On August 25, the A-T Children’s Project sent the below email to A-T researchers about available funds for a grant to see if exon skipping would make sense as a therapeutic approach for A-T.

Dear A-T Researchers,

We wanted to let you know of a funding opportunity through a partnership between the University of Pennsylvania’s Orphan Disease Center and the A-T Children’s Project.

One grant for $53,240 is available to investigate the functional effects and potential therapeutic benefits of exon skipping in specific regions of the ATM gene for the treatment of ataxia telangiectasia (A-T). We are hoping that a researcher will be able to determine what happens when you skip exons in the A-T gene, increasing our understanding about how ATM biology works and to see if exon skipping would make sense as a therapeutic approach for A-T.

The application deadline for Letters of Interest (LOI) is September 15, 2023. You can find the application on the Penn website. The RFA is at the same link (just scroll down to find A-T) and also at the bottom of this email.

Penn website: https://www.orphandiseasecenter.med.upenn.edu/grants/event-five-pk3yg-b7zlm-m4ag9-e5ysf

Please feel free to share this information with respected colleagues who may bring innovative approaches to this scientific question. Thank you for your work which gives hope to all families coping with A-T!

All the best,

Jennifer
Jennifer Thornton, Executive Director

RFA for A-T:
Ataxia-Telangiectasia (A-T): A grant of $53,240 has been provided by Team Derek’s Dreams and the A-T Children’s Project to support a crucial research endeavor aimed at investigating the functional effects and potential therapeutic benefits of exon skipping in specific regions of the ATM gene for the treatment of ataxia telangiectasia (A-T). Targeted exon-skipping therapies, which have had success in other diseases, hold promise for children with A-T as they could potentially enable disease-causing mutations to be ignored. However, the application of this approach to A-T necessitates a more comprehensive understanding of the structure, function, and consequences of exon skipping within the ATM gene. By employing a combination of in silico and in vitro techniques to systematically examine each exon, the recipient of this grant will make groundbreaking contributions to the field, not only enlightening the A-T research community but providing actionable insights to developers of genetic medicines regarding regions within the ATM gene that could be omitted.

If you are an A-T or ATM researcher and would like to be added to our mailing list, please email us at info@atcp.org. Thank you!

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