Current progress and limitations of AAV mediated delivery of protein therapeutic genes and the importance of developing quantitative pharmacokinetic/pharmacodynamic (PK/PD) models
Published 2021 View Full Article
- Home
- Publications
- Publication Search
- Publication Details
Title
Current progress and limitations of AAV mediated delivery of protein therapeutic genes and the importance of developing quantitative pharmacokinetic/pharmacodynamic (PK/PD) models
Authors
Keywords
AAV, Protein therapeutics, Gene therapy, Immunogenecity, PK/PD
Journal
ADVANCED DRUG DELIVERY REVIEWS
Volume -, Issue -, Pages -
Publisher
Elsevier BV
Online
2021-01-22
DOI
10.1016/j.addr.2021.01.017
References
Ask authors/readers for more resources
Related references
Note: Only part of the references are listed.- Gene Therapy for Glaucoma by Ciliary Body Aquaporin 1 Disruption Using CRISPR-Cas9
- (2020) Jiahui Wu et al. MOLECULAR THERAPY
- Engineering adeno-associated virus vectors for gene therapy
- (2020) Chengwen Li et al. NATURE REVIEWS GENETICS
- Capsid-specific removal of circulating antibodies to adeno-associated virus vectors
- (2020) Berangere Bertin et al. Scientific Reports
- Successful Transduction with AAV Vectors after Selective Depletion of Anti-AAV Antibodies by Immunoadsorption
- (2020) Alejandro Orlowski et al. Molecular Therapy-Methods & Clinical Development
- Brain Shuttle Neprilysin reduces central Amyloid-β levels
- (2020) Christopher R. Campos et al. PLoS One
- Positron emission tomography imaging of novel AAV capsids maps rapid brain accumulation
- (2020) Jai Woong Seo et al. Nature Communications
- Long-Term Delivery of an Anti-SIV Monoclonal Antibody With AAV
- (2020) José M. Martinez-Navio et al. Frontiers in Immunology
- Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy
- (2020) James M. Wilson et al. HUMAN GENE THERAPY
- Intramuscular Delivery of Gene Therapy for Targeting the Nervous System
- (2020) Andrew P. Tosolini et al. Frontiers in Molecular Neuroscience
- Intra-CSF AAV9 and AAVrh10 Administration in Nonhuman Primates: Promising Routes and Vectors for Which Neurological Diseases?
- (2020) Karim Bey et al. Molecular Therapy-Methods & Clinical Development
- Translational Feasibility of Lumbar Puncture for Intrathecal AAV Administration
- (2020) Christian Hinderer et al. Molecular Therapy-Methods & Clinical Development
- Transduction patterns in the CNS following various routes of AAV-5-mediated gene delivery
- (2020) K. L. Pietersz et al. GENE THERAPY
- Anti-drug antibody responses impair prophylaxis mediated by AAV-delivered HIV-1 broadly neutralizing antibodies
- (2019) Matthew R. Gardner et al. MOLECULAR THERAPY
- Adeno-Associated Virus Delivery of Anti-HIV Monoclonal Antibodies Can Drive Long-Term Virologic Suppression
- (2019) José M. Martinez-Navio et al. IMMUNITY
- A translational platform PBPK model for antibody disposition in the brain
- (2019) Hsueh-Yuan Chang et al. JOURNAL OF PHARMACOKINETICS AND PHARMACODYNAMICS
- Therapeutic hFIX activity achieved after single AAV5-hFIX treatment in hemophilia B patients and NHPs with pre-existing anti-AAV5 NABs
- (2019) Anna Majowicz et al. Molecular Therapy-Methods & Clinical Development
- Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here?
- (2019) Allison M. Keeler et al. Annual Review of Virology
- Threshold for Pre-existing Antibody Levels Limiting Transduction Efficiency of Systemic rAAV9 Gene Delivery: Relevance for Translation
- (2019) Aaron S. Meadows et al. Molecular Therapy-Methods & Clinical Development
- The Impact of Pre-existing Immunity on the Non-clinical Pharmacodynamics of AAV5-Based Gene Therapy
- (2019) Brian R. Long et al. Molecular Therapy-Methods & Clinical Development
- Neonatal brain-directed gene therapy rescues a mouse model of neurodegenerative CLN6 Batten disease
- (2019) Sophia-Martha kleine Holthaus et al. HUMAN MOLECULAR GENETICS
- AAV-PHP.B Administration Results in a Differential Pattern of CNS Biodistribution in Non-human Primates Compared with Mice
- (2019) William A. Liguore et al. MOLECULAR THERAPY
- Prevalence of Anti-Adeno-Associated Virus Immune Responses in International Cohorts of Healthy Donors
- (2019) Anita Kruzik et al. Molecular Therapy-Methods & Clinical Development
- Liver Gene Therapy: Reliable and Durable?
