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PCT 124

Question
Does PCT 124 help in case of cftr dele 2,3?
Answer
Dear questioner,

PCT 124 is a medicament whose efficacy is the following: repair the implications of a certain class of mutations (so called nonsense mutations, respectively, class 1 mutations). The mutation CFTRdele2,3 (21kb) does not belong to this mutation class, so that PCT 124 is probably ineffective in this case.

Best regards,
Prof. Stuhrmann-Spangenberg
24.02.2009