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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 ...
In addition, it has been used to engineer stably modified human embryonic stem cell and induced pluripotent stem cell (IPSCs) clones and human erythroid cell lines, [11] [28] to generate knockout C. elegans, [12] knockout rats, [13] knockout mice, [29] and knockout zebrafish. [14] [30] Moreover, the method can be used to generate knockin organisms.
The first injection of the CRISPR-Cas System was confirmed in March 2020. [267] Exagamglogene autotemcel, a CRISPR-based human gene editing therapy, was used for sickle cell and thalassemia in clinical trials. [268]
[12] [13] Animal studies began in March 2020, and recruitment of 510 human participants for a phase I/II trial began on 27 March, [17] [18] [19] and the results were presented in October. [20] On 30 December 2020, the vaccine was approved for use [21] in the UK's vaccination programme.
CRISPR [43] is the leading genetic engineering method. [44] In 2014, Esvelt and coworkers first suggested that CRISPR/Cas9 might be used to build gene drives. [5] In 2015, researchers reported successful engineering of CRISPR-based gene drives in Saccharomyces [45], Drosophila, [46] and mosquitoes.
Live recombinant vaccines can be administered via orally or nasally, instead of injection. Common examples of vaccines with the aforementioned route of admission include the oral polio vaccine and the nasal spray influenza vaccine. [3] [4] These vaccines can stimulate mucosal immunity and eliminate adverse effects associated with injection. [5]
CRISPR-associated transposons or CASTs are mobile genetic elements that have evolved to make use of minimal CRISPR systems for RNA-guided transposition of their DNA. [1] Unlike traditional CRISPR systems that contain interference mechanisms to degrade targeted DNA, CASTs lack proteins and/or protein domains responsible for DNA cleavage. [ 2 ]
In 2009, the first human biological drug produced from such an animal, a goat, was approved. The drug, ATryn, is an anticoagulant which reduces the probability of blood clots during surgery or childbirth was extracted from the goat's milk. [42] Human alpha-1-antitrypsin is another protein that is used in treating humans with this deficiency. [43]