The hottest gene therapy can be CRISPR (clustered regularly interspaced short palindromic repeats), a technology that can spot the mutated gene and correct their sequence into a normal state. It's one of the most promising technologies for cancer treatment, and it seems that the tech can be applied in numerous diseases for it aims at the origin of mutations. However, as the often case, few drawbacks that it needs to be improved are less mentioned than their potential in curing diseases, especially for the ones that are inherited from the last generation.
What CRISPR has been achieved?
Different from the traditional gene therapy, CRISPR correct the gene sequence rather than substitute them with a new piece of gene. In theory, CRISPR would be much better in side effects as gene replacement may carry the risk of installing at wrong places. Besides, another great potential of CRISPR is editing the genes of human embryo, making people free from inherited diseases. It's claimed by related scientists that CRISPR is the easiest way to restore genes.
Gene editing and restoring is just one aspect that CRISPR can be taken advantage of. The advanced tech can be also applied to establishing diseases research model instead of using animals to study what will happen when genes in them are changed. Biomedicine is another aspect the tech can be made use of, and tissue-based treatments for cancer and other diseases is under research.
Besides, CRISPR is also used as a tool to probe other matters, like cells living mechanism. They study how to manipulate under the help of gene-editing tech, and then apply the knowledge to cultivating cells for drug screening and test.
With the research concentration on CRISPR continuously increasing in recent years, it has been one of the most attractive topics for both scientific practitioners and average people. In the upcoming annual meeting of the American Society of Gene and Cell Therapy, there will be 93 abstracts on CRISPR discussed and the number of that for last year was only 33, from which it can be seen that how hot the tech is. They hold magic ability in giving hope to people who are desperate to the available therapy.
How about the present situation of the tech? Will the prime time for it come?
Though the tech has made some breakthrough in the modern medical history, it's far away to get into a mature state. Most involved treatments are still in a research stage, and it's still unknown for them to be effective or not to be used on humans. That's the general situation. To be specific, there are several hindrances the tech is faced with and waiting to be addressed for further development.
First, the Cas9 enzyme that CRISPR uses to cut genes is at the danger to cut off target, and such wrong spot may result in cancer directly. Second, for most diseases, the sequence-editing process are required to handle on patients to ensure higher survival level of the cells rather than operate out of bodies and in labs.