- (2019) Roland W. Herzog et al. MOLECULAR THERAPY
- Type I IFN Sensing by cDCs and CD4+ T Cell Help Are Both Requisite for Cross-Priming of AAV Capsid-Specific CD8+ T Cells
- (2019) Jamie L. Shirley et al. MOLECULAR THERAPY
- The Effect of CpG Sequences on Capsid-Specific CD8+ T Cell Responses to AAV Vector Gene Transfer
- (2019) ZhiQuan Xiang et al. MOLECULAR THERAPY
- Delivering genes across the blood-brain barrier: LY6A, a novel cellular receptor for AAV-PHP.B capsids
- (2019) Qin Huang et al. PLoS One
- Divergent engagements between adeno-associated viruses with their cellular receptor AAVR
- (2019) Ran Zhang et al. Nature Communications
- Gene delivery of a modified antibody to Aβ reduces progression of murine Alzheimer’s disease
- (2019) Bradford M. Elmer et al. PLoS One
- rAAV2-Retro Enables Extensive and High-Efficient Transduction of Lower Motor Neurons following Intramuscular Injection
- (2019) Zhilong Chen et al. Molecular Therapy-Methods & Clinical Development
- The role of brain barriers in fluid movement in the CNS: is there a ‘glymphatic’ system?
- (2018) N. Joan Abbott et al. ACTA NEUROPATHOLOGICA
- Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN
- (2018) Christian Hinderer et al. HUMAN GENE THERAPY
- Assessment of AAV Vector Tropisms for Mouse and Human Pluripotent Stem Cell–Derived RPE and Photoreceptor Cells
- (2018) Anai Gonzalez-Cordero et al. HUMAN GENE THERAPY
- Intramuscular Adeno-Associated Virus–Mediated Expression of Monoclonal Antibodies Provides 100% Protection Against Ebola Virus Infection in Mice
- (2018) Laura P van Lieshout et al. JOURNAL OF INFECTIOUS DISEASES
- Antibody pharmacokinetics in rat brain determined using microdialysis
- (2018) Hsueh-Yuan Chang et al. mAbs
- Mapping the Structural Determinants Required for AAVrh.10 Transport across the Blood-Brain Barrier
- (2018) Blake H. Albright et al. MOLECULAR THERAPY
- AAV8-antiVEGFfab Ocular Gene Transfer for Neovascular Age-Related Macular Degeneration
- (2018) Yuanyuan Liu et al. MOLECULAR THERAPY
- Systemic AAV micro-dystrophin gene therapy for Duchenne muscular dystrophy
- (2018) Dongsheng Duan MOLECULAR THERAPY
- In Vivo Selection of a Computationally Designed SCHEMA AAV Library Yields a Novel Variant for Infection of Adult Neural Stem Cells in the SVZ
- (2018) David S. Ojala et al. MOLECULAR THERAPY
- Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity
- (2018) Nicole K. Paulk et al. MOLECULAR THERAPY
- The Neurotropic Properties of AAV-PHP.B Are Limited to C57BL/6J Mice
- (2018) Juliette Hordeaux et al. MOLECULAR THERAPY
- Intravenous administration of the adeno-associated virus-PHP.B capsid fails to upregulate transduction efficiency in the marmoset brain
- (2018) Yasunori Matsuzaki et al. NEUROSCIENCE LETTERS
- AAV8 virions hijack serum proteins to increase hepatocyte binding for transduction enhancement
- (2018) Xiaolei Pei et al. VIROLOGY
- Nonclinical Safety Evaluation of scAAV8- RLBP1 for Treatment of RLBP1 Retinitis Pigmentosa
- (2018) Timothy K. MacLachlan et al. Molecular Therapy-Methods & Clinical Development
- Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Human Alpha-L-Iduronidase in Rhesus Macaques
- (2018) Juliette Hordeaux et al. Molecular Therapy-Methods & Clinical Development
- Emerging Issues in AAV-Mediated In Vivo Gene Therapy
- (2018) Pasqualina Colella et al. Molecular Therapy-Methods & Clinical Development
- Subcutaneous Administration of Biotherapeutics: An Overview of Current Challenges and Opportunities
- (2018) Beate Bittner et al. BIODRUGS
- Intrathecal viral vector delivery of trastuzumab prevents or inhibits tumor growth of human HER2-positive xenografts in mice
- (2018) William T Rothwell et al. CANCER RESEARCH
- Human Cardiac Gene Therapy
- (2018) Kiyotake Ishikawa et al. CIRCULATION RESEARCH
- Serotype survey of AAV gene delivery via subconjunctival injection in mice
- (2018) Liujiang Song et al. GENE THERAPY
- Anti-Amyloid-β Single-Chain Antibody Brain Delivery Via AAV Reduces Amyloid Load But May Increase Cerebral Hemorrhages in an Alzheimer's Disease Mouse Model
- (2018) Jinghong Kou et al. JOURNAL OF ALZHEIMERS DISEASE
- Gene therapy for neurological disorders: progress and prospects
