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。An evolved AAV variant enables efficient genetic engineering of murine T cells
William A. Nyberg et al.
CELL (2023)
Genetic Modification of the AAV5 Capsid with Lysine Residues Results in a Lung-Tropic Liver-Detargeted Gene Transfer Vector
Katie M. Stiles et al.
HUMAN GENE THERAPY (2022)
Addressing high dose AAV toxicity - 'one and done' or 'slower and lower'?
Takashi Kei Kishimoto et al.
EXPERT OPINION ON BIOLOGICAL THERAPY (2022)
Engineered AAVs for non-invasive gene delivery to rodent and non-human primate nervous systems
Xinhong Chen et al.
NEURON (2022)
Structural basis for the neurotropic AAV9 and the engineered AAVPHP.eB recognition with cellular receptors
Guangxue Xu et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2022)
Structural basis of receptor usage by the engineered capsid AAV-PHP.eB
Seongmin Jang et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2022)
Increasing the Specificity of AAV-Based Gene Editing through Self-Targeting and Short-Promoter Strategies
Camilo Breton et al.
MOLECULAR THERAPY (2021)
Gene therapy community grapples with toxicity issues, as pipeline matures
Asher Mullard
NATURE REVIEWS DRUG DISCOVERY (2021)
Long-term stable reduction of low-density lipoprotein in nonhuman primates following in vivo genome editing of PCSK9
Lili Wang et al.
MOLECULAR THERAPY (2021)
Rapid evolution of blood-brain-barrier-penetrating AAV capsids by RNA-driven biopanning
Mathieu Nonnenmacher et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2021)
Multivalent Affinity Profiling: Direct Visualization of the Superselective Binding of Influenza Viruses
Nico J. Overeem et al.
ACS NANO (2021)
Directed evolution of a family of AAV capsid variants enabling potent muscle-directed gene delivery across species
Mohammadsharif Tabebordbar et al.
CELL (2021)
Context-Specific Function of the Engineered Peptide Domain of PHP.B
R. Alexander Martino et al.
JOURNAL OF VIROLOGY (2021)
GPR108 Is a Highly Conserved AAV Entry Factor
Amanda M. Dudek et al.
MOLECULAR THERAPY (2020)
Single-Cell Transcriptome Atlas of Murine Endothelial Cells
Joanna Kalucka et al.
CELL (2020)
Ly6a Differential Expression in Blood-Brain Barrier Is Responsible for Strain Specific Central Nervous System Transduction Profile of AAV-PHP.B
Ana Rita Batista et al.
HUMAN GENE THERAPY (2020)
Multiplexed Cre-dependent selection yields systemic AAVs for targeting distinct brain cell types
Sripriya Ravindra Kumar et al.
NATURE METHODS (2020)
Broader Implications of Progressive Liver Dysfunction and Lethal Sepsis in Two Boys following Systemic High-Dose AAV
Leon Morales et al.
MOLECULAR THERAPY (2020)
MicroRNA-mediated inhibition of transgene expression reduces dorsal root ganglion toxicity by AAV vectors in primates
Juliette Hordeaux et al.
SCIENCE TRANSLATIONAL MEDICINE (2020)
Identification of a myotropic AAV by massively parallel in vivo evaluation of barcoded capsid variants
Jonas Weinmann et al.
NATURE COMMUNICATIONS (2020)
The GPI-Linked Protein LY6A Drives AAV-PHP.B Transport across the Blood-Brain Barrier
Juliette Hordeaux et al.
MOLECULAR THERAPY (2019)
Mobility-Based Quantification of Multivalent Virus-Receptor Interactions: New Insights Into Influenza A Virus Binding Mode
Matthias Mueller et al.
NANO LETTERS (2019)
Onasemnogene Abeparvovec: First Global Approval
Sheridan M. Hoy
DRUGS (2019)
Standard screening methods underreport AAV-mediated transduction and gene editing
Jonathan F. Lang et al.
NATURE COMMUNICATIONS (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)
EndoDB: a database of endothelial cell transcriptomics data
Shawez Khan et al.
NUCLEIC ACIDS RESEARCH (2019)
Selection of an Efficient AAV Vector for Robust CNS Transgene Expression
Killian S. Hanlon et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2019)
AVXS-101 (Onasemnogene Abeparvovec) for SMA 1: Comparative Study with a Prospective Natural History Cohort
Samiah A. Al-Zaidy et al.
JOURNAL OF NEUROMUSCULAR DISEASES (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)
The Neurotropic Properties of AAV-PHP.B Are Limited to C57BL/6J Mice
Juliette Hordeaux et al.
