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Hematopoietic stem-cell gene therapy is associated with restored white matter microvascular function in cerebral adrenoleukodystrophy.
Lauer A, Speroni SL, Choi M, Da X, Duncan C, McCarthy S, Krishnan V, Lusk CA, Rohde D, Hansen MB, Kalpathy-Cramer J, Loes DJ, Caruso PA, Williams DA, Mouridsen K, Emblem KE, Eichler FS, Musolino PL. Lauer A, et al. Among authors: duncan c. Nat Commun. 2023 Apr 5;14(1):1900. doi: 10.1038/s41467-023-37262-w. Nat Commun. 2023. PMID: 37019892 Free PMC article.
Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy.
Eichler F, Duncan C, Musolino PL, Orchard PJ, De Oliveira S, Thrasher AJ, Armant M, Dansereau C, Lund TC, Miller WP, Raymond GV, Sankar R, Shah AJ, Sevin C, Gaspar HB, Gissen P, Amartino H, Bratkovic D, Smith NJC, Paker AM, Shamir E, O'Meara T, Davidson D, Aubourg P, Williams DA. Eichler F, et al. Among authors: duncan c. N Engl J Med. 2017 Oct 26;377(17):1630-1638. doi: 10.1056/NEJMoa1700554. Epub 2017 Oct 4. N Engl J Med. 2017. PMID: 28976817 Free PMC article. Clinical Trial.
Gene Therapy for Cerebral Adrenoleukodystrophy.
Williams DA, Eichler F, Duncan C. Williams DA, et al. Among authors: duncan c. N Engl J Med. 2018 Feb 1;378(5):490-491. doi: 10.1056/NEJMc1715427. N Engl J Med. 2018. PMID: 29385368 No abstract available.
The Changing Face of Adrenoleukodystrophy.
Zhu J, Eichler F, Biffi A, Duncan CN, Williams DA, Majzoub JA. Zhu J, et al. Endocr Rev. 2020 Aug 1;41(4):577-93. doi: 10.1210/endrev/bnaa013. Endocr Rev. 2020. PMID: 32364223 Free PMC article. Review.
Variables affecting outcomes after allogeneic hematopoietic stem cell transplant for cerebral adrenoleukodystrophy.
Chiesa R, Boelens JJ, Duncan CN, Kühl JS, Sevin C, Kapoor N, Prasad VK, Lindemans CA, Jones SA, Amartino HM, Algeri M, Bunin N, Diaz-de-Heredia C, Loes DJ, Shamir E, Timm A, McNeil E, Dietz AC, Orchard PJ. Chiesa R, et al. Among authors: duncan cn. Blood Adv. 2022 Mar 8;6(5):1512-1524. doi: 10.1182/bloodadvances.2021005294. Blood Adv. 2022. PMID: 34781360 Free PMC article.
Gene Therapy for Pediatric Neurologic Disease.
Jimenez-Kurlander L, Duncan CN. Jimenez-Kurlander L, et al. Hematol Oncol Clin North Am. 2022 Aug;36(4):853-864. doi: 10.1016/j.hoc.2022.05.003. Epub 2022 Jun 25. Hematol Oncol Clin North Am. 2022. PMID: 35760664 Review.
1,869 results