How can CRISPR gene therapy impact medicine?
Several clinical conditions responsible for leading causes of morbidity and mortality can be impacted using CRISPR applications. HIV infection lead to AIDS, and the only existing strategy geared towards managing this progression was anti-retroviral therapy. However, CRISPR has been found to be effective at excising the HIV genome from T-cells isolated from patients.1 Primary in vivo studies in HIV-infected rodents demonstrated remarkable efficacy in reducing HIV infection and reversing AIDS progression. Similarly, other incurable conditions, including Duchenne muscular dystrophy and Type I diabetes, all have similar potential to be impacted by effective genetic engineering platforms like CRISPR.
Reference:
1. R. Kaminski, et al., "Elimination of HIV-1 Genomes from Human T-lymphoid Cells by CRISPR/Cas9 Gene Editing," Sci Rep 6(22555), 2016.
Reference:
1. R. Kaminski, et al., "Elimination of HIV-1 Genomes from Human T-lymphoid Cells by CRISPR/Cas9 Gene Editing," Sci Rep 6(22555), 2016.