In vivo somatic cell base editing and prime editing

GA Newby, DR Liu - Molecular Therapy, 2021 - cell.com
Recent advances in genome editing technologies have magnified the prospect of single-
dose cures for many genetic diseases. For most genetic disorders, precise DNA correction is …

In vivo delivery of CRISPR-Cas9 genome editing components for therapeutic applications

K Huang, D Zapata, Y Tang, Y Teng, Y Li - Biomaterials, 2022 - Elsevier
Since its mechanism discovery in 2012 and the first application for mammalian genome
editing in 2013, CRISPR-Cas9 has revolutionized the genome engineering field and created …

In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels

T Rothgangl, MK Dennis, PJC Lin, R Oka… - Nature …, 2021 - nature.com
Most known pathogenic point mutations in humans are C• G to T• A substitutions, which can
be directly repaired by adenine base editors (ABEs). In this study, we investigated the …

[HTML][HTML] The functional role of lipoproteins in atherosclerosis: novel directions for diagnosis and targeting therapy

Y Lu, X Cui, L Zhang, X Wang, Y Xu, Z Qin… - Aging and …, 2022 - ncbi.nlm.nih.gov
Dyslipidemia, characterized by a high level of lipids (cholesterol, triglycerides, or both), can
increase the risk of developing and progressing atherosclerosis. As atherosclerosis …

Statins and PCSK9 inhibitors: A new lipid-lowering therapy

E Gallego-Colon, A Daum, C Yosefy - European journal of pharmacology, 2020 - Elsevier
The clinical benefit of lipid-lowering therapies is to reduce circulating levels of atherogenic
particles and to ameliorate the risk of atherosclerotic cardiovascular disease (ASCVD). The …

Precise mutagenesis in zebrafish using cytosine base editors

M Rosello, M Serafini, JP Concordet, F Del Bene - Nature Protocols, 2023 - nature.com
Base editing is a powerful CRISPR-based technology for introducing precise substitutions
into the genome. This technology greatly advances mutagenesis possibilities in vivo …

Development of small-molecule PCSK9 inhibitors for the treatment of hypercholesterolemia

S Ahamad, S Mathew, WA Khan, K Mohanan - Drug Discovery Today, 2022 - Elsevier
When secreted into the circulation, proprotein convertase subtilisin kexin type 9 (PCSK9)
blocks the low-density lipoprotein receptors (LDL-R) and, as a consequence, low-density …

Gene editing and its applications in biomedicine

G Li, X Li, S Zhuang, L Wang, Y Zhu, Y Chen… - Science China Life …, 2022 - Springer
The steady progress in genome editing, especially genome editing based on the use of
clustered regularly interspaced short palindromic repeats (CRISPR) and programmable …

A Type II-B Cas9 nuclease with minimized off-targets and reduced chromosomal translocations in vivo

B Bestas, S Wimberger, D Degtev, A Madsen… - Nature …, 2023 - nature.com
Abstract Streptococcus pyogenes Cas9 (SpCas9) and derived enzymes are widely used as
genome editors, but their promiscuous nuclease activity often induces undesired mutations …

Advances in biological therapies for dyslipidemias and atherosclerosis

EK Valanti, K Dalakoura-Karagkouni, G Siasos… - Metabolism, 2021 - Elsevier
Atherosclerosis is a multifactorial disease influenced by genetics, lifestyle and
environmental factors. Despite therapeutic advances that reduce the risk of cardiovascular …