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Genetic Engineering Publications - GEG Tech top picks
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DNA transposition by protein transduction of the piggyBac transposase from lentiviral Gag precursors
BigField GEG Tech's insight:
The smart design of a lentiviral vector that brings a genome editing tool such as a protein. A useful tool for gene transfer!
BigField GEG Tech's curator insight,
December 5, 2013 9:09 AM
The smart design of a lentiviral vector that brings a genome editing tool such as a protein. A useful tool for gene transfer!
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Scoop.it!
BigField GEG Tech's insight:
The authors demonstrated in vivo as well as ex vivo genome editing using adeno-associated virus (AAV)-, lentivirus-, or particle-mediated delivery of guide RNA in neurons, immune cells, and endothelial cells using Cre-dependent Cas9 knockin mouse.
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The first CAR-T immunotherapy directed against BCMA (idecabtagene vicleucel) for patients with relapsed or refractory multiple myeloma developed by partners Bristol Myers Squibb and bluebird bio was approved by the U.S. Food and Drug Administration on March 26, 2021.
This therapy recognizes and binds BCMA (B cell maturation antigen) which is a cell surface protein that is expressed on a majority of cancerous B cells in multiple myeloma. The approval is based on the KarMMa trial of 127 patients, 100 of whom received the personalized treatment with genetically engineered autologous T cells. Their overall response rate was 72%, and 28% achieved a strict complete response. For Bristol Myers Squibb, this is the second approval for a CAR-T cell therapy. The first was for a therapy directed against CD19.
Bristol Myers Squibb and bluebird will jointly market their immunotherapy at a list price of $419,500.