Top Tweets for #basediting
Promising work in SMA models by @davidrliu and colleagues. Real hope for restoring native SMN expression to physiologic concentration through endogenous regulation mechanisms. #basediting #genomeediting #biotech
Today we report in @ScienceMagazine a one-time base editing treatment for Spinal Muscular Atrophy (SMA), the leading genetic cause of infant mortality worldwide, affecting ~1 in 10,000 births. https://t.co/oLp8VN0Iiw
PDF: https://t.co/JoKm9BxW89
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"off-the-shell" CAR-Ts are closer to the clinic. Great step forward by Beam therapeutics. #Basediting showing its potential!
Very pleased to announce BEAM-201, a multiplex base edited allo CAR-T therapy for T-ALL. 4x simultaneous base edits (96-99% editing at all four targets), 85% CAR+, and robust, dose dependent tumor control in vitro/in vivo. Fantastic achievement by the whole team @BeamTx.

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