Which technique and which vector are used to combat Parkinson’s through gene technology?

Answer

Dear Sona,

In genetic engineering, lentiviruses or adeno-associated viruses (AAV) are often used. AAV viruses are most often used in clinical applications involving the central nervous system. To put it simply, gene therapy creates a virus that has a piece of RNA that codes for a specific protein. This viral RNA will then integrate into the DNA of the patient’s cells, after which the cell will be able to produce this protein itself.

Gene therapy is a very new clinical technology and is still in its infancy. A well-known example of gene therapy is Zolgensma, which is used for SMA. For Parkinson’s, a number of therapies are in the clinical trial phase, but to date no commonly used therapy is available.

Best regards,

Ann