Stem Cell-based Therapies for CF, Other Diseases Supported by $20.5M in Grants from CIRM

Three Stanford University School of Medicine researchers have won grants to advance the development of stem cell-based therapies, including gene editing, for cystic fibrosis (CF), autoimmune disorders and liver disease.

The grants are part of $20.5 million awarded to 11 researchers by the California Institute for Regenerative Medicine (CIRM) during the current round of funding, and are included in the state stem cell agency’s Quest program.

Matthew Porteus, MD, associate professor of pediatrics at Stanford, received $2.2 million to investigate the use of gene editing to correct CF mutations in airway stem cells. Such research has begun to demonstrate the possibility of replacing mutated genes through the use of CRISPR-Cas9. This system uses a protein-RNA complex composed of an enzyme (or protein) – Cas9 – bound to a guide RNA (gRNA) molecule that recognizes the wrong DNA sequence and cuts it out. The cell can then fill the excised portion with the correct gene sequence.

In addition, Stanford’s Rosa Bacchetta, MD, associate professor of pediatrics, received $1.1 million for her research into a gene-editing technique to repair blood stem cells from patients with a rare genetic autoimmune disease called IPEX. Roeland Nusse, PhD, professor of developmental biology, won $1.7 million to investigate how to grow liver stem cells in the lab while maintaining their regenerative capacity – cells that could potentially treat severe liver disease or alleviate a shortage of organs from donors.

CF is caused by a mutation in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR) protein. Normal CFTR proteins channel water and charged ions in and out of cells, creating thin mucus that protects and lubricates internal organs. The defective CFTR protein, however, alters this process, resulting in thick mucus that obstructs airways and traps bacteria.

“At CIRM, we never underestimate the importance of early stage scientific research; it is the birthplace of groundbreaking discoveries,” Randal Mills, PhD, president and CEO of CIRM, said in a press release. “We hope these Quest awards will not only help these incredibly creative researchers deepen our understanding of several different diseases, but also lead to new approaches on how best to use stem cells to develop treatments.”