Cargando…

Preclinical Potency and Biodistribution Studies of an AAV 5 Vector Expressing Human Interferon-β (ART-I02) for Local Treatment of Patients with Rheumatoid Arthritis

INTRODUCTION: Proof of concept for local gene therapy for the treatment of arthritis with immunomodulatory cytokine interferon beta (IFN-β) has shown promising results in animal models of rheumatoid arthritis (RA). For the treatment of RA patients, we engineered a recombinant adeno-associated seroty...

Descripción completa

Detalles Bibliográficos
Autores principales:	Aalbers, Caroline J., Bevaart, Lisette, Loiler, Scott, de Cortie, Karin, Wright, J. Fraser, Mingozzi, Federico, Tak, Paul P., Vervoordeldonk, Margriet J.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Public Library of Science 2015
Materias:	Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4479517/ https://www.ncbi.nlm.nih.gov/pubmed/26107769 http://dx.doi.org/10.1371/journal.pone.0130612

Ejemplares similares

Advancements in adeno-associated viral gene therapy approaches: exploring a new horizon
por: Aalbers, Caroline J., et al.
Publicado: (2011)

Local delivery of beta interferon using an adeno-associated virus type 5 effectively inhibits adjuvant arthritis in rats
por: Adriaansen, J., et al.
Publicado: (2007)

Prevalence and pharmacological modulation of humoral immunity to AAV vectors in gene transfer to synovial tissue
por: Mingozzi, F, et al.
Publicado: (2013)

Two Novel α7 Nicotinic Acetylcholine Receptor Ligands: In Vitro Properties and Their Efficacy in Collagen-Induced Arthritis in Mice
por: van Maanen, Marjolein A., et al.
Publicado: (2015)

Preclinical biodistribution, tropism, and efficacy of oligotropic AAV/Olig001 in a mouse model of congenital white matter disease
por: Francis, Jeremy S., et al.
Publicado: (2021)

Switching of serotype can improve local AAV-mediated gene therapy in RA
por: Adriaansen, J, et al.
Publicado: (2004)

Characterization and biodistribution of under-employed gene therapy vector AAV7
por: Yost, Samantha A., et al.
Publicado: (2023)

Treatment with recombinant interferon-β reduces inflammation and slows cartilage destruction in the collagen-induced arthritis model of rheumatoid arthritis
por: van Holten, Judith, et al.
Publicado: (2004)

Semirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disorders
por: El Andari, Jihad, et al.
Publicado: (2022)

Local interferon-β gene therapy ameliorates adjuvant arthritis in rats
por: Adriaansen, P, et al.
Publicado: (2004)

Emerging Issues in AAV-Mediated In Vivo Gene Therapy
por: Colella, Pasqualina, et al.
Publicado: (2017)

Gene Therapy Targeting Nuclear Factor-κB: Towards Clinical Application in Inflammatory Diseases and Cancer
por: Tas, Sander W., et al.
Publicado: (2009)

Cell-Mediated Immunity to AAV Vectors, Evolving Concepts and Potential Solutions
por: Basner-Tschakarjan, Etiena, et al.
Publicado: (2014)

Human Immune Responses to Adeno-Associated Virus (AAV) Vectors
por: Ronzitti, Giuseppe, et al.
Publicado: (2020)

Biodistribution of Mesenchymal Stem/Stromal Cells in a Preclinical Setting
por: Sensebé, Luc, et al.
Publicado: (2013)

Comparative strategies for stem cell biodistribution in a preclinical study
por: Wang, Fang, et al.
Publicado: (2019)

Regulatory guidelines and preclinical tools to study the biodistribution of RNA therapeutics
por: Vervaeke, P., et al.
Publicado: (2022)

Systemic AAV6-synapsin-GFP administration results in lower liver biodistribution, compared to AAV1&2 and AAV9, with neuronal expression following ultrasound-mediated brain delivery
por: Weber-Adrian, Danielle, et al.
Publicado: (2021)

