125459

: Assessing the risk of "insertional mutagenesis"—where the therapy might accidentally interfere with the patient's existing DNA.

: Evaluating whether the viral vector or therapeutic gene could unintentionally be passed on to future generations. 125459

: Characterizing potential immune responses to the viral vector or the new protein produced by the therapy. 125459

: Checking if the patient might "shed" or spread the virus/vector through secretions. 125459

The number most commonly refers to a specific scientific guideline established by the European Medicines Agency (EMA) regarding the development of Gene Therapy Medicinal Products (GTMPs) .

: This guideline is a cornerstone for developers of Advanced Therapy Medicinal Products (ATMPs) in the European Union.