Ex vivo vs In Vivo Gene Therapy



P.J. Brooks, PhD, Acting Director of the Office of Rare Diseases Research at the National Center for Advancing Translational Sciences (NCATS), describes the two main types of gene therapy currently in development.

Gene therapy works best on monogenic disorders, which account for the majority of rare diseases.  As Dr. Brooks explains, there are two general types of gene therapies – ex vivo therapy and in vivo gene therapy. 

Ex vivo gene therapy usually involves the removal of specific cells from a person, genetically alter them, and then transplant them back into the person. Most ex vivo gene therapy use hematopoietic stem cells (HSCs) and used to treat blood and immunological diseases.

In vivo gene therapy often employ adeno-associated virus (AAV) gene therapies that use a modified version of AAV to deliver a working copy of a defective gene into the relevant cells for a given disease, including cells in the eye, liver, brain, muscle and other organs.  Numerous programs are underway at NCATS to develop more efficient methods to develop and deliver gene therapies for rare diseases, including the Bespoke Gene Therapy Consortium (BGTC).

In theory, the testing of new AAV gene therapies for safety and efficacy could be streamlined during preclinical investigations to save both time and money during the final clinical stages. 

To stay up to date with the latest NCATS and RDCRN news, visit checkrare.com/rdcdrn