Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy

  title={Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy},
  author={Nathalie Cartier and Salima Hacein-Bey-Abina and Cynthia C. Bartholomae and Gabor Istvan Veres and Manfred Schmidt and Ina Kutschera and Michel Vidaud and Ulrich Abel and Liliane Dal-Cortivo and Laure Caccavelli and Nizar Mahlaoui and V{\'e}ronique Kiermer and Denice M. Mittelstaedt and C{\'e}line Bellesme and Najiba Lahlou and François Lefrère and St{\'e}phane Blanche and Muriel Audit and Emmanuel Payen and Philippe Leboulch and Bruno L’homme and Pierre Bougn{\`e}res and Christof von Kalle and Alain Fischer and Marina Cavazzana‐Calvo and Patrick R. Aubourg},
  pages={818 - 823}
Slowing Brain Disease with Gene Therapy X-linked adrenoleukodystrophy (ALD), the hereditary brain demyelinating disorder that was featured in the movie “Lorenzo's Oil,” is typically treated by transplantation of bone marrow from matched donors. This treatment slows progression of the disease by introducing cells that differentiate into myelin-producing cells. Cartier et al. (p. 818; see Perspective by Naldini) tested an alternative gene therapy–based approach in two young patients without… 

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