Scot Wolfe receives funding for collaborative research to develop genome editing strategy for frontotemporal dementia

Scot Wolfe, PhD, is part of a multidisciplinary team at UMass Chan, along with Erik Sontheimer, Fen-Biao Gao and Jonathan Watts in the RNA Therapeutics Institute, that has received an NIH grant from the National Institute of Neurological Disorders and Stroke (NINDS) to develop precision editing of granulin mutations that cause frontotemporal dementia.

Frontotemporal dementia (FTD) is the most common form of dementia in people under 60, and currently there is no cure or treatment to slow the progression of the disease. The collaborative grant aims to develop an effective precision genome editing approach to correct mutations in the Granulin (GRN) gene, which account for approximately 20–25% of hereditary FTD cases, or about 10% of total FTD cases. The efficacy of the candidate genome editing approaches will be tested in induced pluripotent stem cell (iPSC)-derived neurons and glial cells, and their effectiveness in preventing disease progression will be assessed using a humanized FTD mouse model.

The research aims to not only lead to an effective treatment strategy for FTD associated with GRN mutations, but also to establish a roadmap for developing clinical genome editing approaches to correct mutations in other genes that cause FTD and other forms of dementia.