Gene therapy for mucopolysaccharidosis (Q24631135)

Expert Opinion on Biological Therapy

Europe PubMed Central

title

Gene therapy for mucopolysaccharidosis (English)

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Katherine P Ponder

1

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Mark E Haskins

series ordinal

2

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language of work or name

English

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publication date

September 2007

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published in

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volume

7

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page(s)

1333-45

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issue

9

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cites work

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A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome)

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Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

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Correction of clinical manifestations of canine mucopolysaccharidosis I with neonatal retroviral vector gene therapy

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The phiC31 integrase system for gene therapy

27 September 2017

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Effective suicide gene therapy for leukemia in a model of insertional oncogenesis in mice

27 September 2017

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Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity

27 September 2017

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Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer

27 September 2017

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Cell and gene-based therapies for the lysosomal storage diseases

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Viral gene therapy strategies: from basic science to clinical application

27 September 2017

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Successful correction of the human Cooley's anemia beta-thalassemia major phenotype using a lentiviral vector flanked by the chicken hypersensitive site 4 chromatin insulator

27 September 2017

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Overcoming the blood-brain barrier with high-dose enzyme replacement therapy in murine mucopolysaccharidosis VII.

27 September 2017

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Neonatal gene therapy of MPS I mice by intravenous injection of a lentiviral vector

27 September 2017

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Enzyme replacement and enhancement therapies for lysosomal diseases

27 September 2017

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Gene therapy for lysosomal storage diseases: the lessons and promise of animal models

27 September 2017

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Cord-blood transplants from unrelated donors in patients with Hurler's syndrome

27 September 2017

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Improvement of skeletal lesions in mice with mucopolysaccharidosis type vii by neonatal adenoviral gene transfer

27 September 2017

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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340574

Intraventricular brain injection of adeno-associated virus type 1 (AAV1) in neonatal mice results in complementary patterns of neuronal transduction to AAV2 and total long-term correction of storage lesions in the brains of beta-glucuronidase-defici

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340574

Phase 1 trial of FVIII gene transfer for severe hemophilia A using a retroviral construct administered by peripheral intravenous infusion

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340574

Evaluation of pathological manifestations of disease in mucopolysaccharidosis VII mice after neonatal hepatic gene therapy.

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340574

Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340574

Functional correction of established central nervous system deficits in an animal model of lysosomal storage disease with feline immunodeficiency virus-based vectors

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340574

Treatment of a lysosomal storage disease, mucopolysaccharidosis VII, with microencapsulated recombinant cells

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340574

Systemic and central nervous system correction of lysosomal storage in mucopolysaccharidosis type VII mice

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340574

Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease.

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340574

Adenovirus-mediated gene transfer and expression of human beta-glucuronidase gene in the liver, spleen, and central nervous system in mucopolysaccharidosis type VII mice

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340574

Correction of lysosomal storage in the liver and spleen of MPS VII mice by implantation of genetically modified skin fibroblasts

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340574

Phenotype correction in retinal pigment epithelium in murine mucopolysaccharidosis VII by adenovirus-mediated gene transfer

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340574

The HIV Tat protein transduction domain improves the biodistribution of beta-glucuronidase expressed from recombinant viral vectors.

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340574

Bone marrow transplantation for Maroteaux-Lamy syndrome (MPS VI): long-term follow-up.

30 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340574

Hurler syndrome: II. Outcome of HLA-genotypically identical sibling and HLA-haploidentical related donor bone marrow transplantation in fifty-four children. The Storage Disease Collaborative Study Group

30 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340574

Myoblast gene therapy in canine mucopolysaccharidosis. I: Abrogation by an immune response to alpha-L-iduronidase

30 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340574

A follow-up study of MPS I patients treated with laronidase enzyme replacement therapy for 6 years.

30 May 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Long-term follow-up following bone marrow transplantation for Hunter disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Correction of Hunter syndrome in the MPSII mouse model by AAV2/8-mediated gene delivery

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Liver-directed neonatal gene therapy prevents cardiac, bone, ear, and eye disease in mucopolysaccharidosis I mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Effect of neonatal administration of a retroviral vector expressing alpha-L-iduronidase upon lysosomal storage in brain and other organs in mucopolysaccharidosis I mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Clinical response to persistent, low-level beta-glucuronidase expression in the murine model of mucopolysaccharidosis type VII.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Correction of metabolic, craniofacial, and neurologic abnormalities in MPS I mice treated at birth with adeno-associated virus vector transducing the human alpha-L-iduronidase gene

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Improvements in mucopolysaccharidosis I mice after adult retroviral vector-mediated gene therapy with immunomodulation.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Gene therapy ameliorates cardiovascular disease in dogs with mucopolysaccharidosis VII.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Neonatal retroviral vector-mediated hepatic gene therapy reduces bone, joint, and cartilage disease in mucopolysaccharidosis VII mice and dogs

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Expression in blood cells may contribute to biochemical and pathological improvements after neonatal intravenous gene therapy for mucopolysaccharidosis VII in dogs

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Widespread correction of lysosomal storage following intrahepatic injection of a recombinant adeno-associated virus in the adult MPS VII mouse

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Neonatal intramuscular injection with recombinant adeno-associated virus results in prolonged beta-glucuronidase expression in situ and correction of liver pathology in mucopolysaccharidosis type VII mice.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Reduction of GAG storage in MPS II mouse model following implantation of encapsulated recombinant myoblasts

