Cargando…

Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction

Mostrar otras versiones (1)

Pompe disease results from acid α-glucosidase (GAA) deficiency, and enzyme replacement therapy (ERT) with recombinant human (rh) GAA has clinical benefits, although its limitations include the short half-life of GAA and the formation of antibody responses. The present study compared the efficacy of...

Descripción completa

Detalles Bibliográficos
Autores principales:	Han, Sang-oh, Ronzitti, Giuseppe, Arnson, Benjamin, Leborgne, Christian, Li, Songtao, Mingozzi, Federico, Koeberl, Dwight
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2017
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5363303/ https://www.ncbi.nlm.nih.gov/pubmed/28344998 http://dx.doi.org/10.1016/j.omtm.2016.12.010

Ejemplares similares

Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction
por: Han, Sang-oh, et al.
Publicado: (2019)

Comparisons of Infant and Adult Mice Reveal Age Effects for Liver Depot Gene Therapy in Pompe Disease
por: Han, Sang-oh, et al.
Publicado: (2019)

Enzyme replacement therapy (ERT) in pompe disease
por: Fiumara, Agata
Publicado: (2014)

S3.1 Are there ERT defined guidelines for Pompe disease?
por: Toscano, Antonio, et al.
Publicado: (2011)

Hydrostatic Isolated Limb Perfusion with Adeno-associated Virus Vectors Enhances Correction of Skeletal Muscle in Pompe Disease
por: Sun, Baodong, et al.
Publicado: (2010)

Immune Responses and Hypercoagulation in ERT for Pompe Disease Are Mutation and rhGAA Dose Dependent
por: Nayak, Sushrusha, et al.
Publicado: (2014)

Deficiency in MyD88 Signaling Results in Decreased Antibody Responses to an Adeno-Associated Virus Vector in Murine Pompe Disease
por: Zhang, Ping, et al.
Publicado: (2012)

An immune tolerance approach using transient low-dose methotrexate in the ERT-naïve setting of patients treated with a therapeutic protein: experience in infantile-onset Pompe disease
por: Kazi, Zoheb B., et al.
Publicado: (2018)

L-alanine supplementation in Pompe disease (IOPD): a potential therapeutic implementation for patients on ERT? A case report
por: Rovelli, Valentina, et al.
Publicado: (2022)

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)

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)

Non-depleting anti-CD4 monoclonal antibody induces immune tolerance to ERT in a murine model of Pompe disease
por: Sun, Baodong, et al.
Publicado: (2014)

CRIM-negative infantile Pompe disease: Characterization of immune responses in patients treated with ERT monotherapy
por: Berrier, Kathryn L., et al.
Publicado: (2015)

Three-dimensional tissue-engineered human skeletal muscle model of Pompe disease
por: Wang, Jason, et al.
Publicado: (2021)

Effect of enzyme replacement therapy (ERT) added to Home Mechanical Ventilation (HMV) in Adult Pompe disease
por: Sayeed, Nadia, et al.
Publicado: (2015)

Positive Impact of Home ERT for Mucopolysaccharidoses and Pompe Disease: The Lesson Learnt from the COVID-19 Pandemic
por: Fiumara, Agata, et al.
Publicado: (2023)

Teaching tolerance: New approaches to enzyme replacement therapy for Pompe disease
por: Cousens, Leslie P., et al.
Publicado: (2012)

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

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

Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid α-Glucosidase
por: Cagin, Umut, et al.
Publicado: (2020)

Successful twin pregnancy in a 38-year-old woman with Pompe disease despite interruption of enzyme replacement therapy (ERT)
por: Tiling, N, et al.
Publicado: (2013)

Enzyme Replacement Therapy (ERT) on Heart Function Changes the Outcome in Patients with Infantile-Onset Pompe Disease: A Familial History
por: Lecis, Marco, et al.
Publicado: (2023)

Albuterol as an adjunctive treatment to enzyme replacement therapy in infantile-onset Pompe disease()
por: Chien, Yin-Hsiu, et al.
Publicado: (2017)

Role of Regulatory T Cell and Effector T Cell Exhaustion in Liver-Mediated Transgene Tolerance in Muscle
por: Poupiot, Jérôme, et al.
Publicado: (2019)

Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients
por: Masat, Elisa, et al.
Publicado: (2016)

AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice
por: Colella, Pasqualina, et al.
Publicado: (2018)

A novel therapeutic strategy for skeletal disorders: Proof of concept of gene therapy for X-linked hypophosphatemia
por: Zhukouskaya, Volha V., et al.
Publicado: (2021)

Overcoming the Challenges Imposed by Humoral Immunity to AAV Vectors to Achieve Safe and Efficient Gene Transfer in Seropositive Patients
por: Gross, David-Alexandre, et al.
Publicado: (2022)

Screening data from 19 patients with late‐onset Pompe disease for a phase I clinical trial of AAV8 vector‐mediated gene therapy
por: Hannah, William B., et al.
Publicado: (2023)

Algorithm for the Early Diagnosis and Treatment of Patients with Cross Reactive Immunologic Material-Negative Classic Infantile Pompe Disease: A Step towards Improving the Efficacy of ERT
por: Banugaria, Suhrad G., et al.
Publicado: (2013)

Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction
por: Fitzpatrick, Zachary, et al.
Publicado: (2018)

Long term outcome and clinical experience on immune tolerance induction therapies in infantile Pompe disease
por: Austin, Stephanie, et al.
Publicado: (2013)

Transposon-mediated Generation of Cellular and Mouse Models of Splicing Mutations to Assess the Efficacy of snRNA-based Therapeutics
por: Barbon, Elena, et al.
Publicado: (2016)

Safety outcomes and patients’ preferences for home-based intravenous enzyme replacement therapy (ERT) in pompe disease and mucopolysaccharidosis type I (MPS I) disorder: COVID-19 and beyond
por: Toscano, Antonio, et al.
Publicado: (2023)

Hackertámadás érte a svájci részecskegyorsító rendszerét
Publicado: (2008)

Rekord energiaszintet ért el az LHC
Publicado: (2009)

ERT, HRT, Raloxifine, Calcitonin, or Bisphosphonates for Osteoporosis
por: Curtiss, Frederic R.
Publicado: (2003)

Analysis of barley mutants ert-c.1 and ert-d.7 reveals two loci with additive effect on plant architecture
por: Lu, Qiongxian, et al.
Publicado: (2021)

Warning regarding hematological toxicity of tamoxifen activated CreERT2 in young Rosa26CreERT2 mice
por: Rossi, Martina, et al.
Publicado: (2023)

ERT impact on left ventricular mass in Fabry disease
por: Ortiz, Alberto
Publicado: (2019)

Cannot write session to /tmp/vufind_sessions/sess_84r2cuo4072klaqvfmf2jlm88g