Targeted gene addition in human CD34+ hematopoietic cells for correction of X-linked chronic granulomatous disease

Nature Biotechnology - Tập 34 Số 4 - Trang 424-429 - 2016
Suk See De Ravin1, Andreas Reik2, Peiqi Liu2, Linhong Li3, Xiaolin Wu4, Ling Su4, Castle Raley4, Narda Theobald1, Uimook Choi1, Alexander H. Song2, Andy Chan2, Jocelynn Pearl2, David E. Paschon2, Janet Lee1, Hannah Newcombe1, Sherry Koontz1, Colin L. Sweeney1, David A. Shivak2, Kol A. Zarember1, Madhusudan V. Peshwa4, Philip D. Gregory2, Fyodor D. Urnov2, Harry L. Malech1
1Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland, USA
2Sangamo BioSciences Inc., Richmond, California, USA
3MaxCyte Inc., Gaithersburg, Maryland, USA
4Cancer Research Technology Program, Leidos Biomedical Research, Inc., Frederick, Maryland, USA

Tóm tắt

Từ khóa


Tài liệu tham khảo

Biffi, A. et al. Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy. Science 341, 1233158 (2013).

Hacein-Bey-Abina, S. et al. Efficacy of gene therapy for X-linked severe combined immunodeficiency. N. Engl. J. Med. 363, 355–364 (2010).

Cavazzana-Calvo, M. et al. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 288, 669–672 (2000).

Hacein-Bey-Abina, S. et al. A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N. Engl. J. Med. 348, 255–256 (2003).

Stein, S. et al. Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease. Nat. Med. 16, 198–204 (2010).

Carroll, D. Genome engineering with targetable nucleases. Annu. Rev. Biochem. 83, 409–439 (2014).

Tebas, P. et al. Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV. N. Engl. J. Med. 370, 901–910 (2014).

DeKelver, R.C. et al. Functional genomics, proteomics, and regulatory DNA analysis in isogenic settings using zinc finger nuclease-driven transgenesis into a safe harbor locus in the human genome. Genome Res. 20, 1133–1142 (2010).

Genovese, P. et al. Targeted genome editing in human repopulating haematopoietic stem cells. Nature 510, 235–240 (2014).

Carroll, D. & Beumer, K.J. Genome engineering with TALENs and ZFNs: repair pathways and donor design. Methods 69, 137–141 (2014).

Urnov, F.D. et al. Highly efficient endogenous human gene correction using designed zinc-finger nucleases. Nature 435, 646–651 (2005).

Moehle, E.A. et al. Targeted gene addition into a specified location in the human genome using designed zinc finger nucleases. Proc. Natl. Acad. Sci. USA 104, 3055–3060 (2007).

Vierstra, J. et al. Functional footprinting of regulatory DNA. Nat. Methods 12, 927–930 (2015).

Song, L. et al. Optimizing the transduction efficiency of capsid-modified AAV6 serotype vectors in primary human hematopoietic stem cells in vitro and in a xenograft mouse model in vivo. Cytotherapy 15, 986–998 (2013).

Song, L. et al. High-efficiency transduction of primary human hematopoietic stem cells and erythroid lineage-restricted expression by optimized AAV6 serotype vectors in vitro and in a murine xenograft model in vivo. PLoS One 8, e58757 (2013).

Orlando, S.J. et al. Zinc-finger nuclease-driven targeted integration into mammalian genomes using donors with limited chromosomal homology. Nucleic Acids Res. 38, e152 (2010).

Kuhns, D.B. et al. Residual NADPH oxidase and survival in chronic granulomatous disease. N. Engl. J. Med. 363, 2600–2610 (2010).

Marciano, B.E. et al. Common severe infections in chronic granulomatous disease. Clin. Infect. Dis. 60, 1176–1183 (2015).

Challita, P.M. et al. Multiple modifications in cis elements of the long terminal repeat of retroviral vectors lead to increased expression and decreased DNA methylation in embryonic carcinoma cells. J. Virol. 69, 748–755 (1995).

Astrakhan, A. et al. Ubiquitous high-level gene expression in hematopoietic lineages provides effective lentiviral gene therapy of murine Wiskott-Aldrich syndrome. Blood 119, 4395–4407 (2012).

Wang, J. et al. Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors. Nat. Biotechnol. 33, 1256–1263 (2015).

Langmead, B. & Salzberg, S.L. Fast gapped-read alignment with Bowtie 2. Nat. Methods 9, 357–359 (2012).

Gabriel, R. et al. An unbiased genome-wide analysis of zinc-finger nuclease specificity. Nat. Biotechnol. 29, 816–823 (2011).

Brenner, S. et al. Concentrated RD114-pseudotyped MFGS-gp91phox vector achieves high levels of functional correction of the chronic granulomatous disease oxidase defect in NOD/SCID/β2-microglobulin−/− repopulating mobilized human peripheral blood CD34+ cells. Blood 102, 2789–2797 (2003).