Human immunodeficiency virus (HIV) is a type of retrovirus, that if left untreated can lead to AIDs (acquired immunodeficiency syndrome).
CRISPR-Edited Stem Cells in a Patient with HIV and Acute Lymphocytic Leukemia
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.