Thirteen months after He Jiankui shook the international scientific community with the news of an experiment to create the first gene-edited babies, a Chinese court has sentenced He and two colleagues to three years in prison and a fine of roughly $400,000. Producing the CRISPR babies, which did not gain the support of the scientific community, brought the ethics that surround CRISPR genome editing technology to the forefront of scientific and ethical debates.
Researchers have succeeded in inhibiting the immune response induced by AAV antibodies using Imlifidase (IdeS), an enzyme that is able to degrade circulating IgG. This advance provides a potential solution to the limitations that exist due to pre-existing antibodies to AAV-based gene therapy, and may allow for repeated administration of gene therapy. The results open up new therapeutic prospects and the possibility of treating more patients.
Mosquito-specific refinements to gene-editing system may streamline development of transgenic strains for field releases.
Allergan subsidiary exercises option to commercialize CRISPR genome editing treatment candidate EDIT-101 globally for Leber Congenital Amaurosis type 10, a development effort that could generate up to $40 million for Editas.
Editing enzymes delivered by viral vector to the striatum in brain.
Results from study using peptide nucleic acid gene editing in mouse model suggests technology could be used to treat genetic disorders in humans before birth.
Scientists have used CRISPR-Cas9 technology to identify potential new targets for glioblastoma, the most common type of primary brain tumor in adults. The approach used genome editing to effectively reverse engineer patient-derived, tumor-propagating glioblastoma stem cells gene by gene, and identify genes that are essential for tumor growth. The studies provided new insights into the basis of glioblastoma resistance to temozolomide chemotherapy, and potential strategies for combination treatment.
Using induced pluripotent stem cells and deleting a key gene, researchers have created natural killer cells with measurably stronger activity against a form of leukemia, both in vivo and in vitro. Deleting the gene removes a brake on IL-15 signaling, which improves NK cell activation and function. It also leads to metabolic reprogramming of the NK cells, which become more efficient at energy utilization. The new approach has other advantages. iPSCs provide a stable platform for gene modification, and NK cells can be used as allogeneic cells that do not need to be matched to individual patients.
Genomic medicine is borrowing from Greek theatre a device called God in the Machine. In genomic medicine, the device manifests as AI technology, but it can still resolve complicated situations.
Automation includes not only the bioprocess but also testing and all other manufacturing-associated functions such as assurance, supply chain, and storage.