Allele Prospector: Leveraging human genetic variation to enable therapeutic genome editing in hundreds of disease genes

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Grant Award Details

Grant Number:
DISC0-17363
Investigator(s):
Award Value:
$5,112,209
Status:
Pre-Active

Grant Application Details

Application Title:

Allele Prospector: Leveraging human genetic variation to enable therapeutic genome editing in hundreds of disease genes

Public Abstract:
Research Objective

We are building a foundational platform for developing genome editing technologies that increase patient coverage by 20-40X and can be applied to over 700 genetic diseases in diverse populations.

Impact

Many genetic diseases have hundreds of different mutations so correcting mutations one-by-one is impractical. We are developing an approach that can reach more patients with curative treatments.