Isogenic gaa-ko murine muscle cell lines mimicking severe pompe mutations as preclinical models for the screening of potential gene therapy strategies

HIGHLIGHTS

• who: Araceli Aguilar-Gonzu00e1lez and colleagues from the GENYO, Centre for Genomics and Oncological Research: Pfizer, University of Granada, Andalusian Regional , Granada, Spain have published the Article: Isogenic GAA-KO Murine Muscle Cell Lines Mimicking Severe Pompe Mutations as Preclinical Models for the Screening of Potential Gene Therapy Strategies, in the Journal: (JOURNAL) of 26/01/2022
• what: One of the aims of this manuscript was to generate a murine-murine cellular model to investigate rationally designed murine GAA chimeras to refine the strategy before translation into animal models.
• how: Mice were sacrificed . . .
  

   

  

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