再生医学ジャーナル

Editorial Note on Gene therapy

Mounica Merihelan

Gene therapy is a Medical Experimental technique uses therapeutic genes to treat or to prevent diseases. Most of the inherited diseases and genetic diseases caused by the mutation deletion or insertion of the base pairs onto the genetic material causing Misfolding or absence of protein synthesis in translation. The missing or incorrect gene is replaced by the New synthetic gene and introduce intothe cell as a Functional substitute. Biological vectors like Viruses have been using to transport or to deliver the nucleic acids or genes into the patient’s cells as a drug. AAV (adeno-associated virus), lentivirus is used for performing gene insertions In vivo and Ex vivo