摘要
In a new Science publication,Arbab and colleagues observe a substantial improvement in life span and motor functions of adenosine-base edited spinal muscular atrophy(SMA)mice with restored endogenous survival motor neuron(SMN)protein expression and regulation.1 This study provides proof-of-principle for the use of adenosine base editing(ABE)as a possible one-time,permanent therapy for SMA.
基金
This work was supported by project grants from the German Research Foundation(Deutsche Forschungsgemeinschaft,project numbers CA 893/18-1 and GA 402/25-1)
by TU Dresden.