AAV capsid CD8+ T-cell epitopes are highly conserved across AAV serotypes
por: Hui, Daniel J, et al.
Publicado: (2015)

Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome
por: Collaud, Fanny, et al.
Publicado: (2018)

Unraveling the Complex Story of Immune Responses to AAV Vectors Trial After Trial
por: Vandamme, Céline, et al.
Publicado: (2017)

Biodistribution of adeno-associated virus serotype 9 (AAV9) vector after intrathecal and intravenous delivery in mouse
por: Schuster, Daniel J., et al.
Publicado: (2014)

Strategies to generate high-titer, high-potency recombinant AAV3 serotype vectors
por: Ling, Chen, et al.
Publicado: (2016)

CRISPR Activation Enhances In Vitro Potency of AAV Vectors Driven by Tissue-Specific Promoters
por: McDougald, Devin S., et al.
Publicado: (2019)

Local treatment with the selective IκB kinase β inhibitor NEMO-binding domain peptide ameliorates synovial inflammation
por: Tas, Sander W, et al.
Publicado: (2006)

Assessment of toxicity and biodistribution of recombinant AAV8 vector–mediated immunomodulatory gene therapy in mice with Pompe disease
por: Wang, Gensheng, et al.
Publicado: (2014)

Characterization of the humanized FRG mouse model and development of an AAV-LK03 variant with improved liver lobular biodistribution
por: Cabanes-Creus, Marti, et al.
Publicado: (2023)

Preclinical Study on Biodistribution of Mesenchymal Stem Cells after Local Transplantation into the Brain
por: Bashyal, Narayan, et al.
Publicado: (2023)

Immunomodulation in Administration of rAAV: Preclinical and Clinical Adjuvant Pharmacotherapies
por: Chu, Wing Sum, et al.
Publicado: (2021)

Preclinical toxicity evaluation of AAV for pain: evidence from human AAV studies and from the pharmacology of analgesic drugs
por: Pleticha, Josef, et al.
Publicado: (2014)

Corrigendum to “Assessment of toxicity and biodistribution of recombinant AAV8 vector–mediated immunomodulatory gene therapy in mice with Pompe disease”
por: Wang, Gensheng, et al.
Publicado: (2015)

Preclinical biodistribution and dosimetry and human biodistribution comparing (18)F-rhPSMA-7 and single isomer (18)F-rhPSMA-7.3
por: Knorr, Karina, et al.
Publicado: (2022)

In vivo engineering of lymphocytes after systemic exosome-associated AAV delivery
por: Breuer, Cort B., et al.
Publicado: (2020)

IL12-Mediated Liver Inflammation Reduces the Formation of AAV Transcriptionally Active Forms but Has No Effect over Preexisting AAV Transgene Expression
por: Gil-Fariña, Irene, et al.
Publicado: (2013)

AAV-Mediated Gene Therapy for Choroideremia: Preclinical Studies in Personalized Models
por: Vasireddy, Vidyullatha, et al.
Publicado: (2013)

Development of a one-step RT-ddPCR method to determine the expression and potency of AAV vectors
por: Clarner, Pete, et al.
Publicado: (2021)

Pharmacological Modulation of Humoral Immunity in a Nonhuman Primate Model of AAV Gene Transfer for Hemophilia B
por: Mingozzi, Federico, et al.
Publicado: (2012)

Safety of AAV Factor IX Peripheral Transvenular Gene Delivery to Muscle in Hemophilia B Dogs
por: Haurigot, Virginia, et al.
Publicado: (2010)

Biodistribution and Tolerability of AAV-PHP.B-CBh-SMN1 in Wistar Han Rats and Cynomolgus Macaques Reveal Different Toxicologic Profiles
por: Palazzi, Xavier, et al.
Publicado: (2022)

Toxicology and Biodistribution: The Clinical Value of Animal Biodistribution Studies
por: Silva Lima, Beatriz, et al.
Publicado: (2018)

Cannot write session to /tmp/vufind_sessions/sess_gsvd9reo0dapodrtrp20758ebu