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。Deep diversification of an AAV capsid protein by machine learning
Drew H. Bryant et al.
NATURE BIOTECHNOLOGY (2021)
A mouse model for SARS-CoV-2-induced acute respiratory distress syndrome
Weiqi Hong et al.
SIGNAL TRANSDUCTION AND TARGETED THERAPY (2021)
The promise and challenge of therapeutic genome editing
Jennifer A. Doudna
NATURE (2020)
Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses
Jonathan M. Levy et al.
NATURE BIOMEDICAL ENGINEERING (2020)
Management of Neuroinflammatory Responses to AAV-Mediated Gene Therapies for Neurodegenerative Diseases
Barbara A. Perez et al.
BRAIN SCIENCES (2020)
CRISPR-Based Therapeutic Genome Editing: Strategies and In Vivo Delivery by AAV Vectors
Dan Wang et al.
CELL (2020)
Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy
James M. Wilson et al.
HUMAN GENE THERAPY (2020)
Genome editing with CRISPR-Cas nucleases, base editors, transposases and prime editors
Andrew V. Anzalone et al.
NATURE BIOTECHNOLOGY (2020)
In Vivo Repair of a Protein Underlying a Neurological Disorder by Programmable RNA Editing
John R. Sinnamon et al.
CELL REPORTS (2020)
Base editing: advances and therapeutic opportunities
Elizabeth M. Porto et al.
NATURE REVIEWS DRUG DISCOVERY (2020)
Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy
Christopher E. Nelson et al.
NATURE MEDICINE (2019)
Identification of preexisting adaptive immunity to Cas9 proteins in humans
Carsten T. Charlesworth et al.
NATURE MEDICINE (2019)
Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality
Eloise Hudry et al.
NEURON (2019)
The Autism and Angelman Syndrome Protein Ube3A/E6AP: The Gene, E3 Ligase Ubiquitination Targets and Neurobiological Functions
Natasha Khatri et al.
FRONTIERS IN MOLECULAR NEUROSCIENCE (2019)
Search-and-replace genome editing without double-strand breaks or donor DNA
Andrew V. Anzalone et al.
NATURE (2019)
Delivering genes across the blood-brain barrier: LY6A, a novel cellular receptor for AAV-PHP.B capsids
Qin Huang et al.
PLOS ONE (2019)
High levels of AAV vector integration into CRISPR-induced DNA breaks
Killian S. Hanlon et al.
NATURE COMMUNICATIONS (2019)
Target-Specific Precision of CRISPR-Mediated Genome Editing
Anob M. Chakrabarti et al.
MOLECULAR CELL (2019)
Predicting the mutations generated by repair of Cas9-induced double-strand breaks
Felicity Allen et al.
NATURE BIOTECHNOLOGY (2019)
High prevalence of Streptococcus pyogenes Cas9-reactive T cells within the adult human population
Dimitrios L. Wagner et al.
NATURE MEDICINE (2019)
Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN
Christian Hinderer et al.
HUMAN GENE THERAPY (2018)
Gene therapy clinical trials worldwide to 2017: An update
Samantha L. Ginn et al.
JOURNAL OF GENE MEDICINE (2018)
The Neurotropic Properties of AAV-PHP.B Are Limited to C57BL/6J Mice
Juliette Hordeaux et al.
MOLECULAR THERAPY (2018)
A perspective on cure for Rett syndrome
Angus John Clarke et al.
ORPHANET JOURNAL OF RARE DISEASES (2018)
Nanoparticle delivery of CRISPR into the brain rescues a mouse model of fragile X syndrome from exaggerated repetitive behaviours
Bumwhee Lee et al.
NATURE BIOMEDICAL ENGINEERING (2018)
Predictable and precise template-free CRISPR editing of pathogenic variants
Max W. Shen et al.
NATURE (2018)
Prevalence and architecture of de novo mutations in developmental disorders
Jeremy F. McRae et al.
NATURE (2017)
Programmable base editing of A.T to G.C in genomic DNA without DNA cleavage
Nicole M. Gaudelli et al.
NATURE (2017)
Efficient genome editing in the mouse brain by local delivery of engineered Cas9 ribonucleoprotein complexes
Brett T. Staahl et al.
NATURE BIOTECHNOLOGY (2017)
Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems
Ken Y. Chan et al.
NATURE NEUROSCIENCE (2017)
Non-homologous DNA end joining and alternative pathways to double-strand break repair
Howard H. Y. Chang et al.
NATURE REVIEWS MOLECULAR CELL BIOLOGY (2017)
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
J. R. Mendell et al.
NEW ENGLAND JOURNAL OF MEDICINE (2017)
RNA editing with CRISPR-Cas13
David B. T. Cox et al.
SCIENCE (2017)
Persistent Expression of Dopamine-Synthesizing Enzymes 15 Years After Gene Transfer in a Primate Model of Parkinson's Disease
Yoshihide Sehara et al.
