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On 26 November 2018, The CRISPR Journal published ahead of print an article by He, Ryan Ferrell, Chen Yuanlin, Qin Jinzhou, and Chen Yangran in which the authors justified the ethical use of CRISPR gene editing in humans. [74] As the news of CRISPR babies broke out, the editors reexamined the paper and retracted it on 28 December, announcing:
Modifying human embryos to give the CCR5 Δ32 allele protects them from the disease. An other use would be to cure genetic disorders. In the first study published regarding human germline engineering, the researchers attempted to edit the HBB gene which codes for the human β-globin protein. HBB mutations produce β-thalassaemia, which can be ...
In February 2020, a US trial showed safe CRISPR gene editing on three cancer patients. [38] In October 2020, researchers Emmanuelle Charpentier and Jennifer Doudna were awarded the Nobel Prize in Chemistry for their work in this field. [39] [40] They made history as the first two women to share this award without a male contributor. [41] [5]
Image source: CRISPR Therapeutics. 2. Profitability remains elusive. There's some optimism that CRISPR Therapeutics is still in the early stages of a significant long-term opportunity.
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 order for gene editing technologies to make the leap towards safe and widespread use in the clinic, the rate of off-target modification needs to be rendered obsolete. The safety of gene therapy treatment is of utmost concern, especially during clinical trials when off-target modifications can block the further development of a candidate ...
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 ]
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