MOLECULAR THERAPY (2018)
Meganuclease targeting of PCSK9 in macaque liver leads to stable reduction in serum cholesterol
Lili Wang et al.
NATURE BIOTECHNOLOGY (2018)
Antibody affinity and valency impact brain uptake of transferrin receptor-targeted gold nanoparticles
Kasper Bendix Johnsen et al.
THERANOSTICS (2018)
A Rationally Engineered Capsid Variant of AAV9 for Systemic CNS-Directed and Peripheral Tissue-Detargeted Gene Delivery in Neonates
Dan Wang et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2018)
Synergistic and independent action of endogenous microRNAs 122a and 199a for post-transcriptional liver detargeting of gene vectors
Bijay Dhungel et al.
SCIENTIFIC REPORTS (2018)
Single-cell transcriptomics of 20 mouse organs creates a Tabula Muris
Nicholas Schaum et al.
NATURE (2018)
Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial
Stephen Russell et al.
LANCET (2017)
Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems
Ken Y. Chan et al.
NATURE NEUROSCIENCE (2017)
An essential receptor for adeno-associated virus infection
S. Pillay 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)
Organization, evolution and functions of the human and mouse Ly6/uPAR family genes
Chelsea L. Loughner et al.
HUMAN GENOMICS (2016)
Transferrin receptor (TfR) trafficking determines brain uptake of TfR antibody affinity variants
Nga Bien-Ly et al.
JOURNAL OF EXPERIMENTAL MEDICINE (2014)
Multiplexed immunohistochemistry, imaging, and quantitation: A review, with an assessment of Tyramide signal amplification, multispectral imaging and multiplex analysis
Edward C. Stack et al.
METHODS (2014)
Intramuscular Injection of AAV8 in Mice and Macaques Is Associated with Substantial Hepatic Targeting and Transgene Expression
Jenny A. Greig et al.
PLOS ONE (2014)
Receptor binding by a ferret-transmissible H5 avian influenza virus
Xiaoli Xiong et al.
NATURE (2013)
In Vivo-Directed Evolution of a New Adeno-Associated Virus for Therapeutic Outer Retinal Gene Delivery from the Vitreous
Deniz Dalkara et al.
SCIENCE TRANSLATIONAL MEDICINE (2013)
Receptor Concentration and Diffusivity Control Multivalent Binding of Sv40 to Membrane Bilayers
Oliwia M. Szklarczyk et al.
PLOS COMPUTATIONAL BIOLOGY (2013)
ALIPOGENE TIPARVOVEC FOR THE TREATMENT OF LIPOPROTEIN LIPASE DEFICIENCY
K. Haddley
DRUGS OF TODAY (2013)
Glycan Binding Avidity Determines the Systemic Fate of Adeno-Associated Virus Type 9
Shen Shen et al.
JOURNAL OF VIROLOGY (2012)
Liver-specific microRNA-122 target sequences incorporated in AAV vectors efficiently inhibits transgene expression in the liver
C. Qiao et al.
GENE THERAPY (2011)
Engineering Liver-detargeted AAV9 Vectors for Cardiac and Musculoskeletal Gene Transfer
Nagesh Pulicherla et al.
MOLECULAR THERAPY (2011)
Designing super selectivity in multivalent nano-particle binding
Francisco J. Martinez-Veracoechea et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2011)
Rapid, Simple, and Versatile Manufacturing of Recombinant Adeno-Associated Viral Vectors at Scale
Martin Lock et al.
HUMAN GENE THERAPY (2010)
Adeno-Associated Virus-Mediated Gene Transfer to Nonhuman Primate Liver Can Elicit Destructive Transgene-Specific T Cell Responses
Guangping Gao et al.
HUMAN GENE THERAPY (2009)
Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection
Carmela Zincarelli et al.
MOLECULAR THERAPY (2008)
Simultaneous visualization of multiple antigens with tyramide signal amplification using antibodies from the same species
Zsuzsanna E. Toth et al.
JOURNAL OF HISTOCHEMISTRY & CYTOCHEMISTRY (2007)
Analysis of tumors arising in male B6C3F1 mice with and without AAV vector delivery to liver
Peter Bell et al.
MOLECULAR THERAPY (2006)
Biology of AAV serotype vectors in liver-directed gene transfer to nonhuman primates
GP Gao et al.
MOLECULAR THERAPY (2006)
Random peptide libraries displayed on adeno-associated virus to select for targeted gene therapy vectors
OJ Müller et al.
NATURE BIOTECHNOLOGY (2003)
分享论文