HUMAN GENE THERAPY CLINICAL DEVELOPMENT (2017)
Improved MECP2 Gene Therapy Extends the Survival of MeCP2-Null Mice without Apparent Toxicity after Intracisternal Delivery
Sarah E. Sinnett et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2017)
Development of a Novel AAV Gene Therapy Cassette with Improved Safety Features and Efficacy in a Mouse Model of Rett Syndrome
Kamal K. E. Gadalla et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2017)
DNA Repair Profiling Reveals Nonrandom Outcomes at Cas9-Mediated Breaks
Megan van Overbeek et al.
MOLECULAR CELL (2016)
Efficient introduction of specific homozygous and heterozygous mutations using CRISPR/Cas9
Dominik Paquet et al.
NATURE (2016)
Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage
Alexis C. Komor et al.
NATURE (2016)
Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain
Benjamin E. Deverman et al.
NATURE BIOTECHNOLOGY (2016)
Repair Pathway Choices and Consequences at the Double-Strand Break
Raphael Ceccaldi et al.
TRENDS IN CELL BIOLOGY (2016)
Minimizing off-Target Mutagenesis Risks Caused by Programmable Nucleases
Kentaro Ishida et al.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2015)
Ube3a reinstatement identifies distinct developmental windows in a murine Angelman syndrome model
Sara Silva-Santos et al.
JOURNAL OF CLINICAL INVESTIGATION (2015)
Therapeutic genome editing: prospects and challenges
David Benjamin Turitz Cox et al.
NATURE MEDICINE (2015)
Large-scale discovery of novel genetic causes of developmental disorders
T. W. Fitzgerald et al.
NATURE (2015)
Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo
John A. Zuris et al.
NATURE BIOTECHNOLOGY (2015)
Robust ZFN-mediated genome editing in adult hemophilic mice
Xavier M. Anguela et al.
BLOOD (2013)
Immune responses to AAV vectors: overcoming barriers to successful gene therapy
Federico Mingozzi et al.
BLOOD (2013)
Improved Survival and Reduced Phenotypic Severity Following AAV9/MECP2 Gene Transfer to Neonatal and Juvenile Male Mecp2 Knockout Mice
Kamal K. E. Gadalla et al.
MOLECULAR THERAPY (2013)
Correction of mutations within the cystic fibrosis transmembrane conductance regulator by site-directed RNA editing
Maria Fernanda Montiel-Gonzalez et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2013)
Multiplex Genome Engineering Using CRISPR/Cas Systems
Le Cong et al.
SCIENCE (2013)
Morphological and functional reversal of phenotypes in a mouse model of Rett syndrome
Lianne Robinson et al.
BRAIN (2012)
Long-Term Evaluation of a Phase 1 Study of AADC Gene Therapy for Parkinson's Disease
Gabriele Mittermeyer et al.
HUMAN GENE THERAPY (2012)
Cas9-crRNA ribonucleoprotein complex mediates specific DNA cleavage for adaptive immunity in bacteria
Giedrius Gasiunas et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2012)
A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity
Martin Jinek et al.
SCIENCE (2012)
State-of-the-art gene-based therapies: the road ahead
Mark A. Kay
NATURE REVIEWS GENETICS (2011)
Worldwide Epidemiology of Neutralizing Antibodies to Adeno-Associated Viruses
Roberto Calcedo et al.
JOURNAL OF INFECTIOUS DISEASES (2009)
Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes
Kevin D. Foust et al.
NATURE BIOTECHNOLOGY (2009)
Comparison of nonhomologous end joining and homologous recombination in human cells
Zhiyong Mao et al.
DNA REPAIR (2008)
Reversal of neurological defects in a mouse model of Rett syndrome
Jacky Guy et al.
SCIENCE (2007)
Duplication of the MECP2 region is a frequent cause of severe mental retardation and progressive neurological symptoms in males
H Van Esch et al.
AMERICAN JOURNAL OF HUMAN GENETICS (2005)
Expression of MeCP2 in postmitotic neurons rescues Rett syndrome in mice
S Luikenhuis et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2004)
Adeno-associated virus vectors integrate at chromosome breakage sites
DG Miller et al.
NATURE GENETICS (2004)
Mild overexpression of MeCP2 causes a progressive neurological disorder in mice
AL Collins et al.
HUMAN MOLECULAR GENETICS (2004)
Pathways of DNA double-strand break repair during the mammalian cell cycle
K Rothkamm et al.
MOLECULAR AND CELLULAR BIOLOGY (2003)
Human gene targeting by adeno-associated virus vectors is enhanced by DNA double-strand breaks
DG Miller et al.
MOLECULAR AND CELLULAR BIOLOGY (2003)
Non-homologous DNA end joining in the mature rat brain
KQ Ren et al.
JOURNAL OF NEUROCHEMISTRY (2002)
A mouse Mecp2-null mutation causes neurological symptoms that mimic Rett syndrome
J Guy et al.
NATURE GENETICS (2001)
Deficiency of methyl-CpG binding protein-2 in CNS neurons results in a Rett-like phenotype in mice
RZ Chen et al.
NATURE GENETICS (2001)
分享论文
