Towards a cure for hars disease

HIGHLIGHTS

• who: Sarah D. P. Wilhelm et al. from the Department of Biochemistry, The University of Western Ontario, London, ON N A , Canada have published the research work: Towards a Cure for HARS Disease, in the Journal: Genes 2023, 254 of /2023/
• how: Recent data showed that both loss-of-function and gain-of-function of different aaRSs can lead to CMT phenotypes and both mechanisms may not be mutually exclusive.
• future: For USH3B amino_acid supplementation as a treatment is currently being considered in a clinical trial and with the astonishing progress in RNA . . .
  

   

  

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