Anne Marie Mazzola

In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates

Kiran Musunuru, Alexandra C. Chadwick, Taiji Mizoguchi et al. · Nature · 2021 · 768 citations

Life Sciences, Genetics, Molecular Biology +12

Activating <i>ESR1</i> Mutations Differentially Affect the Efficacy of ER Antagonists

Weiyi Toy, Hazel M. Weir, Pedram Razavi et al. · Cancer Discovery · 2016 · 370 citations · Full text

Medicine, Endocrinology, Oncology +17

Efficacy and Safety of an Investigational Single-Course CRISPR Base-Editing Therapy Targeting <i>PCSK9</i> in Nonhuman Primate and Mouse Models

Richard Lee, Anne Marie Mazzola, Maurine Braun et al. · Circulation · 2022 · 220 citations

Life Sciences, Biotechnology, Genetics +16

AZD9496: An Oral Estrogen Receptor Inhibitor That Blocks the Growth of ER-Positive and <i>ESR1</i> -Mutant Breast Tumors in Preclinical Models

Hazel M. Weir, Robert H. Bradbury, Mandy Lawson et al. · Cancer Research · 2016 · 174 citations

Medicine, Oncology, Pharmacology +16

GalNAc-Lipid nanoparticles enable non-LDLR dependent hepatic delivery of a CRISPR base editing therapy

Lisa N. Kasiewicz, Souvik Biswas, Aaron Beach et al. · Nature Communications · 2023 · 145 citations · Full text

Pharmacy, Biotechnology, Pharmaceutics +17