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Candida albicans is a human pathogen that causes potentially lethal infections in immunocompromised individuals. Efforts to overcome Candida’s innate resistance to many drugs have been thwarted by an absence of tools enabling genetic modifications. Now, using a modified CRISPR-Cas system, Whitehead Institute researchers can edit the fungus’s genome systematically—an approach that could help scientists understand Candida’s unique biology and identify potential drug targets.

Although the genome editing system known as CRISPR/Cas has revolutionized genetic research in cell lines, its overall efficiency has been relatively poor when used to generate genetically altered animals for disease modeling.  Now Whitehead Institute scientists have altered the approach in a manner that could accelerate the production of mice carrying precise mutations of multiple genes.

Induced neural stem cells (iNSCs) hold promise for therapeutic transplantation, but their potential in this capacity has been limited by failed efforts to maintain such cells in their multi-potent NSC state. Now, Whitehead Institute scientists have created iNSCs that remain in the multi-potent state—without ongoing expression of reprogramming factors. This allows the iNSCs to self-renew repeatedly to generate cells in quantities sufficient for therapy.