Biblio

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Journal Article

Eaker S, Armant M, Brandwein H, Burger S, Campbell A, Carpenito C, Clarke D, Fong T, Karnieli O, Niss K, et al. Concise Review: Guidance in Developing Commercializable Autologous/Patient-Specific Cell Therapy Manufacturing. Stem Cells Transl Med. 2013.

Jurkunas UV, Yin J, Johns LK, Li S, Negre H, Shaw KL, Samarakoon L, Ayala AR, Kheirkhah A, Katikireddy K, et al. Cultivated autologous limbal epithelial cell (CALEC) transplantation: Development of manufacturing process and clinical evaluation of feasibility and safety. Sci Adv. 2023;9(33):eadg6470.

Rao S, Yao Y, de Brito JSoares, Yao Q, Shen AH, Watkinson RE, Kennedy AL, Coyne S, Ren C, Zeng J, et al. Dissecting ELANE neutropenia pathogenicity by human HSC gene editing. Cell Stem Cell. 2021.

Cancellieri S, Zeng J, Lin LYingqi, Tognon M, Nguyen MAnh, Lin J, Bombieri N, Maitland SA, Ciuculescu M-F, Katta V, et al. Human genetic diversity alters off-target outcomes of therapeutic gene editing. Nat Genet. 2022.

Tatetsu H, Gao C, Armant M, Wang F, Ueno S, Tian X, Federation A, Qi J, Bradner J, Tenen DG, et al. Maintenance and enhancement of human PBSC engraftment after ex vivo culture via an HDACi/SALL4 axis (3465). Exp Hematol. 2019.

Labrosse R, Chu J, Armant M, Everett JK, Pellin D, Kareddy N, Frelinger AL, Henderson LA, O'Connell AE, Biswas A, et al. Outcomes of Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome. Blood. 2023.

Esrick EB, Lehmann LE, Biffi A, Achebe M, Brendel C, Ciuculescu MF, Daley H, MacKinnon B, Morris E, Federico A, et al. Post-Transcriptional Genetic Silencing of to Treat Sickle Cell Disease. N Engl J Med. 2020.