Mouse Study Alerts Potential Gene Therapy Issues in Female Patients

Mouse Study Alerts Potential Gene Therapy Issues in Female Patients

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Mouse Study Alerts Potential Gene Therapy Issues in Female Patients

A gene therapy-like approach that provided a full-length MECP2 — the gene mutated in most cases of Rett syndrome — to cells safely and effectively lessened disease-specific symptoms in a mouse model of a milder form of Rett, a study shows. However, female mice treated with this approach showed signs of motor-related adverse events associated with excessive, toxic levels of MeCP2, the protein coded by the MECP2 gene and missing or defective in Rett patients.…

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