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In 2019, the Abramson Cancer Center of the University of Pennsylvania in US announced the use of the CRISPR technology to edit cancer genes in humans, [163] and the results of the phase I clinical trial in 2020. [164] The study started in 2018 with an official registration in the US clinical trials registry. [165]
CRISPR has also been used to cure malaria in mosquitos, which could eliminate the vector and the disease in humans. [171] CRISPR may also have applications in tissue engineering and regenerative medicine, such as by creating human blood vessels that lack expression of MHC class II proteins, which often cause transplant rejection. [172]
Targeted gene knockout using CRISPR/Cas9 requires the use of a delivery system to introduce the sgRNA and Cas9 into the cell. Although a number of different delivery systems are potentially available for CRISPR, [37] [38] genome-wide loss-of-function screens are predominantly carried out using third generation lentiviral vectors.
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CRISPR technology is a promising tool not only for genetic disease corrections but also for the prevention of viral and bacterial infections. Utilizing CRISPR–Cas therapies, researchers have targeted viral infections like HSV-1, EBV, HIV-1, HBV, HPV, and HCV, with ongoing clinical trials for an HIV-clearing strategy named EBT-101 ...
Cas9 (or "CRISPR-associated protein 9") is an enzyme that uses CRISPR sequences as a guide to recognize and open up specific strands of DNA that are complementary to the CRISPR sequence. Cas9 enzymes together with CRISPR sequences form the basis of a technology known as CRISPR-Cas9 that can be used to edit genes within living organisms.