Intravenous delivery for treatment of mucopolysaccharidosis type I: A comparison of AAV serotypes 9 and rh10

Intravenous delivery for treatment of mucopolysaccharidosis type I: A comparison of AAV serotypes 9 and rh10

Mucopolysaccharidosis type I (MPS I) is an inherited metabolic disorder caused by deficiency of alpha-L-iduronidase (IDUA), resulting in accumulation of heparan and dermatan sulfate glycosaminoglycans (GAGs). Individuals with the most severe form of the disease (Hurler syndrome) suffer from neurodeg...

Full description

Bibliographic Details
Main Authors:	Lalitha R. Belur, Kelly M. Podetz-Pedersen, Thuy An Tran, Joshua A. Mesick, Nathaniel M. Singh, Maureen Riedl, Lucy Vulchanova, Karen F. Kozarsky, R. Scott McIvor
Format:	Article
Language:	English
Published:	Elsevier 2020-09-01
Series:	Molecular Genetics and Metabolism Reports
Subjects:	Gene therapy AAV9 AAVrh10 Mucopolysaccharidosis Type I (MPS I) Iduronidase (IDUA) Intravenous delivery
Online Access:	http://www.sciencedirect.com/science/article/pii/S2214426920300501

Similar Items

Molecular Detection and Identification of Α-L-Iduronidase Gene Mutations in 5 Iranian Families Suspected for Muller Syndrome (Mucopolysaccharidosis I)
by: B Nasr- Esfahani, et al.
Published: (2007-07-01)

Molecular Analysis of Vietnamese Patients with Mucopolysaccharidosis Type I
by: Ngoc Thi Bich Can, et al.
Published: (2021-10-01)

Mucopolysaccharidosis Type I in the Russian Federation and Other Republics of the Former Soviet Union: Molecular Genetic Analysis and Epidemiology
by: E. Yu Voskoboeva, et al.
Published: (2022-01-01)

A Biochemical Platform to Define the Relative Specific Activity of <i>IDUA</i> Variants Identified by Newborn Screening
by: Seok-Ho Yu, et al.
Published: (2020-11-01)

Mucopolysaccharidosis type I - Clinical and genetic characteristics of Romanian patients
by: Alkhzouz Camelia, et al.
Published: (2020-07-01)

Mucopolysaccharidosis Type I in Mexico: Case-Based Review
by: Consuelo Cantú-Reyna, et al.
Published: (2023-03-01)

Mucopolysaccharidosis Type I: A Review of the Natural History and Molecular Pathology
by: Christiane S. Hampe, et al.
Published: (2020-08-01)

Mucopolysaccharidosis type I due to maternal uniparental disomy of chromosome 4 with partial isodisomy of 4p16.3p15.2
by: Kloth Katja, et al.
Published: (2020-12-01)

Mucopolysaccharidosis type I Hurler-Scheie syndrome: A rare case report
by: Ramesh Tatapudi, et al.
Published: (2011-01-01)

Characterization of the MPS I-H knock-in mouse reveals increased femoral biomechanical integrity with compromised material strength and altered bone geometry
by: Arin K. Oestreich, et al.
Published: (2015-12-01)

Three novel α-L-iduronidase mutations in 10 unrelated Chinese mucopolysaccharidosis type I families
by: Luning Sun, et al.
Published: (2011-01-01)

Three novel α-L-iduronidase mutations in 10 unrelated Chinese mucopolysaccharidosis type I families
by: Luning Sun, et al.
Published: (2011-01-01)

Molecular analysis of mucopolysaccharidosis type I in Tunisia: identification of novel mutation and eight Novel polymorphisms
by: Froissart Roseline, et al.
Published: (2011-04-01)

Quantification of Idua Enzymatic Activity Combined with Observation of Phenotypic Change in Zebrafish Embryos Provide a Preliminary Assessment of Mutated <i>idua</i> Correlated with Mucopolysaccharidosis Type I
by: Cheng-Yung Lin, et al.
Published: (2022-07-01)

Factors influencing transfection efficiency of pIDUA/nanoemulsion complexes in a mucopolysaccharidosis type I murine model
by: Fraga M, et al.
Published: (2017-03-01)

Long-term reestablishment of alpha-L-iduronidase activity in MPS I fibroblasts after non-viral gene transfer
by: Fabiano de Oliveira Poswar, et al.
Published: (2017-12-01)