- (2018) Benjamin E. Deverman et al. NATURE REVIEWS DRUG DISCOVERY
- Progress and potential in organoid research
- (2018) Giuliana Rossi et al. NATURE REVIEWS GENETICS
- AAV-8 and AAV-9 Vectors Cooperate with Serum Proteins Differently Than AAV-1 and AAV-6
- (2018) Jérôme Denard et al. Molecular Therapy-Methods & Clinical Development
- Update on clinical gene therapy for hemophilia
- (2018) George Q. Perrin et al. BLOOD
- Preclinical Evaluation of ADVM-022, a Novel Gene Therapy Approach to Treating Wet Age-Related Macular Degeneration
- (2018) Ruslan Grishanin et al. MOLECULAR THERAPY
- Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration
- (2018) Amine Meliani et al. Nature Communications
- Kinetics and MR-Based Monitoring of AAV9 Vector Delivery into Cerebrospinal Fluid of Nonhuman Primates
- (2018) Kousaku Ohno et al. Molecular Therapy-Methods & Clinical Development
- Manufacturing Clinical Grade Recombinant Adeno-Associated Virus Using Invertebrate Cell Lines
- (2017) Robert M. Kotin et al. HUMAN GENE THERAPY
- Unraveling the Complex Story of Immune Responses to AAV Vectors Trial After Trial
- (2017) Céline Vandamme et al. HUMAN GENE THERAPY
- Recombinant Adeno-Associated Viral Integration and Genotoxicity: Insights from Animal Models
- (2017) Randy J. Chandler et al. HUMAN GENE THERAPY
- Antibody gene transfer with adeno-associated viral vectors as a method for HIV prevention
- (2017) Jacqueline M. Brady et al. IMMUNOLOGICAL REVIEWS
- Intravitreous injection of AAV2-sFLT01 in patients with advanced neovascular age-related macular degeneration: a phase 1, open-label trial
- (2017) Jeffrey S Heier et al. LANCET
- AAV-PHP.B-Mediated Global-Scale Expression in the Mouse Nervous System Enables GBA1 Gene Therapy for Wide Protection from Synucleinopathy
- (2017) Giuseppe Morabito et al. MOLECULAR THERAPY
- Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems
- (2017) Ken Y Chan et al. NATURE NEUROSCIENCE
- Global Representations of Goal-Directed Behavior in Distinct Cell Types of Mouse Neocortex
- (2017) William E. Allen et al. NEURON
- Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
- (2017) Jerry R. Mendell et al. NEW ENGLAND JOURNAL OF MEDICINE
- Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant
- (2017) Lindsey A. George et al. NEW ENGLAND JOURNAL OF MEDICINE
- OneBac 2.0: Sf9 Cell Lines for Production of AAV1, AAV2, and AAV8 Vectors with Minimal Encapsidation of Foreign DNA
- (2017) Mario Mietzsch et al. Human Gene Therapy Methods
- Persistent Expression of Dopamine-Synthesizing Enzymes 15 Years After Gene Transfer in a Primate Model of Parkinson's Disease
- (2017) Yoshihide Sehara et al. Human Gene Therapy Clinical Development
- Regulatory T cells and TLR9 activation shape antibody formation to a secreted transgene product in AAV muscle gene transfer
- (2017) Roland W. Herzog et al. CELLULAR IMMUNOLOGY
- A brain microvasculature endothelial cell‐specific viral vector with the potential to treat neurovascular and neurological diseases
- (2016) Jakob Körbelin et al. EMBO Molecular Medicine
- Direct interaction of human serum proteins with AAV virions to enhance AAV transduction: immediate impact on clinical applications
- (2016) M Wang et al. GENE THERAPY
- Long-term metabolic correction of Wilson’s disease in a murine model by gene therapy
- (2016) Oihana Murillo et al. JOURNAL OF HEPATOLOGY
- Phase I open label liver-directed gene therapy clinical trial for acute intermittent porphyria
- (2016) Delia D’Avola et al. JOURNAL OF HEPATOLOGY
- Trafficking of adeno-associated virus vectors across a model of the blood-brain barrier; a comparative study of transcytosis and transduction using primary human brain endothelial cells
- (2016) Steven F. Merkel et al. JOURNAL OF NEUROCHEMISTRY
- Safety and Efficacy of Anti-Amyloid-β Immunotherapy in Alzheimer’s Disease: A Systematic Review and Meta-Analysis
- (2016) Ross Penninkilampi et al. Journal of Neuroimmune Pharmacology
- Characterization of the Adeno-Associated Virus 1 and 6 Sialic Acid Binding Site
- (2016) Lin-Ya Huang et al. JOURNAL OF VIROLOGY
- Calcium upregulation by percutaneous administration of gene therapy in patients with cardiac disease (CUPID 2): a randomised, multinational, double-blind, placebo-controlled, phase 2b trial
