Seattle Children's Hospital

Yuchi Chiang Honaker

Andrew M Scharenberg

David J Rawlings

Karen Sommer

Ryo Takeuchi

Iram F Khan

Swati Singh

Samuel West

The present application relates to, inter alia, compositions including proteins for expression in host cells to render them resistant to rapamycin. The application further relates to methods of using the proteins, cells, and compositions disclosed therein for modulating cell signaling and for selective expansion of cells.

Rapamycin resistant cells

Aspects of the invention described herein concern the incorporation of a FOXP3 cDNA (e.g., full-length human codon-optimized cDNA) into a FOXP3 gene or a non-FOXP3 locus so as to provide constitutive or regulated FOXP3 expression in a primary human CD34cells or cells derived from edited CD34cells. In some embodiments, guide RNA sequences that are directed to FOXP3, AAVS1, or other candidate loci are used for CRISPR/Cas9-mediated gene regulation, and gene delivery cassettes for HDR based gene-modification are provided.

Expression of FOXP3 in edited CD34+ cells

Growing community of inventors

Seattle, WA, United States of America

Yuchi Chiang Honaker