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    Article

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

    Gene-editing technologies, which include the CRISPR–Cas nucleases1–3 and CRISPR base editors4,5, have the potential to permanently modify disease-causing genes in patients6. The demonstration of durable editing i...

    Kiran Musunuru, Alexandra C. Chadwick, Taiji Mizoguchi, Sara P. Garcia… in Nature (2021)

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    In utero CRISPR-mediated therapeutic editing of metabolic genes

    In utero gene editing has the potential to prenatally treat genetic diseases that result in significant morbidity and mortality before or shortly after birth. We assessed the viral vector–mediated delivery of ...

    Avery C. Rossidis, John D. Stratigis, Alexandra C. Chadwick… in Nature Medicine (2018)

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    Treatment of Dyslipidemia Using CRISPR/Cas9 Genome Editing

    Clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated 9 (Cas9) has recently emerged as a top genome editing technology and has afforded investigators the ability to more easily st...

    Alexandra C. Chadwick, Kiran Musunuru in Current Atherosclerosis Reports (2017)

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    Article

    Genome Editing for the Study of Cardiovascular Diseases

    The opportunities afforded through the recent advent of genome-editing technologies have allowed investigators to more easily study a number of diseases. The advantages and limitations of the most prominent ge...

    Alexandra C. Chadwick, Kiran Musunuru in Current Cardiology Reports (2017)