Researchers hope to develop treatments for a wide range of genetic disorders, and even cancer, using CRISPR-Cas9 gene editing. These clinical interventions may take the form of ex vivo therapy, in which cells are edited in the lab and transfused into patients, or in vivo therapy, which delivers gene-editing machinery directly to the affected tissues.
Approach: Extract cells from the blood, modify them using CRISPR, expand the edited cells, and infuse them back into the body. Alternatively, modified cells from a healthy donor could be expanded and infused into multiple patients.
Examples: Several research groups and companies are developing therapies that modify autologous hematopoietic stem cells to treat sickle cell disease and/or β-thalassemia, with trials beginning as early as this year. Trials are in progress to modify autologous or donor T cells with customized receptors to better fight cancer.
Hurdles: Regulators have little experience with customized, cell-based therapies, and the ...
