Cargando…

Perinatal systemic gene delivery using adeno-associated viral vectors

Neurodegenerative monogenic diseases often affect tissues and organs beyond the nervous system. An effective treatment would require a systemic approach. The intravenous administration of novel therapies is ideal but is hampered by the inability of such drugs to cross the blood–brain barrier (BBB) a...

Descripción completa

Detalles Bibliográficos
Autores principales:	Karda, Rajvinder, Buckley, Suzanne M. K., Mattar, Citra N., Ng, Joanne, Massaro, Giulia, Hughes, Michael P., Kurian, Manju A., Baruteau, Julien, Gissen, Paul, Chan, Jerry K. Y., Bacchelli, Chiara, Waddington, Simon N., Rahim, Ahad A.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Frontiers Media S.A. 2014
Materias:	Neuroscience
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4231876/ https://www.ncbi.nlm.nih.gov/pubmed/25452713 http://dx.doi.org/10.3389/fnmol.2014.00089

Ejemplares similares

Generation of light-producing somatic-transgenic mice using adeno-associated virus vectors
por: Karda, Rajvinder, et al.
Publicado: (2020)

Ascending Vaginal Infection Using Bioluminescent Bacteria Evokes Intrauterine Inflammation, Preterm Birth, and Neonatal Brain Injury in Pregnant Mice
por: Suff, Natalie, et al.
Publicado: (2018)

Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development
por: Massaro, Giulia, et al.
Publicado: (2021)

Perinatal Gene Transfer to the Liver
por: McKay, Tristan R, et al.
Publicado: (2011)

Foamy Virus Vectors Transduce Visceral Organs and Hippocampal Structures following In Vivo Delivery to Neonatal Mice
por: Counsell, John R., et al.
Publicado: (2018)

Argininosuccinic aciduria fosters neuronal nitrosative stress reversed by Asl gene transfer
por: Baruteau, Julien, et al.
Publicado: (2018)

Cervical Gene Delivery of the Antimicrobial Peptide, Human β-Defensin (HBD)-3, in a Mouse Model of Ascending Infection-Related Preterm Birth
por: Suff, Natalie, et al.
Publicado: (2020)

In Utero Transfer of Adeno-Associated Viral Vectors Produces Long-Term Factor IX Levels in a Cynomolgus Macaque Model
por: Mattar, Citra N.Z., et al.
Publicado: (2017)

Systemic gene delivery following intravenous administration of AAV9 to fetal and neonatal mice and late-gestation nonhuman primates
por: Mattar, Citra N., et al.
Publicado: (2015)

Clinical and Molecular Features of Early Infantile Niemann Pick Type C Disease
por: Seker Yilmaz, Berna, et al.
Publicado: (2020)

Therapeutic expression of human clotting factors IX and X following adeno-associated viral vector–mediated intrauterine gene transfer in early-gestation fetal macaques
por: Chan, Jerry K. Y., et al.
Publicado: (2019)

Evidence for Contribution of CD4+CD25+ Regulatory T Cells in Maintaining Immune Tolerance to Human Factor IX following Perinatal Adenovirus Vector Delivery
por: Nivsarkar, Megha S., et al.
Publicado: (2015)

Production of lentiviral vectors using novel, enzymatically produced, linear DNA
por: Karda, Rajvinder, et al.
Publicado: (2019)

Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects
por: Baruteau, Julien, et al.
Publicado: (2017)

A Broad Overview and Review of CRISPR-Cas Technology and Stem Cells
por: Waddington, Simon N., et al.
Publicado: (2016)

Longitudinal in vivo bioimaging of hepatocyte transcription factor activity following cholestatic liver injury in mice
por: Delhove, Juliette M. K. M., et al.
Publicado: (2017)

In vivo bioimaging with tissue-specific transcription factor activated luciferase reporters
por: Buckley, Suzanne M. K., et al.
Publicado: (2015)