- (2016) Barry Greenberg et al. LANCET
- Antibodies to watch in 2017
- (2016) Janice M. Reichert mAbs
- Production of Recombinant Adeno-associated Virus Vectors Using Suspension HEK293 Cells and Continuous Harvest of Vector From the Culture Media for GMP FIX and FLT1 Clinical Vector
- (2016) Joshua C Grieger et al. MOLECULAR THERAPY
- Host Anti-antibody Responses Following Adeno-associated Virus–mediated Delivery of Antibodies Against HIV and SIV in Rhesus Monkeys
- (2016) José M Martinez-Navio et al. MOLECULAR THERAPY
- An essential receptor for adeno-associated virus infection
- (2016) S. Pillay et al. NATURE
- Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain
- (2016) Benjamin E Deverman et al. NATURE BIOTECHNOLOGY
- A Designer AAV Variant Permits Efficient Retrograde Access to Projection Neurons
- (2016) D. Gowanlock R. Tervo et al. NEURON
- AAV9-mediated central nervous system–targeted gene delivery via cisterna magna route in mice
- (2016) Vera Lukashchuk et al. Molecular Therapy-Methods & Clinical Development
- Impact of intravenous infusion time on AAV8 vector pharmacokinetics, safety, and liver transduction in cynomolgus macaques
- (2016) Jenny A Greig et al. Molecular Therapy-Methods & Clinical Development
- Perspectives on Cost and Value in Cancer Care
- (2016) Leonard B. Saltz JAMA Oncology
- Targeting Motor End Plates for Delivery of Adenoviruses: An Approach to Maximize Uptake and Transduction of Spinal Cord Motor Neurons
- (2016) Andrew Paul Tosolini et al. Scientific Reports
- Viral Vectors for Gene Therapy: Translational and Clinical Outlook
- (2015) Melissa A. Kotterman et al. Annual Review of Biomedical Engineering
- Synergy between rapamycin and FLT3 ligand enhances plasmacytoid dendritic cell-dependent induction of CD4+CD25+FoxP3+ Treg
- (2015) M. Biswas et al. BLOOD
- The use of combinations of monoclonal antibodies in clinical oncology
- (2015) Linda M. Henricks et al. CANCER TREATMENT REVIEWS
- Engineering the AAV capsid to optimize vector–host-interactions
- (2015) Hildegard Büning et al. CURRENT OPINION IN PHARMACOLOGY
- Adeno-Associated Virus at 50: A Golden Anniversary of Discovery, Research, and Gene Therapy Success—A Personal Perspective
- (2015) Eric Hastie et al. HUMAN GENE THERAPY
- Unique Roles of TLR9- and MyD88-Dependent and -Independent Pathways in Adaptive Immune Responses to AAV-Mediated Gene Transfer
- (2015) Geoffrey L. Rogers et al. Journal of Innate Immunity
- A Human Monoclonal IgG That Binds A Assemblies and Diverse Amyloids Exhibits Anti-Amyloid Activities In Vitro and In Vivo
- (2015) Y. Levites et al. JOURNAL OF NEUROSCIENCE
- Unique Glycan Signatures Regulate Adeno-Associated Virus Tropism in the Developing Brain
- (2015) Giridhar Murlidharan et al. JOURNAL OF VIROLOGY
- AAV-expressed eCD4-Ig provides durable protection from multiple SHIV challenges
- (2015) Matthew R. Gardner et al. NATURE
- Long-Term Effect of Gene Therapy on Leber’s Congenital Amaurosis
- (2015) James W.B. Bainbridge et al. NEW ENGLAND JOURNAL OF MEDICINE
- Improvement and Decline in Vision with Gene Therapy in Childhood Blindness
- (2015) Samuel G. Jacobson et al. NEW ENGLAND JOURNAL OF MEDICINE
- AAV gene transfer delays disease onset in a TPP1-deficient canine model of the late infantile form of Batten disease
- (2015) Martin L. Katz et al. Science Translational Medicine
- Stability and Compatibility of Recombinant Adeno-Associated Virus Under Conditions Commonly Encountered in Human Gene Therapy Trials
- (2015) Alisha M. Gruntman et al. Human Gene Therapy Methods
- Comparative Analysis of Cesium Chloride- and Iodixanol-Based Purification of Recombinant Adeno-Associated Viral Vectors for Preclinical Applications
- (2015) Benjamin Strobel et al. Human Gene Therapy Methods
- AAV-Delivered Antibody Mediates Significant Protective Effects against SIVmac239 Challenge in the Absence of Neutralizing Activity
- (2015) Sebastian P. Fuchs et al. PLoS Pathogens
- Optimized human factor IX expression cassettes for hepatic-directed gene therapy of hemophilia B
- (2015) Ru Zhang et al. Frontiers of Medicine
- AAV capsid CD8+ T-cell epitopes are highly conserved across AAV serotypes
