标题
Suppression-Replacement
KCNQ1
Gene Therapy for Type 1 Long QT Syndrome
作者
关键词
-
出版物
CIRCULATION
Volume -, Issue -, Pages -
出版商
Ovid Technologies (Wolters Kluwer Health)
发表日期
2021-01-28
DOI
10.1161/circulationaha.120.051836
参考文献
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。- Gene therapy for inherited arrhythmias
- (2020) Vassilios J Bezzerides et al. CARDIOVASCULAR RESEARCH
- Impact of age and motor function in a phase 1/2A study of infants with SMA Type 1 receiving single-dose gene replacement therapy
- (2019) Linda P. Lowes et al. PEDIATRIC NEUROLOGY
- The genetic architecture of long QT syndrome: A critical reappraisal
- (2018) John R. Giudicessi et al. TRENDS IN CARDIOVASCULAR MEDICINE
- Human Induced Pluripotent Stem Cell-Derived Cardiac Cell Sheets Expressing Genetically Encoded Voltage Indicator for Pharmacological and Arrhythmia Studies
- (2018) Naim Shaheen et al. Stem Cell Reports
- Contemporary Outcomes in Patients With Long QT Syndrome
- (2017) Ram K. Rohatgi et al. JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY
- Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial
- (2017) Stephen Russell et al. LANCET
- ClinVar: improving access to variant interpretations and supporting evidence
- (2017) Melissa J Landrum et al. NUCLEIC ACIDS RESEARCH
- Allele-Specific Quantitative PCR for Accurate, Rapid, and Cost-Effective Genotyping
- (2016) Han B. Lee et al. HUMAN GENE THERAPY
- Mechanism and regulation of the nonsense-mediated decay pathway
- (2016) Nele Hug et al. NUCLEIC ACIDS RESEARCH
- Recessive cardiac phenotypes in induced pluripotent stem cell models of Jervell and Lange-Nielsen syndrome: Disease mechanisms and pharmacological rescue
- (2014) Miao Zhang et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Allele-specific RNA interference rescues the long-QT syndrome phenotype in human-induced pluripotency stem cell cardiomyocytes
- (2013) Elena Matsa et al. EUROPEAN HEART JOURNAL
- HRS/EHRA/APHRS Expert Consensus Statement on the Diagnosis and Management of Patients with Inherited Primary Arrhythmia Syndromes
- (2013) Silvia G. Priori et al. HEART RHYTHM
- Disease characterization using LQTS-specific induced pluripotent stem cells
- (2012) Toru Egashira et al. CARDIOVASCULAR RESEARCH
- Differentiation of Human Embryonic Stem Cells and Induced Pluripotent Stem Cells to Cardiomyocytes
- (2012) Christine L. Mummery et al. CIRCULATION RESEARCH
- Mutation-independent rescue of a novel mouse model of Retinitis Pigmentosa
- (2012) D L Greenwald et al. GENE THERAPY
- RNA interference targeting E637K mutation rescues hERG channel currents and restores its kinetic properties
- (2012) Xiaoli Lu et al. HEART RHYTHM
- HRS/EHRA Expert Consensus Statement on the State of Genetic Testing for the Channelopathies and Cardiomyopathies
- (2011) Michael J. Ackerman et al. HEART RHYTHM
- Implantable cardioverter defibrillator therapy for congenital long QT syndrome: A single-center experience
- (2010) Justin M. Horner et al. HEART RHYTHM
- Patient-Specific Induced Pluripotent Stem-Cell Models for Long-QT Syndrome
- (2010) Alessandra Moretti et al. NEW ENGLAND JOURNAL OF MEDICINE
- Prevalence of the Congenital Long-QT Syndrome
- (2009) Peter J. Schwartz et al. CIRCULATION
- Mutation in the S3 segment of KCNQ1 results in familial lone atrial fibrillation
- (2009) Saumya Das et al. HEART RHYTHM
- Adeno-Associated Virus Vector Genomes Persist as Episomal Chromatin in Primate Muscle
- (2008) M. Penaud-Budloo et al. JOURNAL OF VIROLOGY
- An intronic mutation leading to incomplete skipping of exon-2 in KCNQ1 rescues hearing in Jervell and Lange-Nielsen syndrome
- (2008) Zahurul A. Bhuiyan et al. PROGRESS IN BIOPHYSICS & MOLECULAR BIOLOGY
Find the ideal target journal for your manuscript
Explore over 38,000 international journals covering a vast array of academic fields.
SearchBecome a Peeref-certified reviewer
The Peeref Institute provides free reviewer training that teaches the core competencies of the academic peer review process.
Get Started