Rapid and inexpensive purification of adenovirus vectors using an optimised aqueous two-phase technology
por: Suleman, Saqlain, et al.
Publicado: (2022)

Eliminating HIV-1 Packaging Sequences from Lentiviral Vector Proviruses Enhances Safety and Expedites Gene Transfer for Gene Therapy
por: Vink, Conrad A., et al.
Publicado: (2017)

Continual conscious bioluminescent imaging in freely moving somatotransgenic mice
por: Karda, Rajvinder, et al.
Publicado: (2017)

Re-structuring lentiviral vectors to express genomic RNA via cap-dependent translation
por: Counsell, John R., et al.
Publicado: (2020)

Dopamine Transporter Deficiency Syndrome (DTDS): Expanding the Clinical Phenotype and Precision Medicine Approaches
por: Ng, Joanne, et al.
Publicado: (2023)

Fetal gene therapy for neurodegenerative disease of infants
por: Massaro, Giulia, et al.
Publicado: (2018)

Regionally-Specified Second Trimester Fetal Neural Stem Cells Reveals Differential Neurogenic Programming
por: Fan, Yiping, et al.
Publicado: (2014)

Long-Term Reproducible Expression in Human Fetal Liver Hematopoietic Stem Cells with a UCOE-Based Lentiviral Vector
por: Dighe, Niraja, et al.
Publicado: (2014)

A comparison of intrauterine hemopoietic cell transplantation and lentiviral gene transfer for the correction of severe β-thalassemia in a HbbTh3/+ murine model
por: Dighe, Niraja M., et al.
Publicado: (2018)

Urea Cycle Related Amino Acids Measured in Dried Bloodspots Enable Long-Term In Vivo Monitoring and Therapeutic Adjustment
por: Baruteau, Julien, et al.
Publicado: (2019)

Systemic AAV9 gene therapy using the synapsin I promoter rescues a mouse model of neuronopathic Gaucher disease but with limited cross-correction potential to astrocytes
por: Massaro, Giulia, et al.
Publicado: (2020)

Clinical applications for exosomes: Are we there yet?
por: Perocheau, Dany, et al.
Publicado: (2021)

Assessment of Pre-Clinical Liver Models Based on Their Ability to Predict the Liver-Tropism of Adeno-Associated Virus Vectors
por: Westhaus, Adrian, et al.
Publicado: (2023)

Systemic gene therapy rescues retinal dysfunction and hearing loss in a model of Norrie disease
por: Pauzuolyte, Valda, et al.
Publicado: (2023)

Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys
por: Baruteau, Julien, et al.
Publicado: (2021)

Adeno-Associated Viral Vectors in Neuroscience Research
por: Haggerty, David L., et al.
Publicado: (2019)

Comparison of analytical techniques to quantitate the capsid content of adeno-associated viral vectors
por: Werle, Amanda K., et al.
Publicado: (2021)

Age-Related Seroprevalence of Antibodies Against AAV-LK03 in a UK Population Cohort
por: Perocheau, Dany P., et al.
Publicado: (2019)

Treating Transthyretin Amyloidosis via Adeno-Associated Virus Vector Delivery of Meganucleases
por: Greig, Jenny A., et al.
Publicado: (2022)

In Situ Detection of Adeno-associated Viral Vector Genomes with SABER-FISH
por: Wang, Sean K., et al.
Publicado: (2020)

Novel Cytotoxic Vectors Based on Adeno-Associated Virus
por: Kohlschütter, Johannes, et al.
Publicado: (2010)

Production and Titering of Recombinant Adeno-associated Viral Vectors
por: McClure, Christina, et al.
Publicado: (2011)

Accurate Quantification and Characterization of Adeno-Associated Viral Vectors
por: Dobnik, David, et al.
Publicado: (2019)

Cannot write session to /tmp/vufind_sessions/sess_t24dp2o85fr56ir3aske49nu4t