Gene therapy is emerging as a powerful tool to modulate abnormal gene expression, a
hallmark of most CNS disorders. The transformative potentials of recently approved gene …

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) …

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 …

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

The possibility of collateral RNA degradation poses a concern for transcriptome
perturbations and therapeutic applications using CRISPR–Cas13. We show that collateral …

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 …

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

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

RNA-targeting small molecules (rSMs) have become an attractive modality to tackle
traditionally undruggable proteins and expand the druggable space. Among many …

The complex nature of the immunosuppressive tumor microenvironment (TME) requires
multi-agent combinations for optimal immunotherapy. Here we describe multiplex universal …