P.J. Brooks, Ph.D., 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: ex vivo and in vivo.
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 altering them, and then transplanting them back into the person. Most ex vivo gene therapy uses hematopoietic stem cells (HSCs) and is used to treat blood and immunological diseases.
In vivo gene therapy often employ adeno-associated virus (AAV) gene therapies. These use a modified version of AAV to deliver a working copy of a defective gene into the relevant cells for a given disease. This can include 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. This could help to save both time and money during the final clinical stages.
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