We endeavor to selectively target one allele (one copy) of gene that causes disease, so that we can edit the mutant allele using CRISPR and restore cell and tissue health. To do this, we need to know the most common single nucleotide polymorphisms (SNPs) in the general population and patient populations. We would like to input a genomic region and learn: 1) which SNPs are present and in what frequency, 2) whether they are phased to a mutation (for patient sequencing data), 3) whether SNPs are phased to one another and 3) gRNAs that target that SNP (ie., whether there is a PAM nearby). This then becomes the basis of our gene therapy. A program like http://crispor.tefor.net/ but for allele specific editing would also be very beneficial to the larger community! 

UCSF GenenCal - Fall 2022 Discovery Project
Fall 2022
Public Health