New 2.25 Million Dollar Grant will Support Gene Therapy Research for Alport Syndrome

Doctor Jeffrey Miner, who is a member of the Alport Syndrome Foundation’s (ASF) Scientific Advisory Research Network, was just awarded a Ro1 grant worth 2.25 million dollars for his research in Alport syndrome. The grant was awarded from the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK).

Ro1 grants are the primary research grants given by the NIH. Projects which receive these grants have demonstrated strong preliminary findings and are thought to have great potential for advancing a particular scientific field.

Dr. Miner is an expert in Alport syndrome. He is known internationally as an expert in this rare disease which impacts kidney function, the eyes, and the ears. Alport syndrome is a result of a mutation that affects type IV collagen a345. Dr. Miner hopes to restore this form of collagen.

As things stand, there are not any therapies approved by the FDA for this rare disease. Dr. Miner hopes to change that.

The Grant

This grant money will go toward investigating three different treatments in mouse models of Alport syndrome. First, the team will investigate small-molecule drugs. Next, they will study gene-editing therapy, or CRISPR. Finally, they will study protein biochemistry.

The ultimate goal is to restore normal function to the glomerular basement membrane (GBM) which works to help the kidneys filter as they should. We know that the type IV collagen 1345 protein network is a major component within the GBM. Unfortunately, it’s defective in this rare condition, causing structural abnormalities.

Dr. Miner hopes to bring a treatment to patients that will delay end-stage kidney disease by a significant amount.

Alport Syndrome Foundation (ASF)

The ASF is the leading nonprofit for Alport syndrome in the US. They aim to provide education for patients, conduct advocacy efforts, and fund research for the rare condition. All members of the board of directors have some form of personal connection to the disease, and all are deeply invested in the organization’s mission.

The ASF is extremely excited about this new grant awarded to Dr. Miner who has been involved in the organization for such a long time. Dr. Miner received funding from the ASF earlier this year to investigate CRISPR in Alport syndrome, and this new grant by the NIH will continue to advance the mission of more deeply understanding this condition.

Dr. Miner explains that the support he’s received from the ASF was instrumental to his ability to obtain this most recent grant.

You can read more about this new grant and how it can support Alport syndrome patients here.

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