Researchers used the CRISPR-Cas9 complex to correct a mutation in the hematopoietic stem cells caused by an immunodeficient disorder. The so edited cells managed to coordinate in mice body for up to five months without any glitch.
CRISPR-Cas9 combination is one the most promising gene editing tool of this century. Scientists believe that with this slicer machine, biotechnology is closing gap between genetic disorder and an ultimate solution. A recent advancement might be a great example to fasten the claim about this prestigious gene editor. A group of researchers from American Association for the Advancement of Science used CRISPR-Cas9 complex to edit a mutation in the gene which leads to a lethal immunodeficiency disorder. However, the success of such an editing couldn’t be confirmed without testing on a live model, which they successfully achieved by grafting the cells on test mice for a period of about 5 months. With this new feat, more and more genetic disorders shall be fixed using the similar approach after subtle research and optimization.
Chronic Granulomatous Disease (CGD) is an inherited immunodeficiency disorder which results in increased susceptibility to an infection by bacteria or fungi. Till the date there is no complete cure being developed for CGD, however, stem cell transplantation and long-term antibiotic administration are the currently practiced measures to keep the disease under control. CGD is caused a mutation in the CYBB gene which results in an abnormal protein called as NOX2 or NADPH oxidase 2, which in fact play a crucial role in phagocytosis (destruction of intruders.) In the clinical approach, scientists used the CRISPR-Cas9 mechanism to edit the mutation ex-vivo thereby ensuring that the protein so expressed has restored their ability to actively participate in the immune pathway. In order to ensure its viability in living cells, they engrafted the cells on live mice and observed long-term preservation of the edit without any side effects.
Though the study might sound astounding there are many glitches that need to be fixed before turning it into human machinery. One baffling challenge is the inability to use CRISPR-Cas9 in gene editing without introducing the additional mutation. The selective ability of modification is also one headache with this approach. However many studies are progressing to eliminate these disadvantages to make the CRISPR-Cas9 editor a foolproof tool. The potential of this approach holds many advantages, its versatility and ability to use universally, make it a state of the art gene editing mechanism. Even HIV and Cancer cure is one major scope under this tool and researches are ongoing in many parts of the world.
Source: DOI: 10.1126/scitranslmed.aah3480