CRISPR/Cas9 Correction of a Common Duchenne Muscular Dystrophy (DMD) Deletion

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Grant Year

ANF Development Grant

Grant Type

Stefan Nicolau, MD


Abigail Wexner Research Institute at Nationwide Children's Hospital


Project Summary

Dr. Nicolau’s team is working to develop a new method to correct a common DMD mutation using CRISPR/Cas9 gene editing. “This method makes use of a process called homology-independent targeted integration (HITI), which is a more efficient way of introducing DNA into muscle cells. Unlike most previously described gene editing methods for DMD, this also allows production of the full-length dystrophin protein, rather than a shortened isoform.”

The team’s short term research goals are to develop and optimize a gene editing system based on HITI to correct a common DMD mutation. This will be achieved through a series of experiments in patient-derived cells and in a mouse model of DMD. 

They are also hopeful that their research will help advance HITI and other gene therapy approaches for DMD towards clinical use.

Following completion of the research, Dr. Nicolau will present his findings at an AANEM Annual Meeting. “I am very grateful to the ANF for offering me this opportunity as an early career investigator. I look forward to being able to share results of the research with the ANF and the NM community at large in the coming years.”

  • Up to $50,000 for up to 2 years. Total award $100,000 including indirect costs.
  • Complimentary AANEM Annual Meeting registration in the year the research is presented. This travel award does not need to be included in the grant application. It is paid above in the $50,000 award.
  • Complimentary AANEM membership during funding.

“Although we are planning to initially develop this method for correction of one particular mutation, we anticipate that it can be adapted to correct a broader range of mutations in the same region.”

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