Muscular Dystrophy Association (MDA): Transgene-triggered safety concerns in DMD gene therapies (LOI)

Aim: to enhance understanding and formulate mitigation strategies for transgene-triggered adverse events. Examples of research that might be supported by this RFA include, but are not limited to, the following:

• Identification of ‘at risk’ patient genotypes for transgene-triggered responses as it relates to DMD mutation types
• Further investigation into the nature of immunoreactive epitopes in the dystrophin gene
• Re-design of dystrophin transgene therapies to minimize transgene-triggered responses
• Development of immunosuppression strategies to minimize transgene-triggered responses
• In vitro assays and/or models predictive of transgene-triggered immune responses

Funding: total of $200,000 ($100,000 per year over a two-year period)

https://www.mda.org/science/funding-opportunities