Therapeutic Digest

The Age of Gene Therapy

January 15, 2020

Gene therapy is based on two basic strategies: an integrating vector that is introduced into a stem cell so that the gene integrates at one or more places in the patient’s chromosomes, also known as ex vivo; and gene delivery into a nonintegrating vector to a long-lived postmitotic or slowly dividing cell, which ensures the expression of that gene for the life of that cell, also known as in vivo. Currently, more than 800 cell and gene therapies are in development for more than 100 diseases. This Therapeutic Digest delves into the age of gene therapy and the promising wave of life-changing and life-saving medicines.

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