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. 2020 May 4;41(4):577–593. doi: 10.1210/endrev/bnaa013

Figure 4.

Figure 4.

Treatment protocol for the gene therapy study utilized in the Bluebird Starbeam ALD-102 trial. Abbreviations: ALDP, adrenoleukodystrophy protein; DP, drug product; HSC, hematopoietic stem cell; LTF, long-term follow-up. (Reproduced with permission from Sevin, C. Phase 2/3 trial to assess the safety and efficacy of lenti-D hematopoietic stem cell gene therapy for cerebral adrenoleukodystrophy. Presented at: European Society of Paediatric Neurology, Megaron Athens International Conference Centre, Athens, Greece, September 2019. Oral Abstract Presentation [Abstract OC036]).