The use of CRISPR-Cas technology

CRISPR-Cas is a relatively new technology that allows easy editing of DNA in a very precise and effective way. Within regenerative medicine, this gene correction tool can be used to repair cells that contain a disease-causing mutation or to provide extra functions to cells.

What is CRISPR-Cas and what can it do

Modifying the genetic code has long been a very difficult and time-consuming process. This all changed with the introduction of the CRISPR-Cas system. The technique revolutionized research in the (bio-) medical field, as it allows parts of the genetic code to be easily changed, added or deleted.The CRISPR-Cas gene editing system consists of a specific “guide” that shows a small molecular “scissor” precisely where to make a cut in the DNA. This cut permits introduction of new pieces of DNA in that particular spot. Every cell - human, animal, plant, bacteria - has DNA in their nucleus which contain the genetic instructions for functioning, growth, development, and reproduction. The CRISPR-Cas technology can be used to repair a genetic defect in the cell of a person suffering from a hereditary disorder. This can be done by providing the patient with new cells which have been corrected in the laboratory, or by directly providing the CRISPR-Cas system for in vivo gene editing. Modifying the genetic code of human beings brings along ethical issues that need careful consideration. It is essential that the technique is only used for ethically approved purposes.


State-of the-art and considerations

CRISPR-Cas technology is being extensively perfected. New Cas molecules are identified and tested for more accurate DNA targeting and less side effects. CRISPR-Cas is also used to develop disease models. In a laboratory setting, the genetic code of cells and organisms’ is altered allowing the study of disease development and the effect of drugs on these diseases. For medical purposes, a patient’s own cells can be used to gene correct a disease-causing mutation. At LUMC this is studied, for instance, with cells from patients suffering from hereditary Duchenne muscular dystrophy. Other uses of CRISPR-Cas technology are the addition of certain functions to a cell. One example is counteracting rejection of transplanted cells by adding and removing genes that makes it become invisible to the immune system

Modifying the genetic code of human beings bears ethical issues that need careful consideration. It is essential that the technique is only used for ethically approved purposes. In addition, existing policy and patent issues impact the applicability of this technology as a therapy.


This is what we aim to accomplish in the future

Combining iPSC technology {insert link} and CRISPR-Cas technology holds great promise for regenerative medicine. Skin cells of a patient with a certain genetic disorder can be turned into iPSC, gene-corrected with CRISPR-Cas, and differentiated into any cell type that needs repair to function again for a normal healthy life.