Code

Some of the rarest diseases have also been among the hardest to treat. But a new approach to DNA repair could begin to change that.

The team at the Center for Genome Engineering is developing a technology called Homology Mediated End-Joining. This approach has the potential to create a gene therapy platform for treating multiple DNA repair disorders that have historically been inaccessible to traditional therapy.

These disorders include:

• Fanconi anemia (FA)
• Xeroderma pigmentosum (XP)
• Cockayne syndrome (CS)
• Trichothiodystrophy (TTD)
• Bloom syndrome (BS)
• Nijmegen breakage syndrome (NBS)
• Ataxia telangiectasia (AT)
• Ataxia telangiectasia-like disorder (ATLD)
• Ataxia with oculomotor apraxia (AOA)
• Spinocerebellar ataxia with axonal neuropathy type 1 (SCAN1)
• Severe combined immunodeficiency (SCID)

Dr. Colette Rogers, a postdoctoral researcher in the Moriarity Lab, and her team are leading this groundbreaking research. The next steps include testing these approaches in preclinical mouse models and ultimately working towards clinical trials here at the University of Minnesota. Your donation will move these potential therapies forward!

This new approach is called “Homology Mediated End-Joining” and allows us to create a single gene therapy product to address each of the following DNA repair disorders thought to be inaccessible to traditional gene therapy:

These advances bring researchers closer to safe, effective, and lasting treatments for DNA repair disorders, while creating new possibilities for gene therapy as a whole.

Thank you for joining us in correcting the code and changing lives.