相关参考文献
注意：仅列出部分参考文献，下载原文获取全部文献信息。ITR-Seq, a next-generation sequencing assay, identifies genome-wide DNA editing sites in vivo following adeno-associated viral vector-mediated genome editing
Camilo Breton et al.
BMC GENOMICS (2020)
Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A
K. John Pasi et al.
NEW ENGLAND JOURNAL OF MEDICINE (2020)
Non-viral Delivery of Zinc Finger Nuclease mRNA Enables Highly Efficient In Vivo Genome Editing of Multiple Therapeutic Gene Targets
Anthony Conway et al.
MOLECULAR THERAPY (2019)
Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy
Christopher E. Nelson et al.
NATURE MEDICINE (2019)
High levels of AAV vector integration into CRISPR-induced DNA breaks
Killian S. Hanlon et al.
NATURE COMMUNICATIONS (2019)
Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10
Morgan L. Maeder et al.
NATURE MEDICINE (2019)
Somatic Editing of Ldlr With Adeno-Associated Viral-CRISPR Is an Efficient Tool for Atherosclerosis Research
Kelsey E. Jarrett et al.
ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY (2018)
Reduced Blood Lipid Levels With In Vivo CRISPR-Cas9 Base Editing of ANGPTL3
Alexandra C. Chadwick et al.
CIRCULATION (2018)
The Evolving Future of PCSK9 Inhibitors
Robert S. Rosenson et al.
JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY (2018)
Meganuclease targeting of PCSK9 in macaque liver leads to stable reduction in serum cholesterol
Lili Wang et al.
NATURE BIOTECHNOLOGY (2018)
A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing
Jonathan D. Finn et al.
CELL REPORTS (2018)
Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy
Leonela Amoasii et al.
SCIENCE (2018)
All-in-one adeno-associated virus delivery and genome editing by Neisseria meningitidis Cas9 in vivo
Raed Ibraheim et al.
GENOME BIOLOGY (2018)
Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B
Wolfgang Miesbach et al.
BLOOD (2018)
Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing
Hao Yin et al.
NATURE BIOTECHNOLOGY (2017)
Evolocumab and Clinical Outcomes in Patients with Cardiovascular Disease
Marc S. Sabatine et al.
NEW ENGLAND JOURNAL OF MEDICINE (2017)
A Highly Durable RNAi Therapeutic Inhibitor of PCSK9
Kevin Fitzgerald et al.
NEW ENGLAND JOURNAL OF MEDICINE (2017)
AAV5-Factor VIII Gene Transfer in Severe Hemophilia A
Savita Rangarajan et al.
NEW ENGLAND JOURNAL OF MEDICINE (2017)
PCSK9 Loss-of-Function Variants, Low-Density Lipoprotein Cholesterol, and Risk of Coronary Heart Disease and Stroke Data From 9 Studies of Blacks and Whites
Shia T. Kent et al.
CIRCULATION-CARDIOVASCULAR GENETICS (2017)
In Vivo Base Editing of PCSK9 (Proprotein Convertase Subtilisin/Kexin Type 9) as a Therapeutic Alternative to Genome Editing
Alexandra C. Chadwick et al.
ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY (2017)
Treatment of Dyslipidemia Using CRISPR/Cas9 Genome Editing
Alexandra C. Chadwick et al.
CURRENT ATHEROSCLEROSIS REPORTS (2017)
CRISPR-Cas9 Targeting of PCSK9 in Human Hepatocytes In Vivo-Brief Report
Xiao Wang et al.
ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY (2016)
Gene therapy for hemophilia: past, present and future
Lindsey A. George et al.
SEMINARS IN HEMATOLOGY (2016)
Comparative Study of Liver Gene Transfer With AAV Vectors Based on Natural and Engineered AAV Capsids
Lili Wang et al.
MOLECULAR THERAPY (2015)
In vivo genome editing using Staphylococcus aureus Cas9
F. Ann Ran et al.
NATURE (2015)
GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases
Shengdar Q. Tsai et al.
NATURE BIOTECHNOLOGY (2015)
Evolocumab (AMG 145) for primary hypercholesterolemia
Gisle Langslet et al.
EXPERT REVIEW OF CARDIOVASCULAR THERAPY (2015)
An evaluation of alirocumab for the treatment of hypercholesterolemia
Michel Farnier
EXPERT REVIEW OF CARDIOVASCULAR THERAPY (2015)
Permanent Alteration of PCSK9 With In Vivo CRISPR-Cas9 Genome Editing
Qiurong Ding et al.
CIRCULATION RESEARCH (2014)
Selection and evaluation of clinically relevant AAV variants in a xenograft liver model
Leszek Lisowski et al.
NATURE (2014)
Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B
A. C. Nathwani et al.
NEW ENGLAND JOURNAL OF MEDICINE (2014)
Absolute Determination of Single-Stranded and Self-Complementary Adeno-Associated Viral Vector Genome Titers by Droplet Digital PCR
Martin Lock et al.
HUMAN GENE THERAPY METHODS (2014)
Robust ZFN-mediated genome editing in adult hemophilic mice
Xavier M. Anguela et al.
BLOOD (2013)
The PCSK9 decade
Gilles Lambert et al.
JOURNAL OF LIPID RESEARCH (2012)
Histopathology of Incidental Findings in Cynomolgus Monkeys (Macaca Fascicularis) Used in Toxicity Studies
Junko Sato et al.
JOURNAL OF TOXICOLOGIC PATHOLOGY (2012)
Long-term Safety and Efficacy Following Systemic Administration of a Self-complementary AAV Vector Encoding Human FIX Pseudotyped With Serotype 5 and 8 Capsid Proteins
Amit C. Nathwani et al.
MOLECULAR THERAPY (2011)
The I-CreI meganuclease and its engineered derivatives: applications from cell modification to gene therapy
S. Arnould et al.
PROTEIN ENGINEERING DESIGN & SELECTION (2011)
Rapid, Simple, and Versatile Manufacturing of Recombinant Adeno-Associated Viral Vectors at Scale
Martin Lock et al.
HUMAN GENE THERAPY (2010)
Worldwide Epidemiology of Neutralizing Antibodies to Adeno-Associated Viruses
Roberto Calcedo et al.
JOURNAL OF INFECTIOUS DISEASES (2009)
Proprotein convertase Subtilisin/Kexin type 9 (PCSK9): Hepatocyte-specific low-density lipoprotein receptor degradation and critical role in mouse liver regeneration
Ahmed Zaid et al.
HEPATOLOGY (2008)
The C679X mutation in PCSK9 is present and lowers blood cholesterol in a Southern African population
Amanda J. Hooper et al.
ATHEROSCLEROSIS (2007)
Molecular characterization of loss-of-function mutations in PCSK9 and identification of a compound heterozygote
Zhenze Zhao et al.
AMERICAN JOURNAL OF HUMAN GENETICS (2006)
Decreased plasma cholesterol and hypersensitivity to statins in mice lacking Pcsk9
S Rashid et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2005)
Clades of Adeno-associated viruses are widely disseminated in human tissues
GP Gao et al.
JOURNAL OF VIROLOGY (2004)
分享论文
