Lentiviral hematopoietic stem cell gene therapy in inherited metabolic disorders

doi:10.1089/hum.2014.102

Review

. 2014 Oct;25(10):862-5.

doi: 10.1089/hum.2014.102. Epub 2014 Sep 17.

Lentiviral hematopoietic stem cell gene therapy in inherited metabolic disorders

Gerard Wagemaker¹

Affiliations

PMID: 25184354
PMCID: PMC4179920
DOI: 10.1089/hum.2014.102

Review

Lentiviral hematopoietic stem cell gene therapy in inherited metabolic disorders

Gerard Wagemaker. Hum Gene Ther. 2014 Oct.

. 2014 Oct;25(10):862-5.

doi: 10.1089/hum.2014.102. Epub 2014 Sep 17.

Author

Gerard Wagemaker¹

Affiliation

¹ Erasmus University Rotterdam, 3005 LA Rotterdam, The Netherlands .

PMID: 25184354
PMCID: PMC4179920
DOI: 10.1089/hum.2014.102

Abstract

After more than 20 years of development, lentiviral hematopoietic stem cell gene therapy has entered the stage of initial clinical implementation for immune deficiencies and storage disorders. This brief review summarizes the development and applications, focusing on the lysosomal enzyme deficiencies, especially Pompe disease.

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References

1. Aiuti A., Cassani B., Andolfi G., et al. (2007). Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy. J. Clin. Invest. 117, 2233–2240 - PMC - PubMed
1. Baum C., von Kalle C., Staal F.J., et al. (2004). Chance or necessity? Insertional mutagenesis in gene therapy and its consequences. Mol. Ther. 9, 5–13 - PubMed
1. Biasco L., Ambrosi A., Pellin D., et al. (2011). Integration profile of retroviralvector in gene therapy treated patients is cell-specific according to gene expression and chromatin conformation of target cell. EMBO Mol. Med. 3, 89–101 - PMC - PubMed
1. Biffi A., Montini E., Lorioli L., et al. (2013). Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy. Science 341, 1233158. - PubMed
1. Braun C.J., Boztug K., Paruzynski A., et al. (2014). Gene therapy for Wiskott-Aldrichsyndrome—long-term efficacy and genotoxicity. Sci. Transl. Med. 6, 227ra33 - PubMed

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[1] Aiuti A., Cassani B., Andolfi G., et al. (2007). Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy. J. Clin. Invest. 117, 2233–2240 - PMC - PubMed

[2] Aiuti A., Cassani B., Andolfi G., et al. (2007). Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy. J. Clin. Invest. 117, 2233–2240 - PMC - PubMed

[3] Baum C., von Kalle C., Staal F.J., et al. (2004). Chance or necessity? Insertional mutagenesis in gene therapy and its consequences. Mol. Ther. 9, 5–13 - PubMed

[4] Baum C., von Kalle C., Staal F.J., et al. (2004). Chance or necessity? Insertional mutagenesis in gene therapy and its consequences. Mol. Ther. 9, 5–13 - PubMed

[5] Biasco L., Ambrosi A., Pellin D., et al. (2011). Integration profile of retroviralvector in gene therapy treated patients is cell-specific according to gene expression and chromatin conformation of target cell. EMBO Mol. Med. 3, 89–101 - PMC - PubMed

[6] Biasco L., Ambrosi A., Pellin D., et al. (2011). Integration profile of retroviralvector in gene therapy treated patients is cell-specific according to gene expression and chromatin conformation of target cell. EMBO Mol. Med. 3, 89–101 - PMC - PubMed

[7] Biffi A., Montini E., Lorioli L., et al. (2013). Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy. Science 341, 1233158. - PubMed

[8] Biffi A., Montini E., Lorioli L., et al. (2013). Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy. Science 341, 1233158. - PubMed

[9] Braun C.J., Boztug K., Paruzynski A., et al. (2014). Gene therapy for Wiskott-Aldrichsyndrome—long-term efficacy and genotoxicity. Sci. Transl. Med. 6, 227ra33 - PubMed

[10] Braun C.J., Boztug K., Paruzynski A., et al. (2014). Gene therapy for Wiskott-Aldrichsyndrome—long-term efficacy and genotoxicity. Sci. Transl. Med. 6, 227ra33 - PubMed

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Lentiviral hematopoietic stem cell gene therapy in inherited metabolic disorders

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Lentiviral hematopoietic stem cell gene therapy in inherited metabolic disorders

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