Over the past 20 years, and particularly in the last decade, significant developmental
milestones have driven basic, translational, and clinical advances in the field of stem cell …

Induced pluripotent stem cells (iPSCs) can self-renew indefinitely in culture and differentiate
into all specialized cell types including gametes. iPSCs do not exist naturally and are …

Haploinsufficiency of the progranulin (PGRN)‐encoding gene (GRN) causes frontotemporal
lobar degeneration (GRN‐FTLD) and results in microglial hyperactivation, TREM2 …

The ability to precisely edit the genome of human induced pluripotent stem cell (iPSC) lines
using CRISPR/Cas9 has enabled the development of cellular models that can address …

CRISPR genome editing is a promising tool for translational research but can cause
undesired editing outcomes, both on target at the edited locus and off target at other …

Since its inception, the CRISPR/Cas9 technology has been widely utilized for the targeted
insertion of donor DNAs into mammalian genomes. A shortcoming with the earlier knock-in …

Central norepinephrine (NE) neurons, located mainly in the locus coeruleus (LC), are
implicated in diverse psychiatric and neurodegenerative diseases and are an emerging …

The precise regulation of the activity of Cas9 is crucial for safe and efficient editing. Here we
show that the genome-editing activity of Cas9 can be constrained by the addition of cytosine …

Genome editing technologies hold great promise for numerous applications including the
understanding of cellular and disease mechanisms and the development of gene and …

Dominantly inherited disorders are not typically considered to be therapeutic candidates for
gene augmentation. Here, we utilized induced pluripotent stem cell-derived retinal pigment …