Gene therapy for lysosomal storage diseases

Mark S. Sands; Beverly L. Davidson

doi:10.1016/j.ymthe.2006.01.006

Gene therapy for lysosomal storage diseases

Mark S. Sands, Beverly L. Davidson

Research output: Contribution to journal › Review article › peer-review

227 Scopus citations

Abstract

Lysosomal storage diseases (LSDs) comprise a diverse group of monogenetic disorders with complex clinical phenotypes that include both systemic and central nervous system pathologies. In recent years, the identification or development of mouse models recapitulating the clinical course of the LSDs has been instrumental in evaluating therapeutic strategies. Here, we review the various gene replacement strategies for target organs affected in many LSDs and describe briefly the various vector systems employed to test how best to accomplish long-lasting therapies for these fatal disorders.

Original language	English
Pages (from-to)	839-849
Number of pages	11
Journal	Molecular Therapy
Volume	13
Issue number	5
DOIs	https://doi.org/10.1016/j.ymthe.2006.01.006
State	Published - May 2006

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10.1016/j.ymthe.2006.01.006

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title = "Gene therapy for lysosomal storage diseases",

abstract = "Lysosomal storage diseases (LSDs) comprise a diverse group of monogenetic disorders with complex clinical phenotypes that include both systemic and central nervous system pathologies. In recent years, the identification or development of mouse models recapitulating the clinical course of the LSDs has been instrumental in evaluating therapeutic strategies. Here, we review the various gene replacement strategies for target organs affected in many LSDs and describe briefly the various vector systems employed to test how best to accomplish long-lasting therapies for these fatal disorders.",

author = "Sands, {Mark S.} and Davidson, {Beverly L.}",

note = "Funding Information: The authors would like to acknowledge grant support for this work, NIH NS043205 and NIH DK05786 (MSS), NIH NS34568 and NIH HD33531 (BLD).",

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AU - Sands, Mark S.

AU - Davidson, Beverly L.

N1 - Funding Information: The authors would like to acknowledge grant support for this work, NIH NS043205 and NIH DK05786 (MSS), NIH NS34568 and NIH HD33531 (BLD).

PY - 2006/5

Y1 - 2006/5

N2 - Lysosomal storage diseases (LSDs) comprise a diverse group of monogenetic disorders with complex clinical phenotypes that include both systemic and central nervous system pathologies. In recent years, the identification or development of mouse models recapitulating the clinical course of the LSDs has been instrumental in evaluating therapeutic strategies. Here, we review the various gene replacement strategies for target organs affected in many LSDs and describe briefly the various vector systems employed to test how best to accomplish long-lasting therapies for these fatal disorders.

AB - Lysosomal storage diseases (LSDs) comprise a diverse group of monogenetic disorders with complex clinical phenotypes that include both systemic and central nervous system pathologies. In recent years, the identification or development of mouse models recapitulating the clinical course of the LSDs has been instrumental in evaluating therapeutic strategies. Here, we review the various gene replacement strategies for target organs affected in many LSDs and describe briefly the various vector systems employed to test how best to accomplish long-lasting therapies for these fatal disorders.

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DO - 10.1016/j.ymthe.2006.01.006

M3 - Review article

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SP - 839

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JO - Molecular Therapy

JF - Molecular Therapy

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ER -

Gene therapy for lysosomal storage diseases

Abstract

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