- (2015) Daniel J Hui et al. Molecular Therapy-Methods & Clinical Development
- Adeno-associated viral serotypes produce differing titers and differentially transduce neurons within the rat basal and lateral amygdala
- (2014) Roopashri Holehonnur et al. BMC NEUROSCIENCE
- Subcutaneous Administration of Monoclonal Antibodies in Oncology
- (2014) C. Jackisch et al. GEBURTSHILFE UND FRAUENHEILKUNDE
- Neutralizing antibodies against AAV2, AAV5 and AAV8 in healthy and HIV-1-infected subjects in China: implications for gene therapy using AAV vectors
- (2014) Q Liu et al. GENE THERAPY
- The therapeutic monoclonal antibody market
- (2014) Dawn M Ecker et al. mAbs
- HIV immunity goes direct
- (2014) NATURE BIOTECHNOLOGY
- The special case of gene therapy pricing
- (2014) Troyen A Brennan et al. NATURE BIOTECHNOLOGY
- Engineering adeno-associated viruses for clinical gene therapy
- (2014) Melissa A. Kotterman et al. NATURE REVIEWS GENETICS
- Non-viral vectors for gene-based therapy
- (2014) Hao Yin et al. NATURE REVIEWS GENETICS
- Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B
- (2014) Amit C. Nathwani et al. NEW ENGLAND JOURNAL OF MEDICINE
- Therapeutic bispecific antibodies cross the blood-brain barrier in nonhuman primates
- (2014) Y. J. Yu et al. Science Translational Medicine
- Drawing a high-resolution functional map of adeno-associated virus capsid by massively parallel sequencing
- (2014) Kei Adachi et al. Nature Communications
- Biodistribution of adeno-associated virus serotype 9 (AAV9) vector after intrathecal and intravenous delivery in mouse
- (2014) Daniel J. Schuster et al. Frontiers in Neuroanatomy
- Comparative Efficacy and Safety of Multiple Routes of Direct CNS Administration of Adeno-Associated Virus Gene Transfer Vector Serotype rh.10 Expressing the Human Arylsulfatase A cDNA to Nonhuman Primates
- (2014) Jonathan B. Rosenberg et al. Human Gene Therapy Clinical Development
- Widespread gene transfer in the central nervous system of cynomolgus macaques following delivery of AAV9 into the cisterna magna
- (2014) Christian Hinderer et al. Molecular Therapy-Methods & Clinical Development
- Characterization of Interactions between Heparin/Glycosaminoglycan and Adeno-Associated Virus
- (2013) Fuming Zhang et al. BIOCHEMISTRY
- Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells
- (2013) A. T. Martino et al. BLOOD
- Vectored Expression of the Broadly Neutralizing Antibody FI6 in Mouse Airway Provides Partial Protection against a New Avian Influenza A Virus, H7N9
- (2013) Maria P. Limberis et al. Clinical and Vaccine Immunology
- Gene therapy enters the pharma market: The short story of a long journey
- (2013) Hildegard Büning EMBO Molecular Medicine
- Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates
- (2013) S J Gray et al. GENE THERAPY
- Kinetics of Adeno-Associated Virus Serotype 2 (AAV2) and AAV8 Capsid Antigen Presentation In Vivo Are Identical
- (2013) Yi He et al. HUMAN GENE THERAPY
- OneBac: Platform for Scalable and High-Titer Production of Adeno-Associated Virus Serotype 1–12 Vectors for Gene Therapy
- (2013) Mario Mietzsch et al. HUMAN GENE THERAPY
- Immune Responses to Intramuscular Administration of Alipogene Tiparvovec (AAV1-LPLS447X) in a Phase II Clinical Trial of Lipoprotein Lipase Deficiency Gene Therapy
- (2013) Valerie Ferreira et al. HUMAN GENE THERAPY
- Human Treg responses allow sustained recombinant adeno-associated virus–mediated transgene expression
- (2013) Christian Mueller et al. JOURNAL OF CLINICAL INVESTIGATION
- CpG-depleted adeno-associated virus vectors evade immune detection
- (2013) Susan M. Faust et al. JOURNAL OF CLINICAL INVESTIGATION
- Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy
- (2013) Virginia Haurigot et al. JOURNAL OF CLINICAL INVESTIGATION
- The prevalence of neutralizing antibodies against adeno-associated virus capsids is reduced in young Japanese individuals
- (2013) Jun Mimuro et al. JOURNAL OF MEDICAL VIROLOGY
- C-Reactive Protein (CRP) Is Essential for Efficient Systemic Transduction of Recombinant Adeno-Associated Virus Vector 1 (rAAV-1) and rAAV-6 in Mice
- (2013) J. Denard et al. JOURNAL OF VIROLOGY
