The CRISPR-Cas9 system has proved to be one of the most powerful gene-editing tools that have massive therapeutic potentials on inherited hematologic disorders. Such disorders as the β-thalassemia, sickle cell disease, hemophilia A and B, and Fanconi anemia are majorly monogenic, and therefore, the best targets of genetic correction per se. This paper only has concentrations on pre-clinical testing on animals in order to analyse the usage, effectiveness, and safety of CRISPR-Cas9 therapies. Data translated in mice, dog, and non-human primate models showed great edit efficiencies (between 42% and 65%) that were associated with improvements of hematologic functions, development of haemoglobin and replenishment of clotting factors. Requiring minimal to moderate off-target effects were both found to be dependent upon delivery systems and genetic targets. The research demonstrates the feasibility of the CRISPR-Cas9 to treat disease-causing mutations in vivo and the need of animal studies with long-term follow-ups to go further towards clinic applications. Results emphasize the worth of animal model in filling the gap between laboratory study and human gene therapy on blood conditions.
Chakravarthi et al. (Mon,) studied this question.
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