Cargando…

ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome

Mucopolysaccharidosis type I (MPS I) is a severe disease due to deficiency of the lysosomal hydrolase α-L-iduronidase (IDUA) and the subsequent accumulation of the glycosaminoglycans (GAG), leading to progressive, systemic disease and a shortened lifespan. Current treatment options consist of hemato...

Descripción completa

Detalles Bibliográficos
Autores principales:	Ou, Li, DeKelver, Russell C., Rohde, Michelle, Tom, Susan, Radeke, Robert, St. Martin, Susan J., Santiago, Yolanda, Sproul, Scott, Przybilla, Michael J., Koniar, Brenda L., Podetz-Pedersen, Kelly M., Laoharawee, Kanut, Cooksley, Renee D., Meyer, Kathleen E., Holmes, Michael C., McIvor, R. Scott, Wechsler, Thomas, Whitley, Chester B.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2019
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6319315/ https://www.ncbi.nlm.nih.gov/pubmed/30528089 http://dx.doi.org/10.1016/j.ymthe.2018.10.018

Ejemplares similares

Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing
por: Laoharawee, Kanut, et al.
Publicado: (2018)

Non-invasive intravenous administration of AAV9 transducing iduronate sulfatase leads to global metabolic correction and prevention of neurologic deficits in a mouse model of Hunter syndrome
por: Laoharawee, Kanut, et al.
Publicado: (2023)

Elements of lentiviral vector design toward gene therapy for treating mucopolysaccharidosis I
por: Ou, Li, et al.
Publicado: (2016)

ZFN-mediated in vivo gene editing in hepatocytes leads to supraphysiologic α-Gal A activity and effective substrate reduction in Fabry mice
por: Pagant, Silvere, et al.
Publicado: (2021)

Beneath the floor: re-analysis of neurodevelopmental outcomes in untreated Hurler syndrome
por: Shapiro, Elsa G., et al.
Publicado: (2018)

Early Neonatal Cardiac Phenotype in Hurler Syndrome: Case Report and Literature Review
por: Pillai, Nishitha R., et al.
Publicado: (2022)

Long-term cognitive and somatic outcomes of enzyme replacement therapy in untransplanted Hurler syndrome
por: Eisengart, Julie B., et al.
Publicado: (2017)

Increased Longevity and Metabolic Correction Following Syngeneic Bone Marrow Transplantation in a Murine Model of Mucopolysaccharidosis Type I
por: Wolf, Daniel A., et al.
Publicado: (2011)

Clinical trial of laronidase in Hurler syndrome after hematopoietic cell transplantation
por: Polgreen, Lynda E., et al.
Publicado: (2019)

A longitudinal study of neurocognition and behavior in patients with Hurler-Scheie syndrome heterozygous for the L238Q mutation
por: Ahmed, Alia, et al.
Publicado: (2019)

The prevalence of and survival in Mucopolysaccharidosis I: Hurler, Hurler-Scheie and Scheie syndromes in the UK
por: Moore, David, et al.
Publicado: (2008)

Phenotype prediction for mucopolysaccharidosis type I by in silico analysis
por: Ou, Li, et al.
Publicado: (2017)

Long-term outcomes of systemic therapies for Hurler syndrome: an international multi-center comparison
por: Eisengart, Julie B., et al.
Publicado: (2018)

Engineering of Primary Human B cells with CRISPR/Cas9 Targeted Nuclease
por: Johnson, Matthew J., et al.
Publicado: (2018)

A Broad Range of Dose Optima Achieve High-level, Long-term Gene Expression After Hydrodynamic Delivery of Sleeping Beauty Transposons Using Hyperactive SB100x Transposase
por: Podetz-Pedersen, Kelly M, et al.
Publicado: (2016)

Targeted gene knockout by direct delivery of ZFN proteins
por: Gaj, Thomas, et al.
Publicado: (2012)

Early disease progression of Hurler syndrome
por: Kiely, Bridget T., et al.
Publicado: (2017)

Hurler Syndrome: Orofacial Clinical Findings
por: Rodrigues Barros, Cristina, et al.
Publicado: (2023)

Examination of a blood-brain barrier targeting β-galactosidase-monoclonal antibody fusion protein in a murine model of GM1-gangliosidosis
por: Przybilla, Michael J., et al.
Publicado: (2021)

An optimized two-finger archive for ZFN-mediated gene targeting
por: Gupta, Ankit, et al.
Publicado: (2012)

Management of an anticipated difficult airway in Hurler's syndrome
por: Gurumurthy, T., et al.
Publicado: (2014)

Hurler's Disease with Multiple Atypical Mongolian Spots
por: Sonthalia, Sidharth, et al.
Publicado: (2016)

Open issues in Mucopolysaccharidosis type I-Hurler
por: Parini, Rossella, et al.
Publicado: (2017)

Heart valve disease in Hurler-Scheie syndrome
por: del Carmen García del Rey, María, et al.
Publicado: (2022)

CPP-ZFN: A potential DNA-targeting anti-malarial drug
por: Nain, Vikrant, et al.
Publicado: (2010)

Expanded activity of dimer nucleases by combining ZFN and TALEN for genome editing
por: Yan, Wei, et al.
Publicado: (2013)

HURLER'S SYNDROME : A GENETIC STUDY IN CELL CULTURE
por: Danes, B. Shannon, et al.
Publicado: (1966)

Longitudinal neurocognitive outcome in an adolescent with Hurler-Scheie syndrome
por: Elkin, T David, et al.
Publicado: (2006)

Hurler–Scheie syndrome in Niger: a case series
por: Assadeck, Hamid, et al.
Publicado: (2019)

Correction of Fanconi Anemia Mutations Using Digital Genome Engineering
por: Sipe, Christopher J., et al.
Publicado: (2022)

Magnetic Separation and Antibiotics Selection Enable Enrichment of Cells with ZFN/TALEN-Induced Mutations
por: Kim, Hyojin, et al.
Publicado: (2013)

Finding of a highly efficient ZFN pair for Aqpep gene functioning in murine zygotes
por: FUJII, Wataru, et al.
Publicado: (2015)

Effect of ZFN-edited myostatin loss-of-function mutation on gut microbiota in Meishan pigs
por: Cui, Wen-Tao, et al.
Publicado: (2019)

Inhibition of angiogenesis and suppression of colorectal cancer metastatic to the liver using the Sleeping Beauty Transposon System
por: Belur, Lalitha R, et al.
Publicado: (2011)

Identification of a novel fusion Iduronidase with improved activity in the cardiovascular system
por: Kim, Sarah, et al.
Publicado: (2022)

THE HURLER SYNDROME: A STUDY OF CULTURED LYMPHOID CELL LINES
por: Danes, B. Shannon, et al.
Publicado: (1972)

SOME PARADOXIAL OBSERVATIONS NOTED IN THE PREVALENCE OF HURLERS AND HUNTER'S SYNDROME
por: Narayanan, H.S., et al.
Publicado: (1987)

Hurler syndrome or Morquio syndrome: Intelligence required for diagnosing the case
por: Taksande, Amar, et al.
Publicado: (2008)

Airway management in Hurler's syndrome: A persistent challenge for anaesthesiologists
por: Kerai, Sukhyanti, et al.
Publicado: (2016)

Hurler Syndrome (Mucopolysaccharidosis Type 1): A Case Report
por: Khalid, Noman, et al.
Publicado: (2023)

Cannot write session to /tmp/vufind_sessions/sess_2tii4j7b3tckf7u7t2cd7qqg39