Letter to the Editor. Differences between lentiviral vector–mediated ex vivo hematopoietic stem cell and adeno-associated virus gene therapies for neurological disorders

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  • 1 Medical Affairs, Orchard Therapeutics, Boston, MA
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Illustration from Serrato-Avila (pp 1410–1423). Copyright Johns Hopkins University, Art as Applied to Medicine. Published with permission.

JNS + Pediatrics - 1 year subscription bundle (Individuals Only)

USD  $515.00

JNS + Pediatrics + Spine - 1 year subscription bundle (Individuals Only)

USD  $612.00
  • 1

    Lonser RR, Akhter AS, Zabek M, Elder JB, Bankiewicz KS. Direct convective delivery of adeno-associated virus gene therapy for treatment of neurological disorders. J Neurosurg. 2021;134(6):17511763.

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  • 2

    Piguet F, Alves S, Cartier N. Clinical gene therapy for neurodegenerative diseases: past, present, and future. Hum Gene Ther. 2017;28(11):9881003.

  • 3

    Fumagalli F, Zambon AA, Rancoita PMV, et al. Metachromatic leukodystrophy: a single-center longitudinal study of 45 patients. J Inherit Metab Dis. 2021;44(5):11511164.

  • 4

    Eichler F, Duncan C, Musolino PL, et al. Hematopoietic stem-cell gene therapy for cerebral adrenoleukodystrophy. N Engl J Med. 2017;377(17):16301638.

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