30 Mar 2023
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
30 Mar 2023
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. science.org/doi/10.1126/scie…
PDF: drive.google.com/file/d/1H1k…
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27 Jul 2021
I promised, here it is: biorxiv.org/content/10.1101/…
our new online tool to facilitate sgRNA picking for basediting. predict offtargets and select sgRNAs for non-synonymous and nonsense mutations ✏️ aceofbases.cos.uni-heidelber…
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10 Nov 2020
"off-the-shell" CAR-Ts are closer to the clinic. Great step forward by Beam therapeutics. #Basediting showing its potential!
9 Nov 2020
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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