SMA (Proximal spinal muscular atrophy) : Base editing rescue, in cells and in mice
- Science . 2023 Apr 21;380(6642):eadg6518
Hemophilia : Safety and efficacy of an anti–human APC antibody for prophylaxis of congenital factor deficiencies in preclinical models
- Blood . 2023 Sep 21;142(12):1071-1081
HCC (Hepatocellular carcinoma) : Therapeutic efficacy of FASN inhibition in preclinical models
- Hepatology . 2022 Oct;76(4):951-966
ACADM deficiency : Messenger RNA rescues medium-chain acyl-CoA dehydrogenase deficiency in fibroblasts from patients and a murine mode
- Hum Mol Genet . 2023 Jul 4;32(14):2347-2356
AML (Acute myeloid leukemia) : Flotetuzumab and other T-cell immunotherapies upregulate MHC class II expression on acute myeloid leukemia cells
- Blood . 2023 Apr 6;141(14):1718-1723
B-ALL (Precursor B-cell acute lymphoblastic leukemia) : Phosphorylation stabilized TET1 acts as an oncoprotein and therapeutic target
- Sci Transl Med . 2023 Mar 29;15(689):eabq8513
In vivo HSC prime editing rescues sickle cell disease in a mouse model
- Blood . 2023 Apr 27;141(17):2085-2099
Acute myeloid leukemia : Delivery of PEGylated liposomal doxorubicin by bispecific antibodies improves treatment in models of high-risk childhood leukemia
- Sci Transl Med . 2023 May 17;15(696):eabm1262
Von Willebrand disease : Efficacy of platelet-inspired hemostatic nanoparticles on bleeding in murine models
- Blood . 2023 Jun 8;141(23):2891-2900
CML (Chronic myeloid leukemia) : A single-cell atlas identifies pretreatment features of primary imatinib resistance
- Blood . 2023 Jun 1;141(22):2738-2755
