Literature DB >> 25124321

Peeking into a cool future: genome editing to delete PCSK9 and control hypercholesterolemia in a single shot.

Sergio Fazio1, Hagai Tavori2.   

Abstract

Entities:  

Keywords:  Editorials; PCSK9 protein, human; cholesterol; genetic therapy; lipoproteins

Mesh：

Substances：

Year:  2014        PMID： 25124321      PMCID： PMC4137455          DOI： 10.1161/CIRCRESAHA.114.304575

Source DB:  PubMed          Journal:  Circ Res        ISSN： 0009-7330            Impact factor:   17.367


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  22 in total

1.  The self-inhibited structure of full-length PCSK9 at 1.9 A reveals structural homology with resistin within the C-terminal domain.

Authors:  Eric N Hampton; Mark W Knuth; Jun Li; Jennifer L Harris; Scott A Lesley; Glen Spraggon
Journal:  Proc Natl Acad Sci U S A       Date:  2007-09-05       Impact factor: 11.205

2.  Loss of plasma proprotein convertase subtilisin/kexin 9 (PCSK9) after lipoprotein apheresis.

Authors:  Hagai Tavori; Ilaria Giunzioni; MacRae F Linton; Sergio Fazio
Journal:  Circ Res       Date:  2013-10-11       Impact factor: 17.367

3.  PCSK9 LNA antisense oligonucleotides induce sustained reduction of LDL cholesterol in nonhuman primates.

Authors:  Marie W Lindholm; Joacim Elmén; Niels Fisker; Henrik F Hansen; Robert Persson; Marianne R Møller; Christoph Rosenbohm; Henrik Ørum; Ellen M Straarup; Troels Koch
Journal:  Mol Ther       Date:  2011-11-22       Impact factor: 11.454

4.  Decreased plasma cholesterol and hypersensitivity to statins in mice lacking Pcsk9.

Authors:  Shirya Rashid; David E Curtis; Rita Garuti; Norma N Anderson; Yuriy Bashmakov; Y K Ho; Robert E Hammer; Young-Ah Moon; Jay D Horton
Journal:  Proc Natl Acad Sci U S A       Date:  2005-04-01       Impact factor: 11.205

5.  Molecular characterization of loss-of-function mutations in PCSK9 and identification of a compound heterozygote.

Authors:  Zhenze Zhao; Yetsa Tuakli-Wosornu; Thomas A Lagace; Lisa Kinch; Nicholas V Grishin; Jay D Horton; Jonathan C Cohen; Helen H Hobbs
Journal:  Am J Hum Genet       Date:  2006-07-18       Impact factor: 11.025

6.  Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a randomised, single-blind, placebo-controlled, phase 1 trial.

Authors:  Kevin Fitzgerald; Maria Frank-Kamenetsky; Svetlana Shulga-Morskaya; Abigail Liebow; Brian R Bettencourt; Jessica E Sutherland; Renta M Hutabarat; Valerie A Clausen; Verena Karsten; Jeffrey Cehelsky; Saraswathy V Nochur; Victor Kotelianski; Jay Horton; Timothy Mant; Joseph Chiesa; James Ritter; Malathy Munisamy; Akshay K Vaishnaw; Jared A Gollob; Amy Simon
Journal:  Lancet       Date:  2013-10-03       Impact factor: 79.321

7.  Mutations in PCSK9 cause autosomal dominant hypercholesterolemia.

Authors:  Marianne Abifadel; Mathilde Varret; Jean-Pierre Rabès; Delphine Allard; Khadija Ouguerram; Martine Devillers; Corinne Cruaud; Suzanne Benjannet; Louise Wickham; Danièle Erlich; Aurélie Derré; Ludovic Villéger; Michel Farnier; Isabel Beucler; Eric Bruckert; Jean Chambaz; Bernard Chanu; Jean-Michel Lecerf; Gerald Luc; Philippe Moulin; Jean Weissenbach; Annick Prat; Michel Krempf; Claudine Junien; Nabil G Seidah; Catherine Boileau
Journal:  Nat Genet       Date:  2003-06       Impact factor: 38.330

8.  Multiplex genome engineering using CRISPR/Cas systems.

Authors:  Le Cong; F Ann Ran; David Cox; Shuailiang Lin; Robert Barretto; Naomi Habib; Patrick D Hsu; Xuebing Wu; Wenyan Jiang; Luciano A Marraffini; Feng Zhang
Journal:  Science       Date:  2013-01-03       Impact factor: 47.728

9.  Serum proprotein convertase subtilisin/kexin type 9 and cell surface low-density lipoprotein receptor: evidence for a reciprocal regulation.

Authors:  Hagai Tavori; Daping Fan; John L Blakemore; Patricia G Yancey; Lei Ding; Macrae F Linton; Sergio Fazio
Journal:  Circulation       Date:  2013-05-20       Impact factor: 29.690

10.  Low density lipoprotein binds to proprotein convertase subtilisin/kexin type-9 (PCSK9) in human plasma and inhibits PCSK9-mediated low density lipoprotein receptor degradation.

Authors:  Tanja Kosenko; Mia Golder; Geoffrey Leblond; Willy Weng; Thomas A Lagace
Journal:  J Biol Chem       Date:  2013-02-11       Impact factor: 5.157
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  1 in total

Review 1.  PCSK9 Variants in Familial Hypercholesterolemia: A Comprehensive Synopsis.

Authors:  Qianyun Guo; Xunxun Feng; Yujie Zhou
Journal:  Front Genet       Date:  2020-09-23       Impact factor: 4.599
  1 in total

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