLa-AAVR cells transduced with...

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Literature Corpus work
2123e0b2-c3db-568c-aa9f-892e1b111d57
DOI
10.20944/preprints202603.1833.v1
Open publication

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Development and Validation of a Quantitative LCMS/MS Method for Measuring CYP4V2 Enzyme Activity in rAAV-hCYP4V2 Gene Therapy ProductsDOI 10.20944/preprints202603.1833.v1
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