Catecholaminergic polymorphic ventricular tachycardia (CPVT) is a lethal inherited arrhythmia disorder most commonly caused by missense mutations in the RyR2 gene. The goal of this study was to determ

NIAID Data Ecosystem00

Homology mediated end joining enables efficient non-viral targeted integration of large DNA templates in primary human T cells

基因编辑治疗

CAR-T细胞工程

Adoptive cellular therapy using genetically engineered immune cells holds tremendous promise for the treatment of advanced cancers. While the number of available receptors targeting tumor specific ant

NIAID Data Ecosystem00

PAM-Flexible Genome Editing with an Engineered Chimeric Cas9

CRISPR-Cas9

基因编辑治疗

CRISPR enzymes require a defined protospacer adjacent motif (PAM) flanking a guide RNA-programmed target site, limiting their sequence accessibility for robust genome editing applications. In this stu

NIAID Data Ecosystem10

CRISPR editing of HPFH3 genotype induces gamma-globin expression and reverses sickle cell disease and beta-thalassemia phenotypes

基因编辑治疗

红白血病

Amplicon sequencing to assess the off-target activity of spCas9 at 5' breakpoint and 3' breakpoint sgRNA targetting HPFH3 genotype.

NIAID Data Ecosystem00