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

Denise A. Carbonaro-Sarracino PhD1, Robin LeWinter PhD1, and Leslie Meltzer PhD1
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  • 1 Medical Affairs, Orchard Therapeutics, Boston, MA
Online Publication Date:
10 Dec 2021
Page Range:
1501
Volume/Issue:
Volume 136: Issue 5
DOI link:
https://doi.org/10.3171/2021.10.JNS212456
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Volume 136: Issue 5 (May 2022)

in Journal of Neurosurgery

Illustration from Serrato-Avila (pp 1410–1423). Copyright Johns Hopkins University, Art as Applied to Medicine. Published with permission.

Cover Journal of Neurosurgery

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Contributor Notes

Correspondence Robin LeWinter: robin.lewinter@orchard-tx.com.

INCLUDE WHEN CITING Published online December 10, 2021; DOI: 10.3171/2021.10.JNS212456.

Disclosures All authors are employees of Orchard Therapeutics. Orchard Therapeutics is the sponsor for study NCT03392987 and has provided funding for study NCT04201405, discussed above.

Page Count:
2
  • 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):1751–1763.

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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):988–1003.

  • 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):1151–1164.

  • 4

    Eichler F, Duncan C, Musolino PL, et al. Hematopoietic stem-cell gene therapy for cerebral adrenoleukodystrophy. N Engl J Med. 2017;377(17):1630–1638.

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