Splicing mutations in the cftr gene as therapeutic targets

HIGHLIGHTS

• who: Karine Deletang from the (UNIVERSITY) have published the research: Splicing mutations in the CFTR gene as therapeutic targets, in the Journal: (JOURNAL) of 13/09/2021
• what: This approach has been evaluated in CF and in other diseases, such as hemophilia A, retinitis pigmentosa and SMA .
• how: Potentiators that should increase the amount of mature CFTR at the cell surface) and VX-561 (a modified form of VX-770 that should increase CFTR function) is tested in a phase 3 clinical trial. This review discusses the existing alternative strategies for these patients . . .
  

   

  

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