CRISPR interference (CRISPRi) enables programmable, reversible, and titratable
repression of gene expression (knockdown) in mammalian cells. Initial CRISPRi-mediated …

Mutations in the ataxin-2 gene (ATXN2) cause the neurodegenerative disorders
amyotrophic lateral sclerosis (ALS) and spinocerebellar ataxia type 2 (SCA2). A therapeutic …

Immune cells in the tumor microenvironment play a crucial role in cancer prognosis and
response to immunotherapy. Recent studies highlight the significance of tumor-infiltrating B …

Precise control of gene expression (knock-out, knock-in, knockdown or overexpression) is at
the heart of functional genomics–an approach to dissect the contribution of a gene/protein to …

Leveraging AAVs' versatile tropism and labeling capacity, we expanded the scale of in vivo
CRISPR screening with single-cell transcriptomic phenotyping across embryonic to adult …

Multiplexed genetic perturbations are critical for testing functional interactions among coding
or non-coding genetic elements. Compared to double-stranded DNA cutting, repressive …

The genetic landscape of neurodegenerative diseases encompasses genes affecting
multiple cellular pathways which exert effects in an array of neuronal and glial cell-types …

High-throughput genetic screening is useful for discovering critical genes or gene
sequences that trigger specific cell functions and/or phenotypes. Loss-of-function genetic …

The brain is arguably the most complex part of the human body in form and function. Much
remains unclear about the molecular mechanisms that regulate its normal and pathological …

Multiplexed genetic perturbations are critical for testing functional interactions among coding
or non-coding genetic elements. Compared to double-stranded DNA cutting, repressive …