Myelodysplasia after Lentiviral Gene Therapy

Affiliations

¹ University of California, Los Angeles, Los Angeles, CA dkohn1@mednet.ucla.edu.
² UCL Great Ormond Street Institute of Child Health, London, United Kingdom.
³ San Raffaele Telethon Institute for Gene Therapy, Milan, Italy.

Comment

Donald B Kohn et al. N Engl J Med. 2024.

. 2024 Dec 19;391(24):2382.

doi: 10.1056/NEJMc2414069.

¹ University of California, Los Angeles, Los Angeles, CA dkohn1@mednet.ucla.edu.
² UCL Great Ormond Street Institute of Child Health, London, United Kingdom.
³ San Raffaele Telethon Institute for Gene Therapy, Milan, Italy.

No abstract available

Comment on

Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy.
Duncan CN, Bledsoe JR, Grzywacz B, Beckman A, Bonner M, Eichler FS, Kühl JS, Harris MH, Slauson S, Colvin RA, Prasad VK, Downey GF, Pierciey FJ, Kinney MA, Foos M, Lodaya A, Floro N, Parsons G, Dietz AC, Gupta AO, Orchard PJ, Thakar HL, Williams DA. Duncan CN, et al. N Engl J Med. 2024 Oct 10;391(14):1287-1301. doi: 10.1056/NEJMoa2405541. N Engl J Med. 2024. PMID: 39383458 Clinical Trial.
Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy.
Eichler F, Duncan CN, Musolino PL, Lund TC, Gupta AO, De Oliveira S, Thrasher AJ, Aubourg P, Kühl JS, Loes DJ, Amartino H, Smith N, Folloni Fernandes J, Sevin C, Sankar R, Hussain SA, Gissen P, Dalle JH, Platzbecker U, Downey GF, McNeil E, Demopoulos L, Dietz AC, Thakar HL, Orchard PJ, Williams DA. Eichler F, et al. N Engl J Med. 2024 Oct 10;391(14):1302-1312. doi: 10.1056/NEJMoa2400442. N Engl J Med. 2024. PMID: 39383459 Clinical Trial.