cartier science 2009|Hematopoietic Stem Cell Gene Therapy with a : 2024-10-22 Beginning 14 to 16 months after infusion of the genetically corrected cells, progressive cerebral demyelination in the two patients stopped, a clinical outcome comparable to . Register of Historic Places. Under the Historical Resources Act, the Minister .
0 · Lentiviral Vector Gene Therapy: Effective and Safe?
1 · In Brief
2 · Hematopoietic stem cell gene therapy with a lentiviral vector in X
3 · Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in
4 · Hematopoietic Stem Cell Gene Therapy with a Lentiviral
5 · Hematopoietic Stem Cell Gene Therapy with a
6 · Hematopoietic Stem Cell Gene Therapy with Lentiviral
7 · Hematopoietic Stem
8 · A Comeback for Gene Therapy
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cartier science 2009*******X-linked adrenoleukodystrophy (ALD) is a severe brain demyelinating disease in boys that is caused by a deficiency in ALD protein, an adenosine triphosphate-binding . Cartier et al. (p. 818 ; see Perspective by Naldini ) tested an alternative gene therapy–based approach in two young patients without matched donors. A lentiviral vector was used to introduce a wild-type .Beginning 14 to 16 months after infusion of the genetically corrected cells, progressive cerebral demyelination in the two patients stopped, a clinical outcome comparable to .cartier science 2009 Hematopoietic Stem Cell Gene Therapy with a Cartier et al. tested this strategy for treating childhood ALD, a fatal disorder of the central nervous system caused by mutations in ABCD1, a peroxisomal transporter .
Cartier, N, Hacein-Bey-Abina, S, Bartholomae, CC, et al. Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science 2009;326:818-823 CrossrefHematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy Nathalie Cartier, et al. Science 326, 818 (2009); DOI: .
Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy Cartier, N. et al. Science 326, 818–823 (2009) X-linked .Cartier et al. (p. 818; see Perspective by Naldini) tested an alternative gene therapy–based approach in two young patients without matched donors. A lentiviral vector was used to . Cartier N, Hacein-Bey-Abina S, Bartholomae CC, Veres G, Schmidt M, Kutschera I, et al. Hematopoietic stem cell gene therapy with a lentiviral vector in X . In late 2009, we reported that HSC gene therapy with lentiviral vector was able to arrest the progression of cerebral ALD in two boys who have no HLA- matched .
cartier science 2009 X-linked adrenoleukodystrophy (ALD) is a severe brain demyelinating disease in boys that is caused by a deficiency in ALD protein, an adenosine triphosphate-binding cassette transporter encoded by the ABCD1 gene. ALD progression can be halted by allogeneic hematopoietic cell transplantation (HCT). Cartier et al. (p. 818 ; see Perspective by Naldini ) tested an alternative gene therapy–based approach in two young patients without matched donors. A lentiviral vector was used to introduce a wild-type copy of the ALD gene into the patients' hematopoietic stem cells ex vivo.Beginning 14 to 16 months after infusion of the genetically corrected cells, progressive cerebral demyelination in the two patients stopped, a clinical outcome comparable to that achieved by allogeneic HCT. Thus, lentiviral-mediated gene therapy of hematopoietic stem cells can provide clinical benefits in ALD.
Cartier et al. tested this strategy for treating childhood ALD, a fatal disorder of the central nervous system caused by mutations in ABCD1, a peroxisomal transporter gene. The transporter functions in the turnover of myelin (lipid-rich material that insulates neurons) in oligodendrocytes and microglia, and its deficiency leads to demyelination .
Cartier, N, Hacein-Bey-Abina, S, Bartholomae, CC, et al. Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science 2009;326:818-823 CrossrefHematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy Nathalie Cartier, et al. Science 326, 818 (2009); DOI: 10.1126/science.1171242 The following resources related to this article are available online at www.sciencemag.org (this information is current as of November 10, 2009 ): Updated .
Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy Cartier, N. et al. Science 326, 818–823 (2009) X-linked adrenoleukodystrophy (ALD) is a.
Cartier et al. (p. 818; see Perspective by Naldini) tested an alternative gene therapy–based approach in two young patients without matched donors. A lentiviral vector was used to introduce a wild-type copy of the ALD gene into the .
Cartier N, Hacein-Bey-Abina S, Bartholomae CC, Veres G, Schmidt M, Kutschera I, et al. Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science. 2009; 326 :818–823. In late 2009, we reported that HSC gene therapy with lentiviral vector was able to arrest the progression of cerebral ALD in two boys who have no HLA- matched donor to perform HCT.
X-linked adrenoleukodystrophy (ALD) is a severe brain demyelinating disease in boys that is caused by a deficiency in ALD protein, an adenosine triphosphate-binding cassette transporter encoded by the ABCD1 gene. ALD progression can be halted by allogeneic hematopoietic cell transplantation (HCT).
Hematopoietic Stem Cell Gene Therapy with a Cartier et al. (p. 818 ; see Perspective by Naldini ) tested an alternative gene therapy–based approach in two young patients without matched donors. A lentiviral vector was used to introduce a wild-type copy of the ALD gene into the patients' hematopoietic stem cells ex vivo.
Beginning 14 to 16 months after infusion of the genetically corrected cells, progressive cerebral demyelination in the two patients stopped, a clinical outcome comparable to that achieved by allogeneic HCT. Thus, lentiviral-mediated gene therapy of hematopoietic stem cells can provide clinical benefits in ALD.
Cartier et al. tested this strategy for treating childhood ALD, a fatal disorder of the central nervous system caused by mutations in ABCD1, a peroxisomal transporter gene. The transporter functions in the turnover of myelin (lipid-rich material that insulates neurons) in oligodendrocytes and microglia, and its deficiency leads to demyelination . Cartier, N, Hacein-Bey-Abina, S, Bartholomae, CC, et al. Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science 2009;326:818-823 Crossref
Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy Nathalie Cartier, et al. Science 326, 818 (2009); DOI: 10.1126/science.1171242 The following resources related to this article are available online at www.sciencemag.org (this information is current as of November 10, 2009 ): Updated .Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy Cartier, N. et al. Science 326, 818–823 (2009) X-linked adrenoleukodystrophy (ALD) is a.Cartier et al. (p. 818; see Perspective by Naldini) tested an alternative gene therapy–based approach in two young patients without matched donors. A lentiviral vector was used to introduce a wild-type copy of the ALD gene into the .
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cartier science 2009|Hematopoietic Stem Cell Gene Therapy with a