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A Potent Short Hairpin RNA (SHRNA734) to Positively Select and Eliminate Genetically Modified Cells (UCLA Case No. 2016-524)

BACKGROUND: Gene therapy strategies to modify human stem cells hold great promise for curing many human diseases. However, previous clinical studies have met with limited success, largely due to the low engraftment of gene modified stem cells. One strategy to overcome this challenge involves engineering stem cells in which HPRT expression is knocked...

Published: 3/8/2024 | Inventor(s): Dong Sung An, Saki Shimizu

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Category(s): Therapeutics > Gene Therapy And Editing, Therapeutics > Infectious Diseases