Clinical Pharmacology Considerations on Recombinant Adeno‐Associated Virus–Based Gene Therapy
出版年份 2022 全文链接
标题
Clinical Pharmacology Considerations on Recombinant Adeno‐Associated Virus–Based Gene Therapy
作者
关键词
-
出版物
JOURNAL OF CLINICAL PHARMACOLOGY
Volume 62, Issue S2, Pages -
出版商
Wiley
发表日期
2022-12-03
DOI
10.1002/jcph.2141
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。- The Perspective of DMPK on Recombinant Adeno-Associated Virus-Based Gene Therapy: Past Learning, Current Support, and Future Contribution
- (2022) Nancy Chen et al. AAPS Journal
- Interindividual variability in transgene mRNA and protein production following adeno-associated virus gene therapy for hemophilia A
- (2022) Sylvia Fong et al. NATURE MEDICINE
- Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A
- (2022) Margareth C. Ozelo et al. NEW ENGLAND JOURNAL OF MEDICINE
- Circulating neurofilaments to track dorsal root ganglion toxicity risks with AAV-mediated gene therapy
- (2022) Michelle A. Farrar et al. Molecular Therapy-Methods & Clinical Development
- The clinical landscape for AAV gene therapies
- (2021) Dmitry A. Kuzmin et al. NATURE REVIEWS DRUG DISCOVERY
- Combination of serum and CSF neurofilament-light and neuroinflammatory biomarkers to evaluate ALS
- (2021) Alexandre Brodovitch et al. Scientific Reports
- Engineering adeno-associated viral vectors to evade innate immune and inflammatory responses
- (2021) Ying Kai Chan et al. Science Translational Medicine
- Rational Clinical Dose Selection of Adeno‐Associated Virus‐Mediated Gene Therapy Based on Allometric Principles
- (2021) Fei Tang et al. CLINICAL PHARMACOLOGY & THERAPEUTICS
- Current and Next Steps Toward Prediction of Human Dose for Gene Therapy Using Translational Dose‐Response Studies
- (2021) Sergey Aksenov et al. CLINICAL PHARMACOLOGY & THERAPEUTICS
- Plasma neurofilament light in Huntington's disease: A marker for disease onset, but not symptom progression
- (2021) Georgia M. Parkin et al. PARKINSONISM & RELATED DISORDERS
- Viral Vector‐Based Gene Therapies in the Clinic
- (2021) Zongmin Zhao et al. Bioengineering & Translational Medicine
- Biodistribution of onasemnogene abeparvovec DNA, mRNA and SMN protein in human tissue
- (2021) Gretchen Thomsen et al. NATURE MEDICINE
- Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A
- (2021) Lindsey A. George et al. NEW ENGLAND JOURNAL OF MEDICINE
- Codon Modification and PAMPs in Clinical AAV Vectors: The Tortoise or the Hare?
- (2020) J. Fraser Wright MOLECULAR THERAPY
- Engineering adeno-associated virus vectors for gene therapy
- (2020) Chengwen Li et al. NATURE REVIEWS GENETICS
- Intermittent Starvation Extends the Functional Lifetime of Primary Human Hepatocyte Cultures
- (2020) Matthew D Davidson et al. TOXICOLOGICAL SCIENCES
- Successful Transduction with AAV Vectors after Selective Depletion of Anti-AAV Antibodies by Immunoadsorption
- (2020) Alejandro Orlowski et al. Molecular Therapy-Methods & Clinical Development
- Human Immune Responses to Adeno-Associated Virus (AAV) Vectors
- (2020) Giuseppe Ronzitti et al. Frontiers in Immunology
- AAV-BASED GENE THERAPY FOR LIFE-LONG CORRECTION OF GENETIC DISEASE
- (2020) Christian Brommel et al. HUMAN GENE THERAPY
- Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B
- (2020) Lindsey A. George et al. MOLECULAR THERAPY
- Quantification of CpG Motifs in rAAV Genomes: Avoiding the Toll
- (2020) J. Fraser Wright MOLECULAR THERAPY
- SOD1 Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS
- (2020) Christian Mueller et al. NEW ENGLAND JOURNAL OF MEDICINE
- BAX 335 hemophilia B gene therapy clinical trial results - potential impact of CpG sequences on gene expression
- (2020) Barbara A Konkle et al. BLOOD
- Validation of Serum Neurofilament Light Chain as a Biomarker of Parkinson’s Disease Progression
- (2020) Brit Mollenhauer et al. MOVEMENT DISORDERS
- Induction of ER Stress by an AAV5 BDD FVIII Construct Is Dependent on the Strength of the Hepatic-Specific Promoter
- (2020) Sylvia Fong et al. Molecular Therapy-Methods & Clinical Development
- Rescuing AAV gene transfer from neutralizing antibodies with an IgG-degrading enzyme