- Intramuscular scAAV9-SMN Injection Mediates Widespread Gene Delivery to the Spinal Cord and Decreases Disease Severity in SMA Mice
- (2013) Sofia Benkhelifa-Ziyyat et al. MOLECULAR THERAPY
- Selection and evaluation of clinically relevant AAV variants in a xenograft liver model
- (2013) Leszek Lisowski et al. NATURE
- Photoreceptor precursors derived from three-dimensional embryonic stem cell cultures integrate and mature within adult degenerate retina
- (2013) Anai Gonzalez-Cordero et al. NATURE BIOTECHNOLOGY
- In Vivo-Directed Evolution of a New Adeno-Associated Virus for Therapeutic Outer Retinal Gene Delivery from the Vitreous
- (2013) D. Dalkara et al. Science Translational Medicine
- Intranasal Antibody Gene Transfer in Mice and Ferrets Elicits Broad Protection Against Pandemic Influenza
- (2013) M. P. Limberis et al. Science Translational Medicine
- A survey of ex vivo/in vitro transduction efficiency of mammalian primary cells and cell lines with Nine natural adeno-associated virus (AAV1-9) and one engineered adeno-associated virus serotype
- (2013) Brian L Ellis et al. Virology Journal
- Pre-existing Anti–Adeno-Associated Virus Antibodies as a Challenge in AAV Gene Therapy
- (2013) Vedell Louis Jeune et al. Human Gene Therapy Methods
- Protein Coexpression Using FMDV 2A: Effect of “Linker” Residues
- (2013) Ekaterina Minskaia et al. Biomed Research International
- Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer
- (2012) G. Buchlis et al. BLOOD
- AAVrh.10-Mediated Expression of an Anti-Cocaine Antibody Mediates Persistent Passive Immunization That Suppresses Cocaine-Induced Behavior
- (2012) Jonathan B. Rosenberg et al. HUMAN GENE THERAPY
- Long-Term Evaluation of a Phase 1 Study of AADC Gene Therapy for Parkinson's Disease
- (2012) Gabriele Mittermeyer et al. HUMAN GENE THERAPY
- Structural Insight into the Unique Properties of Adeno-Associated Virus Serotype 9
- (2012) M. A. DiMattia et al. JOURNAL OF VIROLOGY
- Identification of the Galactose Binding Domain of the Adeno-Associated Virus Serotype 9 Capsid
- (2012) C. L. Bell et al. JOURNAL OF VIROLOGY
- Sialic Acid Deposition Impairs the Utility of AAV9, but Not Peptide-modified AAVs for Brain Gene Therapy in a Mouse Model of Lysosomal Storage Disease
- (2012) Yong Hong Chen et al. MOLECULAR THERAPY
- Endgame: Glybera Finally Recommended for Approval as the First Gene Therapy Drug in the European Union
- (2012) Seppo Ylä-Herttuala MOLECULAR THERAPY
- Displaying High-affinity Ligands on Adeno-associated Viral Vectors Enables Tumor Cell-specific and Safe Gene Transfer
- (2012) Robert C Münch et al. MOLECULAR THERAPY
- AAV-Directed Persistent Expression of a Gene Encoding Anti-Nicotine Antibody for Smoking Cessation
- (2012) M. J. Hicks et al. Science Translational Medicine
- Long-Term Follow-Up After Gene Therapy for Canavan Disease
- (2012) P. Leone et al. Science Translational Medicine
- Gene Therapy for Aromatic L-Amino Acid Decarboxylase Deficiency
- (2012) W.-L. Hwu et al. Science Translational Medicine
- Monoclonal antibodies: magic bullets with a hefty price tag
- (2012) A. F. Shaughnessy BMJ-British Medical Journal
- 2A to the Fore – Research, Technology and Applications
- (2011) Garry A. Luke et al. Biotechnology & Genetic Engineering Reviews
- Novel random peptide libraries displayed on AAV serotype 9 for selection of endothelial cell-directed gene transfer vectors
- (2011) K Varadi et al. GENE THERAPY
- Robust spinal motor neuron transduction following intrathecal delivery of AAV9 in pigs
- (2011) T Federici et al. GENE THERAPY
- Schwann cell targeting via intrasciatic injection of AAV8 as gene therapy strategy for peripheral nerve regeneration
- (2011) J Homs et al. GENE THERAPY
- Optimizing Promoters for Recombinant Adeno-Associated Virus-Mediated Gene Expression in the Peripheral and Central Nervous System Using Self-Complementary Vectors
- (2011) Steven J. Gray et al. HUMAN GENE THERAPY
- Phase 2 Clinical Trial of a Recombinant Adeno-Associated Viral Vector Expressing α1-Antitrypsin: Interim Results
- (2011) Terence R. Flotte et al. HUMAN GENE THERAPY
- Adeno-Associated Virus Serotype 9 Transduction in the Central Nervous System of Nonhuman Primates
