We assessed genome editing strategies to treat cardiomyopathy in mice including one or two doses of ABE8e, or one low, medium, or high dose of SaCas9 nuclease We conducted RNA analysis and genomic DNA

NIAID Data Ecosystem60

In vivo base editing extends lifespan of a humanized mouse model of prion disease

体内基因编辑

朊病毒疾病

Prion disease is a fatal neurodegenerative disease caused by the misfolding of prion protein (PrP) encoded by the PRNP gene. While there is currently no cure for the disease, depleting PrP in the brai

NIAID Data Ecosystem40

Structure-guided chemical modification of guide RNA enables potent non-viral Cas9-mediated in vivo genome editing. Mus musculus

体内基因编辑

RNA化学修饰

CRISPR/Cas9 has emerged as a versatile and transformative genome editing technology. However, to date, efficient in vivo genome editing has required, at least in part, the use of viral delivery system

NIAID Data Ecosystem60

In vivo CRISPR screens for Toxoplasma hyperLOPIT-unassigened proteins_2. In vivo CRISPR screens for Toxoplasma hyperLOPIT-unassigened proteins_2

弓形虫基因组

体内基因编辑

We performed in vivo CRISPR screens of Toxoplasma targeting hyperLOPIT-unassigened genes during mouse infection. Overall design: This Unassigned_2 library targeting 295 genes which consisted 2949 gRNA

NIAID Data Ecosystem40

In vivo editing in human hepatocytes using AAVHSC15 Y-121

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