- (2020) Zachary C. Elmore et al. JCI Insight
- Quantitative Whole-Body Imaging of I-124-Labeled Adeno-Associated Viral Vector Biodistribution in Nonhuman Primates
- (2020) Douglas J. Ballon et al. HUMAN GENE THERAPY
- Current Clinical Applications of In Vivo Gene Therapy with AAVs
- (2020) Jerry R. Mendell et al. MOLECULAR THERAPY
- qPCR and qRT-PCR analysis: Regulatory points to consider when conducting biodistribution and vector shedding studies
- (2020) Haiyan Ma et al. Molecular Therapy-Methods & Clinical Development
- Adeno-associated virus vector as a platform for gene therapy delivery
- (2019) Dan Wang et al. NATURE REVIEWS DRUG DISCOVERY
- 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
- The Effect of CpG Sequences on Capsid-Specific CD8+ T Cell Responses to AAV Vector Gene Transfer
- (2019) ZhiQuan Xiang et al. MOLECULAR THERAPY
- Gene Therapy with BMN 270 Results in Therapeutic Levels of FVIII in Mice and Primates and Normalization of Bleeding in Hemophilic Mice
- (2018) Stuart Bunting et al. MOLECULAR THERAPY
- 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
- Update on clinical gene therapy for hemophilia
- (2018) George Q. Perrin et al. BLOOD
- AAV8 Gene Therapy for Crigler-Najjar Syndrome in Macaques Elicited Transgene T Cell Responses That Are Resident to the Liver
- (2018) Jenny A. Greig et al. Molecular Therapy-Methods & Clinical Development
- 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
- AAV5–Factor VIII Gene Transfer in Severe Hemophilia A
- (2017) Savita Rangarajan et al. NEW ENGLAND JOURNAL OF MEDICINE
- Class I-restricted T-cell responses to a polymorphic peptide in a gene therapy clinical trial for α-1-antitrypsin deficiency
- (2017) Roberto Calcedo et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Host determinants of adeno-associated viral vector entry
- (2017) Sirika Pillay et al. Current Opinion in Virology
- Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-hGAA) Gene Therapy in Children Affected by Pompe Disease
- (2017) Manuela Corti et al. Human Gene Therapy Clinical Development
- Non-clinical Safety and Efficacy of an AAV2/8 Vector Administered Intravenously for Treatment of Mucopolysaccharidosis Type VI
- (2017) Rita Ferla et al. Molecular Therapy-Methods & Clinical Development
- Phase I open label liver-directed gene therapy clinical trial for acute intermittent porphyria
- (2016) Delia D’Avola et al. JOURNAL OF HEPATOLOGY
- Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B
- (2014) Amit C. Nathwani et al. NEW ENGLAND JOURNAL OF MEDICINE
- Biodistribution of AAV8 Vectors Expressing Human Low-Density Lipoprotein Receptor in a Mouse Model of Homozygous Familial Hypercholesterolemia
- (2013) Shu-Jen Chen et al. Human Gene Therapy Clinical Development
- Integration Frequency and Intermolecular Recombination of rAAV Vectors in Non-human Primate Skeletal Muscle and Liver
- (2012) Ali Nowrouzi et al. MOLECULAR THERAPY
- Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B
- (2011) Amit C. Nathwani et al. NEW ENGLAND JOURNAL OF MEDICINE
- Molecular Analysis of Vector Genome Structures After Liver Transduction by Conventional and Self-Complementary Adeno-Associated Viral Serotype Vectors in Murine and Nonhuman Primate Models
- (2010) Xun Sun et al. HUMAN GENE THERAPY
- 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
- Dystrophin Immunity in Duchenne's Muscular Dystrophy
- (2010) Jerry R. Mendell et al. NEW ENGLAND JOURNAL OF MEDICINE
- Adeno-Associated Virus-Mediated Gene Transfer to Nonhuman Primate Liver Can Elicit Destructive Transgene-Specific T Cell Responses
- (2009) Guangping Gao et al. HUMAN GENE THERAPY
- Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection
- (2008) Carmela Zincarelli et al. MOLECULAR THERAPY
Publish scientific posters with Peeref
Peeref publishes scientific posters from all research disciplines. Our Diamond Open Access policy means free access to content and no publication fees for authors.
Learn MoreCreate your own webinar
Interested in hosting your own webinar? Check the schedule and propose your idea to the Peeref Content Team.
Create Now