- (2011) Lluis Samaranch et al. HUMAN GENE THERAPY
- Large-scale recombinant adeno-associated virus production
- (2011) R. M. Kotin HUMAN MOLECULAR GENETICS
- TerminalN-Linked Galactose Is the Primary Receptor for Adeno-associated Virus 9
- (2011) Shen Shen et al. JOURNAL OF BIOLOGICAL CHEMISTRY
- Towards a platform PBPK model to characterize the plasma and tissue disposition of monoclonal antibodies in preclinical species and human
- (2011) Dhaval K. Shah et al. JOURNAL OF PHARMACOKINETICS AND PHARMACODYNAMICS
- Preclinical Differences of Intravascular AAV9 Delivery to Neurons and Glia: A Comparative Study of Adult Mice and Nonhuman Primates
- (2011) Steven J Gray et al. MOLECULAR THERAPY
- Enhancers of Adeno-associated Virus AAV2 Transduction via High Throughput siRNA Screening
- (2011) Alexis J Wallen et al. MOLECULAR THERAPY
- AAV Mediated GDNF Secretion From Retinal Glia Slows Down Retinal Degeneration in a Rat Model of Retinitis Pigmentosa
- (2011) Deniz Dalkara et al. MOLECULAR THERAPY
- An Evolved Adeno-associated Viral Variant Enhances Gene Delivery and Gene Targeting in Neural Stem Cells
- (2011) Jae-Hyung Jang et al. MOLECULAR THERAPY
- A Potential Role of Distinctively Delayed Blood Clearance of Recombinant Adeno-associated Virus Serotype 9 in Robust Cardiac Transduction
- (2011) Nicole M Kotchey et al. MOLECULAR THERAPY
- Engineering Liver-detargeted AAV9 Vectors for Cardiac and Musculoskeletal Gene Transfer
- (2011) Nagesh Pulicherla et al. MOLECULAR THERAPY
- Systemic Gene Delivery in Large Species for Targeting Spinal Cord, Brain, and Peripheral Tissues for Pediatric Disorders
- (2011) Adam K Bevan et al. MOLECULAR THERAPY
- Directed Evolution of Adeno-associated Virus for Enhanced Gene Delivery and Gene Targeting in Human Pluripotent Stem Cells
- (2011) Prashanth Asuri et al. MOLECULAR THERAPY
- Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector
- (2011) Dawn E Bowles et al. MOLECULAR THERAPY
- Antibody-based protection against HIV infection by vectored immunoprophylaxis
- (2011) Alejandro B. Balazs et al. NATURE
- Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B
- (2011) Amit C. Nathwani et al. NEW ENGLAND JOURNAL OF MEDICINE
- Prevention and Reversal of Antibody Responses Against Factor IX in Gene Therapy for Hemophilia B
- (2011) Sushrusha Nayak et al. Frontiers in Microbiology
- Intramuscular delivery of a single chain antibody gene prevents brain Aβ deposition and cognitive impairment in a mouse model of Alzheimer’s disease
- (2010) Yan-Jiang Wang et al. BRAIN BEHAVIOR AND IMMUNITY
- Prevalence of Serum IgG and Neutralizing Factors Against Adeno-Associated Virus (AAV) Types 1, 2, 5, 6, 8, and 9 in the Healthy Population: Implications for Gene Therapy Using AAV Vectors
- (2010) Sylvie Boutin et al. HUMAN GENE THERAPY
- Industrialization of mAb production technology: The bioprocessing industry at a crossroads
- (2010) Brian Kelley mAbs
- Aβ-directed Single-chain Antibody Delivery Via a Serotype-1 AAV Vector Improves Learning Behavior and Pathology in Alzheimer's Disease Mice
- (2010) Deborah A Ryan et al. MOLECULAR THERAPY
- Widespread Muscle Expression of an AAV9 Human Mini-dystrophin Vector After Intravenous Injection in Neonatal Dystrophin-deficient Dogs
- (2010) Joe N Kornegay et al. MOLECULAR THERAPY
- Preclinical Safety Evaluation of AAV2-sFLT01— A Gene Therapy for Age-related Macular Degeneration
- (2010) Timothy K MacLachlan et al. MOLECULAR THERAPY
- Dystrophin Immunity in Duchenne's Muscular Dystrophy
- (2010) Jerry R. Mendell et al. NEW ENGLAND JOURNAL OF MEDICINE
- BAAV Transcytosis Requires an Interaction with β-1-4 Linked- Glucosamine and gp96
- (2010) Giovanni Di Pasquale et al. PLoS One
- Systemic Delivery of scAAV9 Expressing SMN Prolongs Survival in a Model of Spinal Muscular Atrophy
- (2010) C. F. Valori et al. Science Translational Medicine
- Mathematical model of the rate-limiting steps for retrovirus-mediated gene transfer into mammalian cells
- (2009) Venkata S. Tayi et al. BIOTECHNOLOGY AND BIOENGINEERING
- Mathematical Modeling of Herpes Simplex Virus Distribution in Solid Tumors: Implications for Cancer Gene Therapy
- (2009) W. Mok et al. CLINICAL CANCER RESEARCH
- Retroviral bicistronic vectors
