Huntington's disease (HD) is a dominantly inherited neurodegenerative disorder caused by
a trinucleotide repeat expansion in the huntingtin gene resulting in long stretches of …

Transcriptome engineering applications in living cells with RNA-targeting CRISPR effectors
depend on accurate prediction of on-target activity and off-target avoidance. Here we design …

Introduction Macromolecular X-ray crystallography and cryo-EM are currently the primary
techniques used to determine the three-dimensional structures of proteins, nucleic acids …

The TDP-43 proteinopathies, which include amyotrophic lateral sclerosis and frontotemporal
dementia, are a devastating group of neurodegenerative disorders that are characterized by …

Mutations in Rhodopsin (RHO) gene commonly cause autosomal dominant retinitis
pigmentosa (adRP) without effective therapeutic treatment so far. Compared with genomic …

CRISPR-Cas13d cleaves RNA and is used in vivo and for diagnostics. However, a
systematic understanding of its RNA binding and cleavage specificity is lacking. Here, we …

One of the more transformative findings in human genetics was the discovery that the
expansion of unstable nucleotide repeats underlies a group of inherited neurological …

Huntington's disease (HD) arises from the abnormal expansion of CAG repeats in the
huntingtin gene (HTT), resulting in the production of the mutant huntingtin protein (mHTT) …

CRISPR‐Cas13 holds substantial promise for tissue repair through its RNA editing
capabilities and swift catabolism. However, conventional delivery methods fall short in …

Human pluripotent stem cells (hPSCs) are uniquely suited to study human development and
disease and promise to revolutionize regenerative medicine. These applications rely on …