Long-term expression and transfer of arylsulfatase A into brain of arylsulfatase A-deficient mice transplanted with bone marrow expressing the arylsulfatase A cDNA from a retroviral vector

Matzner, Ulrich; Harzer, K.; Learish, R.; Ja, Barranger; Gieselmann, Volkmar

doi:10.1038/sj.gt.3301232

Cited by 62 publications

(57 citation statements)

References 27 publications

(30 reference statements)

Supporting

Mentioning

Contrasting

Order By: Relevance

“…Transmission to the CNS was less effective, such that up to 33% of normal enzyme activity was achieved ( Figure 2g and Ref. 14). Semiquantitative PCR revealed that 0.1 to 0.3% of brain cells were of donor type 1 year after transplantation.…”

Section: Discussionmentioning

confidence: 99%

“…Semiquantitative PCR revealed that 0.1 to 0.3% of brain cells were of donor type 1 year after transplantation. 14 This study has evaluated the possible therapeutic effects mediated by this stem cell gene transfer approach. For the determination of the therapeutic outcome, treated mice which continuously expressed hASA were compared about 1 year after BMT with mock-treated mice, ie ASA knockout mice which were transplanted with nontransduced bone marrow from deficient donors and had undergone all processes of BMT, in particular, lethal irradiation.…”

Section: Discussionmentioning

confidence: 99%

“…In the CNS of treated mice up to 10 (brainstem, forebrain) and 14 ng/mg ASA (cerebellum) were detected ( Figure 2g and Ref. 14).…”

Section: Histochemical Analysis Of Sulfatide Storagementioning

confidence: 98%

“…14,17 The activity assay is compromised by high background activities of unspecific sulfatases so that the hASA activity has to be calculated by subtraction of the mean sulfatase background activity (determined in livers of mocktreated, ASA-deficient mice) from the compound sulfatase activity measured (not shown). Division of the calculated data by corresponding ELISA data indicated a specific activity of hASA around 45 mU/ g on average (see below).…”

Section: Histochemical Analysis Of Sulfatide Storagementioning

confidence: 99%

“…This value is close to the normal specific activity of gene-encoded hASA in human tissues and cell lines (40 mU/ g). 14,17 Since the ELISA is specific for hASA and thus superior to the activity assay in the precise evaluation of minor differences in hASA expression levels it was used for all other analyses.…”

Section: Histochemical Analysis Of Sulfatide Storagementioning

confidence: 99%

See 4 more Smart Citations

Bone marrow stem cell-based gene transfer in a mouse model for metachromatic leukodystrophy: effects on visceral and nervous system disease manifestations

et al. 2002

Self Cite

View full text Add to dashboard Cite

Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

“…In the CNS of treated mice up to 10 (brainstem, forebrain) and 14 ng/mg ASA (cerebellum) were detected ( Figure 2g and Ref. 14).…”

Section: Histochemical Analysis Of Sulfatide Storagementioning

confidence: 98%

Section: Histochemical Analysis Of Sulfatide Storagementioning

confidence: 99%

Section: Histochemical Analysis Of Sulfatide Storagementioning

confidence: 99%

See 3 more Smart Citations

Bone marrow stem cell-based gene transfer in a mouse model for metachromatic leukodystrophy: effects on visceral and nervous system disease manifestations

et al. 2002

Self Cite

View full text Add to dashboard Cite

Sulfatases (Sulfohydrolases, Sulfuric Ester Hydrolases)

Dierks

2002

Wiley Encyclopedia of Molecular Medicine

View full text Add to dashboard Cite

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

Ellinwood

Vite

Haskins

2004

The Journal of Gene Medicine

116

View full text Add to dashboard Cite

There are more than 40 different forms of inherited lysosomal storage diseases (LSDs) known to occur in humans and the aggregate incidence has been estimated to approach 1 in 7000 live births. Most LSDs are associated with high morbidity and mortality and represent a significant burden on patients, their families, and health care providers. Except for symptomatic therapies, many LSDs remain untreatable, and gene therapy is among the only viable treatment options potentially available. Therapies for some LSDs do exist, or are under evaluation, including heterologous bone marrow transplantation (BMT), enzyme replacement therapy (ERT), and substrate reduction therapy (SRT), but these treatment options are associated with significant concerns, including high morbidity and mortality (BMT), limited positive outcomes (BMT), incomplete response to therapy (BMT, ERT, and SRT), life-long therapy (ERT, SRT), and cost (BMT, ERT, SRT). Gene therapy represents a potential alternative therapy, albeit a therapy with its own attendant concerns. Animal models of LSDs play a critical role in evaluating the efficacy and safety of therapy for many of these conditions. Naturally occurring animal homologs of LSDs have been described in the mouse, rat, dog, cat, guinea pig, emu, quail, goat, cattle, sheep, and pig. In this review we discuss those animal models that have been used in gene therapy experiments and those with promise for future evaluations.

show abstract

Long-term expression and transfer of arylsulfatase A into brain of arylsulfatase A-deficient mice transplanted with bone marrow expressing the arylsulfatase A cDNA from a retroviral vector

Cited by 62 publications

References 27 publications

Bone marrow stem cell-based gene transfer in a mouse model for metachromatic leukodystrophy: effects on visceral and nervous system disease manifestations

Bone marrow stem cell-based gene transfer in a mouse model for metachromatic leukodystrophy: effects on visceral and nervous system disease manifestations

Sulfatases (Sulfohydrolases, Sulfuric Ester Hydrolases)

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

Contact Info

Product

Resources

About