- (2009) W. Pfützner DRUG NEWS & PERSPECTIVES
- Semi-mechanistic pharmacodynamic modelling of gene expression and silencing processes
- (2009) Pedro Berraondo et al. EUROPEAN JOURNAL OF PHARMACEUTICAL SCIENCES
- Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors
- (2009) Gary C. Pien et al. JOURNAL OF CLINICAL INVESTIGATION
- The TLR9-MyD88 pathway is critical for adaptive immune responses to adeno-associated virus gene therapy vectors in mice
- (2009) Jiangao Zhu et al. JOURNAL OF CLINICAL INVESTIGATION
- Worldwide Epidemiology of Neutralizing Antibodies to Adeno‐Associated Viruses
- (2009) Roberto Calcedo et al. JOURNAL OF INFECTIOUS DISEASES
- Generating Differentially Targeted Amyloid-β Specific Intrabodies as a Passive Vaccination Strategy for Alzheimer's Disease
- (2009) Kelly L Sudol et al. MOLECULAR THERAPY
- Strategies to Modulate Immune Responses: A New Frontier for Gene Therapy
- (2009) Valder R Arruda et al. MOLECULAR THERAPY
- Vector-mediated gene transfer engenders long-lived neutralizing activity and protection against SIV infection in monkeys
- (2009) Philip R Johnson et al. NATURE MEDICINE
- Structural and functional bases for broad-spectrum neutralization of avian and human influenza A viruses
- (2009) Jianhua Sui et al. NATURE STRUCTURAL & MOLECULAR BIOLOGY
- A Novel Adeno-Associated Viral Variant for Efficient and Selective Intravitreal Transduction of Rat Müller Cells
- (2009) Ryan R. Klimczak et al. PLoS One
- Directed evolution of adeno-associated virus to an infectious respiratory virus
- (2009) K. J. D. A. Excoffon et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- PK/PD modelling and simulations: utility in drug development
- (2008) Iris Rajman DRUG DISCOVERY TODAY
- Bidirectional promoter interference between two widely used internal heterologous promoters in a late-generation lentiviral construct
- (2008) J A Curtin et al. GENE THERAPY
- Gene therapy progress and prospects: synthetic polymer-based systems
- (2008) D Schaffert et al. GENE THERAPY
- Construction and analysis of compact muscle-specific promoters for AAV vectors
- (2008) B Wang et al. GENE THERAPY
- Development of optimal bicistronic lentiviral vectors facilitates high-level TCR gene expression and robust tumor cell recognition
- (2008) S Yang et al. GENE THERAPY
- Non-linear pharmacodynamics in the transfection efficiency of a non-viral gene delivery system
- (2008) Rumiko Moriguchi et al. INTERNATIONAL JOURNAL OF PHARMACEUTICS
- Novel AAV serotypes for improved ocular gene transfer
- (2008) Corinna Lebherz et al. JOURNAL OF GENE MEDICINE
- Heparin binding induces conformational changes in Adeno-associated virus serotype 2
- (2008) Hazel C. Levy et al. JOURNAL OF STRUCTURAL BIOLOGY
- In Vitro and In Vivo Gene Therapy Vector Evolution via Multispecies Interbreeding and Retargeting of Adeno-Associated Viruses
- (2008) D. Grimm et al. JOURNAL OF VIROLOGY
- Adeno-Associated Virus Vector Genomes Persist as Episomal Chromatin in Primate Muscle
- (2008) M. Penaud-Budloo et al. JOURNAL OF VIROLOGY
- Expanded Repertoire of AAV Vector Serotypes Mediate Unique Patterns of Transduction in Mouse Brain
- (2008) Cassia N Cearley et al. MOLECULAR THERAPY
- Engineering and Selection of Shuffled AAV Genomes: A New Strategy for Producing Targeted Biological Nanoparticles
- (2008) Wuping Li et al. MOLECULAR THERAPY
- Self-complementary AAV Vectors; Advances and Applications
- (2008) Douglas M McCarty MOLECULAR THERAPY
- Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection
- (2008) Carmela Zincarelli et al. MOLECULAR THERAPY
- PK/PD Modelling and Beyond: Impact on Drug Development
- (2008) Douwe D. Breimer PHARMACEUTICAL RESEARCH
- Heterosubtypic Neutralizing Monoclonal Antibodies Cross-Protective against H5N1 and H1N1 Recovered from Human IgM+ Memory B Cells
- (2008) Mark Throsby et al. PLoS One
- Genetic delivery of an anti-RSV antibody to protect against pulmonary infection with RSV
- (2008) Davor Skaricic et al. VIROLOGY
- AAV8, 9, Rh10, Rh43 Vector Gene Transfer in the Rat Brain: Effects of Serotype, Promoter and Purification Method
- (2007) Ronald L Klein et al. MOLECULAR THERAPY
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