Current advances regarding CRISPR/Cas and its potential as a gene therapy for monogenic diseases
MetadataShow full item record
Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and the CRISPR-associated system (Cas) have been discovered around a decade ago, however since then it has slowly been developed into a gene editing tool. The improvements that have been made to the CRISPR/Cas system, allow for it to be used in both cells in a culture or directly in the body. One type of diseases that can be especially helped by further development of the CRISPR/Cas system, are monogenic diseases. These types of diseases are caused by a mutation in only one gene. Often, due to this mutation the function of the gene is lost as it is partly, or even completely missing. The fact that only one gene is affected, makes it a lot easier for the CRISPR/Cas system to fix. Recently, the number of CRISPR/Cas techniques that are available have increased and thus a growing number of mutations can be fixed. Besides the repair of a (partly) missing gene or the removal of a gene, now the editing of specific bases is, amongst others, also possible. Moreover, in this review the different techniques that have recently been developed for use in cultured cells and directly into animal models of monogenic diseases are discussed. From these studies, it can be seen that repair of mutated genes is often possible via CRISPR/Cas techniques. Moreover, some studies have even made it onto the clinical trial phases and are now being tested in humans. Preliminary results from these trials show great promise. Furthermore, we discuss the potential ways in which the discovered CRISPR/Cas techniques can be used for the treatment of monogenic kidney diseases, as currently no curative treatments are available and often a kidney transplant is needed. However, since the fact that repair of only one copy of the gene could already lead to disappearance of disease symptoms and thus present a clinical benefit for these patients, in theory, the success rate of gene repair in these monogenic kidney diseases could be high. Especially with the information from current studies, a lot of possibilities are available, which are briefly discussed in two examples. However, with use of the CRISPR/Cas system, also some limitations arise, such as how to get the system into the body and possible side-effects that can come along with this. Nevertheless, there are also options available which can overcome these limitations and improve the technique. When all this information is taken together, it is obvious that CRISPR/Cas is a powerful tool for gene editing. Yet, it is also obvious that the technique still needs to be improved and adapted before it can be, safely, used in humans. However, overall, CRISPR/Cas proves to be a realistic therapy for the treatment of monogenic diseases and could therefore benefit this group of patients.