Case Report: Mucopolysaccharidosis Type I Treatment With α-L-Iduronidase Replacement Therapy
by: Ying Li, et al.
Published: (2022-04-01)

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
by: Kanut Laoharawee, et al.
Published: (2023-03-01)

Bull's eye maculopathy and subfoveal deposition in two mucopolysaccharidosis type I patients on long-term enzyme replacement therapy
by: Heather G. Mack, et al.
Published: (2018-03-01)

Enzyme replacement therapy for Mucopolysaccharidosis Type I among patients followed within the MPS Brazil Network
by: Alícia Dorneles Dornelles, et al.
Published: (2014-01-01)

Identification of a novel compound heterozygous IDUA mutation underlies Mucopolysaccharidoses type I in a Chinese pedigree
by: Yong‐An Zhou, et al.
Published: (2020-01-01)

Mucopolysaccharidosis Type I Presenting with Persistent Neonatal Respiratory Distress: A Case Report
by: Ali Alsuheel Asseri, et al.
Published: (2023-04-01)

The Effects of Enzyme Replacement Therapy in Patients with Mucopolysaccharidosis Type 1: A Case Series Study
by: Peyman Eshraghi, et al.
Published: (2022-04-01)

Ocular features in mucopolysaccharidosis: diagnosis and treatment
by: Alessandra Del Longo, et al.
Published: (2018-11-01)

Comparative dose effectiveness of intravenous and intrathecal AAV9.CB7.hIDS, RGX-121, in mucopolysaccharidosis type II mice
by: Miles C. Smith, et al.
Published: (2024-03-01)

Transcranial pulsed ultrasound facilitates brain uptake of laronidase in enzyme replacement therapy for Mucopolysaccharidosis type I disease
by: Yu-Hone Hsu, et al.
Published: (2017-06-01)

Genotypic and bioinformatic evaluation of the alpha-l-iduronidase gene and protein in patients with mucopolysaccharidosis type I from Colombia, Ecuador and Peru
by: Tatiana Pineda, et al.
Published: (2014-01-01)

Intraparenchymal convection enhanced delivery of AAV in sheep to treat Mucopolysaccharidosis IIIC
by: Claire O’Leary, et al.
Published: (2023-07-01)

Ocular Tolerability and Immune Response to Corneal Intrastromal AAV-IDUA Gene Therapy in New Zealand White Rabbits
by: Liujiang Song, et al.
Published: (2020-09-01)

Selective transduction of cerebellar Purkinje and granule neurons using delivery of AAV-PHP.eB and AAVrh10 vectors at axonal terminal locations
by: Magdalena Surdyka, et al.
Published: (2022-09-01)

Description of the molecular and clinical characteristics of the mucopolysaccharidosis type VII Iberian cohort
by: Antonio Gónzalez-Meneses, et al.
Published: (2021-10-01)

Reduction of lysosome abundance and GAG accumulation after odiparcil treatment in MPS I and MPS VI models
by: Pascale Tuyaa-Boustugue, et al.
Published: (2023-12-01)

Triamterene Functions as an Effective Nonsense Suppression Agent for MPS I-H (Hurler Syndrome)
by: Amna Siddiqui, et al.
Published: (2023-02-01)

The factors affecting lipid profile in adult patients with Mucopolysaccharidosis
by: Karolina M. Stepien, et al.
Published: (2017-09-01)

Liver-Targeted AAV8 Gene Therapy Ameliorates Skeletal and Cardiovascular Pathology in a Mucopolysaccharidosis IVA Murine Model
by: Kazuki Sawamoto, et al.
Published: (2020-09-01)

Report of Five Years of Experience in Neonatal Screening for Mucopolysaccharidosis Type I and Review of the Literature
by: Vincenza Gragnaniello, et al.
Published: (2020-11-01)

Neurocognitive and somatic stabilization in pediatric patients with severe Mucopolysaccharidosis Type I after 52 weeks of intravenous brain-penetrating insulin receptor antibody-iduronidase fusion protein (valanafusp alpha): an open label phase 1-2 trial
by: Roberto Giugliani, et al.
Published: (2018-07-01)

Supraspinal gene transfer by intrathecal adeno-associated virus serotype 5
by: Daniel J. Schuster, et al.
Published: (2014-08-01)

Mucopolysaccharidosis: A broad review
by: Ritu Nagpal, et al.
Published: (2022-01-01)

Capturing phenotypic heterogeneity in MPS I: results of an international consensus procedure
by: de Ru Minke H, et al.
Published: (2012-04-01)