Lisa J Garrett

Tissue Specific Requirement for the Inv(16) Oncogene CBFB::MYH11 in Acute Myeloid Leukemia

This study shows that CBFB::MYH11 is required for leukemia survival in spleen and peripheral blood but not in bone marrow, where it can be bypassed.

Disruption of the FGFR1-FGF23-Phosphate Axis and Targeted Therapy in a Murine Model of Osteoglophonic Dysplasia

This study creates a murine model of Osteoglophonic Dysplasia by introducing a gain-of-function FGFR1 variant, revealing pathogenic mechanisms and demonstrating therapeutic potential with infigratinib.

A humanized knock-in Col6a1 mouse recapitulates a deep-intronic splice-activating variant

The paper creates a humanized knock-in mouse model with a deep-intronic splice-activating variant to test antisense therapeutics for Collagen VI-related muscular dystrophies.

Highly-efficient Cpf1-mediated gene targeting in mice following high concentration pronuclear injection

This study shows that Cpf1 can achieve high gene targeting rates in mice, sometimes surpassing Cas9, but requires higher mRNA and guide concentrations.