Gene Therapy Approaches

There are many possible ways to target a disease or genetic conditions, so there are difference gene and cell therapy approaches that can be considered.  Common approaches include:

Gene Addition - adds in a working gene that has instructions for the cell to make more of a specific protein. Most commonly VECTORS (see below) are used to deliver the working gene to the cells nucleus.

Gene Editing - corrects pieces of DNA by changing or deleting the information within the affected individual’s genes.

Gene Silencing - where delivered genetic material prevents or inhibits the activity of a gene that is already present in a cell.  This approach is most commonly used to decrease the amount of a specific protein being made.

DNA Therapy - is the use of DNA that codes for the production of specific RNA or protein to treat a disorder. To have the therapeutic effect the DNA must be delivered to the nucleus of a cell, where it can be used by the cell to affect protein expression.

RNA Therapy - is the use of shorter sequences of genetic material in RNA format to treat or prevent disease.

Watch video Gene Therapy Approaches (American Society of Gene and Cell Therapy)