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Researchers in low- and middle-income countries are developing their own IP, scaling up local manufacturing, and looking for biomarkers — all in the hope of bringing costs down and getting therapies to people who need them.
Expression profiles of single live cells are generated from Raman microscopy using deep learning, enabling us to track expression dynamics along cell reprogramming or differentiation.
We developed synthetic serum markers that can be expressed in the brain but are transported into the blood, enabling minimally invasive monitoring of gene expression in the brain with a simple blood test.
Repetitive DNA sequences known as tandem repeats (TRs) are linked to dozens of monogenic diseases and to cancer. We developed a computational method to characterize TRs using PacBio HiFi sequencing. This software can be used to discover and profile pathogenic repeat expansions and to catalog genetic and epigenetic variation in TR regions.
Chimeric antigen receptor (CAR) T cells in the solid tumor microenvironment enter a partially dysfunctional state called T cell exhaustion. Interleukin (IL)-10-producing CAR T cells retain their metabolic fitness, resist T cell exhaustion and display unprecedented antitumor activity indicated by high cure rates and durable responses in mice. Clinical studies of IL-10-producing CAR T cells are underway.
pA regulator is an RNA-based, non-immunogenic regulation system of gene expression that achieves up to 900-fold induction by using an inducer drug within the clinically safe dose range.
We developed luciferase-based splicing reporters to evaluate 718 RNA-binding proteins (RBPs) for the ability to activate exon inclusion. Our screen detected RBPs with no previously characterized roles in splicing and utilized RBP domains displaying potent exon activation to develop programmable splicing modulators with improved strength, generalizability and size over previous versions.