Musunuru, Kiran

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

Musunuru, K., Chadwick, A. C., Mizoguchi, T., Garcia, S. P., DeNizio, J. E., Reiss, C. W., Wang, K., Iyer, S., Dutta, C., Clendaniel, V., et al. (2021) · Nature

CRISPR and Genetic EngineeringLipoproteins and Cardiovascular Health

Cited by 793

Exome Sequencing, ANGPTL3 Mutations, and Familial Combined Hypolipidemia

Musunuru, K., Pirruccello, J. P., Do, R., Peloso, G. M., Guiducci, C., Sougnez, C., Garimella, K. V., Fisher, S., Abreu, J., Barry, A. J., et al. (2010) · New England Journal of Medicine

Lipid metabolism and disordersLipoproteins and Cardiovascular Health

Cited by 754

Engineered Virus-like Particles for Efficient in Vivo Delivery of Therapeutic Proteins

Banskota, S., Raguram, A., Suh, S., Du, S. W., Davis, J. R., Choi, E. H., Wang, X., Nielsen, S. C., Newby, G. A., Randolph, P. B., et al. (2022) · Cell

RNA Interference and Gene DeliveryCRISPR and Genetic Engineering

Cited by 668

A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice

Yang, Y., Wang, L., Bell, P., McMenamin, D., He, Z., White, J., Yu, H., Xu, C., Morizono, H., Musunuru, K., et al. (2016) · Nature Biotechnology

Virus-based gene therapy researchCRISPR and Genetic Engineering

Cited by 556

Permanent Alteration of PCSK9 With In Vivo CRISPR-Cas9 Genome Editing

Ding, Q., Strong, A., Patel, K. M., Ng, S.-L., Gosis, B. S., Regan, S. N., Cowan, C. A., Rader, D. J., & Musunuru, K. (2014) · Circulation Research

CRISPR and Genetic EngineeringLipoproteins and Cardiovascular Health

Cited by 522

Efficient ablation of genes in human hematopoietic stem and effector cells using CRISPR/Cas9

Mandal, P. K., Ferreira, L. M. R., Collins, R., Meissner, T. B., Boutwell, C. L., Friesen, M., Vrbanac, V., Garrison, B. S., Stortchevoi, A., Bryder, D., et al. (2014) · Cell stem cell

CRISPR and Genetic EngineeringCAR-T cell therapy research

Cited by 475

Low Incidence of Off-Target Mutations in Individual CRISPR-Cas9 and TALEN Targeted Human Stem Cell Clones Detected by Whole-Genome Sequencing

Veres, A., Gosis, B. S., Ding, Q., Collins, R., Ragavendran, A., Brand, H., Erdin, S., Cowan, C. A., Talkowski, M. E., & Musunuru, K. (2014) · Cell Stem Cell

CRISPR and Genetic EngineeringRNA regulation and disease

Cited by 474

ANGPTL3 deficiency and protection against coronary artery disease

Stitziel, N. O., Khera, A. V., Wang, X., Bierhals, A. J., Vourakis, A. C., Sperry, A. E., Natarajan, P., Klarin, D., Emdin, C. A., Zekavat, S. M., et al. (2017) · Journal of the American College of Cardiology

Lipid metabolism and disordersLipoproteins and Cardiovascular Health

Cited by 467

Human Germline Genome Editing

Ormond, K. E., Mortlock, D. P., Scholes, D. T., Bombard, Y., Brody, L. C., Faucett, W. A., Garrison, N. A., Hercher, L., Isasi, R., Middleton, A., et al. (2017) · The American Journal of Human Genetics

CRISPR and Genetic EngineeringBiomedical Ethics and Regulation

Cited by 252

In utero CRISPR-mediated therapeutic editing of metabolic genes

Rossidis, A. C., Stratigis, J. D., Chadwick, A. C., Hartman, H. A., Ahn, N. J., Li, H., Singh, K., Coons, B. E., Li, L., Lv, W., et al. (2018) · Nature Medicine

CRISPR and Genetic EngineeringVirus-based gene therapy research

Cited by 216

Surprises from genetic analyses of lipid risk factors for atherosclerosis

Musunuru, K., & Kathiresan, S. (2016) · Circulation Research

Lipoproteins and Cardiovascular HealthLipid metabolism and disorders

Cited by 159

Plasma HDL cholesterol and risk of myocardial infarction: a mendelian randomisation study

Voight, B. F., Peloso, G. M., Orho-Melander, M., Frikke-Schmidt, R., Barbalic, M., Jensen, M. K., Hindy, G., Hólm, H., Ding, E. L., Johnson, T., et al. (2012) · The Lancet

Genetic Associations and EpidemiologyLipoproteins and Cardiovascular Health

Cited by 148

A mouse model of the human Fragile X syndrome I304N mutation

Zang, J. B., Nosyreva, E. D., Spencer, C. M., Volk, L. J., Musunuru, K., Zhong, R., Stone, E. F., Yuva-Paylor, L. A., Huber, K. M., Paylor, R., et al. (2009) · PLoS Genetics

Genetics and Neurodevelopmental DisordersNeuroscience and Neuropharmacology Research

Cited by 138

In utero gene editing for monogenic lung disease

Alapati, D., Zacharias, W. J., Hartman, H. A., Rossidis, A. C., Stratigis, J. D., Ahn, N. J., Coons, B., Zhou, S., Li, H., Singh, K., et al. (2019) · Science Translational Medicine

CRISPR and Genetic EngineeringVirus-based gene therapy research

Cited by 123

In utero adenine base editing corrects multi-organ pathology in a lethal lysosomal storage disease

Bose, S. K., White, B. M., Kashyap, M. V., Dave, A., De Bie, F. R., Li, H., Singh, K., Menon, P., Wang, T., Teerdhala, S., et al. (2021) · Nature Communications

Lysosomal Storage Disorders ResearchVirus-based gene therapy research

Cited by 67

Low incidence of off-target mutations in individual CRISPR-Cas9 and TALEN targeted human stem cell clones detected by whole-genome sequencing

Veres, A., Gosis, B. S., Ding, Q., Collins, R., Ragavendran, A., Brand, H., Erdin, S., Cowan, C. A., Talkowski, M. E., & Musunuru, K. (2014) · Cell stem cell

CRISPR and Genetic EngineeringRNA regulation and disease

Cited by 37

Genome-edited human pluripotent stem cell–derived macrophages as a model of reverse cholesterol transport—brief report

Gupta, R. M., Meissner, T. B., Cowan, C. A., & Musunuru, K. (2016) · Arteriosclerosis, Thrombosis, and Vascular Biology

Cholesterol and Lipid MetabolismPluripotent Stem Cells Research

Cited by 37