Human immunodeficiency virus (HIV) is a type of retrovirus, that if left untreated can lead to AIDs (acquired immunodeficiency syndrome).
Researchers based in China used CRISPR to edit hematopoietic stem and progenitor cells (HSPCs) to make the gene CCR5 non-functional, which makes cells resistant to HIV-1. The CRISPR-edited CCR5-ablated HSPCs were transplanted into a patient with HIV-1 infection and acute lymphoblastic leukemia, which was in remission. Although there was long-term engraftment of the edited HSPCs, only about 5% of lymphocytes contained the CCR5 disruption.
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- CRISPRClustered regularly interspaced short palindromic repeats (CRISPR) is a prokaryotic adaptive immune response that provides immunity against foreign nucleic acids, such as viral DNA and bacterial plasmids, through the use of crRNAs (CRISPR RNAs) and associated Cas genes.
- CRISPR/Cas ToolsClustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR associated (Cas) proteins perform adaptive immune functions in prokaryotic organisms defending against foreign nucleic acids such as viruses. CRISPR/Cas tools have been adapted for use in genome editing and other DNA and RNA targeting applications.