Mark A. Behlke

UNCOVERseq is a workflow for sensitive off-target nomination in CRISPR-Cas editing, demonstrating superior performance across multiple modalities and systems.

The paper analyzes CRISPR-Cas9 off-target mutations and translocations at single-cell and tissue-specific levels in mice, revealing context-dependent variability in editing outcomes.

The paper identifies how 3-mer oligonucleotides with base and sugar modifications modulate TLR7 and TLR8 responses, leading to the development of a dual inhibitory oligonucleotide for autoimmune disease.

The paper identifies 2'-O-methyl-guanosine RNA fragments as natural antagonists of TLR7/8, preventing auto-immunity by inhibiting TLR signaling.

This study shows that silencing CCR2 in sensory neurons blocks bone cancer progression and alleviates pain by disrupting neuropeptide-driven tumor growth.

This study compares CRISPR off-target detection tools using ex vivo edited CD34+ HSPCs, finding that HiFi Cas9 reduces off-target sites and some methods outperform others in sensitivity and specificity.

This study uses gene editing in hematopoietic stem cells to treat Pyruvate Kinase Deficiency Hemolytic Anemia, restoring RPK function and ATP levels in erythroid cells.

CRISPAltRations is a cloud-based tool that improves the analysis of CRISPR-induced DNA repair events by outperforming existing software in annotating indels and HDR events.

This study replaces the HBA1 gene with HBB in β-thalassemia-derived HSPCs using CRISPR, normalizing globin ratios and restoring functional hemoglobin.

This paper identifies engineered transfer RNAs (ACE-tRNA) that suppress premature termination codons in mammalian cells and mice, showing high suppression activity for common disease-causing nonsense codons.

Cas9-mediated correction of the sickle mutation in human hematopoietic stem cells achieves curative levels of editing.