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Gene transfer of low levels of beta-glucuronidase corrects hepatic lysosomal storage in a large animal model of mucopolysaccharidosis VII.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Human CD34+ hematopoietic progenitor cell-directed lentiviral-mediated gene therapy in a xenotransplantation model of lysosomal storage disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Treatment of the mouse model of mucopolysaccharidosis I with retrovirally transduced bone marrow

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Retrovirally transduced bone marrow has a therapeutic effect on brain in the mouse model of mucopolysaccharidosis IIIB.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Gene therapy for canine alpha-L-iduronidase deficiency: in utero adoptive transfer of genetically corrected hematopoietic progenitors results in engraftment but not amelioration of disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

In utero injection of alpha-L-iduronidase-carrying retrovirus in canine mucopolysaccharidosis type I: infection of multiple tissues and neonatal gene expression

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Gene targeting in vivo by adeno-associated virus vectors.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

alpha-L-Iduronidase transport in neurites.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Prevention of neuropathology in the mouse model of Hurler syndrome

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Improved behavior and neuropathology in the mouse model of Sanfilippo type IIIB disease after adeno-associated virus-mediated gene transfer in the striatum.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Long-term and significant correction of brain lesions in adult mucopolysaccharidosis type VII mice using recombinant AAV vectors.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Adeno-associated virus type 5 reduces learning deficits and restores glutamate receptor subunit levels in MPS VII mice CNS.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Adeno-associated virus vector-mediated transduction in the cat brain

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Functional correction of CNS phenotypes in a lysosomal storage disease model using adeno-associated virus type 4 vectors.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Genetic correction of the fetal brain increases the lifespan of mice with the severe multisystemic disease mucopolysaccharidosis type VII.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Brain transplantation of genetically modified bone marrow stromal cells corrects CNS pathology and cognitive function in MPS VII mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Neurological correction of lysosomal storage in a mucopolysaccharidosis IIIB mouse model by adeno-associated virus-mediated gene delivery

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Widespread correction of lysosomal storage in the mucopolysaccharidosis type VII mouse brain with a herpes simplex virus type 1 vector expressing beta-glucuronidase

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Reversal of pathology in the entire brain of mucopolysaccharidosis type VII mice after lentivirus-mediated gene transfer.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Intracranial injection of recombinant adeno-associated virus improves cognitive function in a murine model of mucopolysaccharidosis type VII.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Elimination of lysosomal storage in brains of MPS VII mice treated by intrathecal administration of an adeno-associated virus vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Gene therapy of the brain in the dog model of Hurler's syndrome.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

AAV-mediated intravitreal gene therapy reduces lysosomal storage in the retinal pigmented epithelium and improves retinal function in adult MPS VII mice.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Adenovirus-mediated gene therapy for corneal clouding in mice with mucopolysaccharidosis type VII.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Phenotypic rescue after adeno-associated virus-mediated delivery of 4-sulfatase to the retinal pigment epithelium of feline mucopolysaccharidosis VI.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Identification and characterization of 13 new mutations in mucopolysaccharidosis type I patients.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Mucopolysaccharidosis I cats mount a cytotoxic T lymphocyte response after neonatal gene therapy that can be blocked with CTLA4-Ig.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Gene therapy for a mucopolysaccharidosis type I murine model with lentiviral-IDUA vector.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Limited transgene immune response and long-term expression of human alpha-L-iduronidase in young adult mice with mucopolysaccharidosis type I by liver-directed gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Adenovirus-mediated gene therapy for mucopolysaccharidosis VII: involvement of cross-correction in wide-spread distribution of the gene products and long-term effects of CTLA-4Ig coexpression

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Oncogenesis following delivery of a nonprimate lentiviral gene therapy vector to fetal and neonatal mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

AAV vector integration sites in mouse hepatocellular carcinoma.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Enhanced secretion and uptake of beta-glucuronidase improves adeno-associated viral-mediated gene therapy of mucopolysaccharidosis type VII mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Long-term normalization in the central nervous system, ocular manifestations, and skeletal deformities by a single systemic adenovirus injection into neonatal mice with mucopolysaccharidosis VII.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.9.1333

Intrathecal administration of AAV vectors for the treatment of lysosomal storage in the brains of MPS I mice

21 January 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340574

Intravitreal gene therapy reduces lysosomal storage in specific areas of the CNS in mucopolysaccharidosis VII mice.

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340574

Distribution of a lysosomal enzyme in the adult brain by axonal transport and by cells of the rostral migratory stream.

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340574

Tandem mass spectrometric analysis of dried blood spots for screening of mucopolysaccharidosis I in newborns

28 November 2018

1 reference

12 December 2020

Systemic hyperosmolality improves beta-glucuronidase distribution and pathology in murine MPS VII brain following intraventricular gene transfer

1 reference

12 December 2020

Newborn screening for lysosomal storage disorders

1 reference

12 December 2020

Delivery of a retroviral vector expressing human beta-glucuronidase to the liver and spleen decreases lysosomal storage in mucopolysaccharidosis VII mice

1 reference

12 December 2020

Allogeneic bone marrow transplantation for lysosomal storage diseases. The European Group for Bone Marrow Transplantation

1 reference

12 December 2020

Enzyme replacement therapy for mucopolysaccharidosis VI: A phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, o

1 reference

12 December 2020

Cumulative incidence rates of the mucopolysaccharidoses in Germany

1 reference

12 December 2020

Genetically corrected autologous stem cells engraft, but host immune responses limit their utility in canine alpha-L-iduronidase deficiency

1 reference

12 December 2020