CRISPR/Cas9 Gene Editing in Nonhuman Primate Model Demonstrates No Intrinsic Clonal Expansion of PNH HSPCs

Overview

Journal Blood

Publisher Elsevier

Specialty Hematology

Date 2019 Apr 21

PMID 31003997

Citations 12

Authors

Tae-Hoon Shin

Eun Jung Baek

Marcus A F Corat

Shirley Chen

Jean-Yves Metais

Aisha A Aljanahi

Yifan Zhou

Robert E Donahue

Kyung-Rok Yu

Cynthia E Dunbar

Affiliations

Soon will be listed here.

Citing Articles

Impact of CRISPR/HDR editing versus lentiviral transduction on long-term engraftment and clonal dynamics of HSPCs in rhesus macaques.

Lee B, Gin A, Wu C, Singh K, Grice M, Mortlock R Cell Stem Cell. 2024; 31(4):455-466.e4.

PMID: 38508195 PMC: 10997443. DOI: 10.1016/j.stem.2024.02.010.

Case report: Immune pressure on hematopoietic stem cells can drastically expand glycosylphosphatidylinositol-deficient clones in paroxysmal nocturnal hemoglobinuria.

Shingai N, Mizumaki H, Najima Y, Yamada Y, Tran D, Haraguchi K Front Immunol. 2024; 14:1329403.

PMID: 38288112 PMC: 10822943. DOI: 10.3389/fimmu.2023.1329403.

Long noncoding RNA FAM157C contributes to clonal proliferation in paroxysmal nocturnal hemoglobinuria.

Wang H, Liu H, Li L, Chen Y, Liu Z, Li L Ann Hematol. 2023; 102(2):299-309.

PMID: 36607351 PMC: 9889514. DOI: 10.1007/s00277-022-05055-8.

A macaque clonal hematopoiesis model demonstrates expansion of TET2-disrupted clones and utility for testing interventions.

Shin T, Zhou Y, Chen S, Cordes S, Grice M, Fan X Blood. 2022; 140(16):1774-1789.

PMID: 35714307 PMC: 9837449. DOI: 10.1182/blood.2021014875.

Modeling genetic diseases in nonhuman primates through embryonic and germline modification: Considerations and challenges.

Schmidt J, Jones K, Van Vleck T, Emborg M Sci Transl Med. 2022; 14(634):eabf4879.

PMID: 35235338 PMC: 9373237. DOI: 10.1126/scitranslmed.abf4879.

References

Kim M, Yu K, Kenderian S, Ruella M, Chen S, Shin T . Genetic Inactivation of CD33 in Hematopoietic Stem Cells to Enable CAR T Cell Immunotherapy for Acute Myeloid Leukemia. Cell. 2018; 173(6):1439-1453.e19. PMC: 6003425. DOI: 10.1016/j.cell.2018.05.013. View

Hu R, Mukhina G, Piantadosi S, Barber J, Jones R, Brodsky R . PIG-A mutations in normal hematopoiesis. Blood. 2005; 105(10):3848-54. PMC: 1895084. DOI: 10.1182/blood-2004-04-1472. View

Koelle S, Espinoza D, Wu C, Xu J, Lu R, Li B . Quantitative stability of hematopoietic stem and progenitor cell clonal output in rhesus macaques receiving transplants. Blood. 2017; 129(11):1448-1457. PMC: 5356453. DOI: 10.1182/blood-2016-07-728691. View

Araten D, Bessler M, McKenzie S, Castro-Malaspina H, Childs B, Boulad F . Dynamics of hematopoiesis in paroxysmal nocturnal hemoglobinuria (PNH): no evidence for intrinsic growth advantage of PNH clones. Leukemia. 2002; 16(11):2243-8. DOI: 10.1038/sj.leu.2402694. View

Brodsky R, Vala M, Barber J, Medof M, Jones R . Resistance to apoptosis caused by PIG-A gene mutations in paroxysmal nocturnal hemoglobinuria. Proc Natl Acad Sci U S A. 1997; 94(16):8756-60. PMC: 23114. DOI: 10.1073/pnas.94.16.8756. View

Kosicki M, Tomberg K, Bradley A . Repair of double-strand breaks induced by CRISPR-Cas9 leads to large deletions and complex rearrangements. Nat Biotechnol. 2018; 36(8):765-771. PMC: 6390938. DOI: 10.1038/nbt.4192. View

Young N, Maciejewski J . Genetic and environmental effects in paroxysmal nocturnal hemoglobinuria: this little PIG-A goes "Why? Why? Why?". J Clin Invest. 2000; 106(5):637-41. PMC: 381297. DOI: 10.1172/JCI11002. View

Rosti V, Tremml G, Soares V, Pandolfi P, Luzzatto L, Bessler M . Murine embryonic stem cells without pig-a gene activity are competent for hematopoiesis with the PNH phenotype but not for clonal expansion. J Clin Invest. 1997; 100(5):1028-36. PMC: 508277. DOI: 10.1172/JCI119613. View

Luzzatto L, Bessler M, Rotoli B . Somatic mutations in paroxysmal nocturnal hemoglobinuria: a blessing in disguise?. Cell. 1997; 88(1):1-4. DOI: 10.1016/s0092-8674(00)81850-4. View

10.

Papapetrou E, Lee G, Malani N, Setty M, Riviere I, Tirunagari L . Genomic safe harbors permit high β-globin transgene expression in thalassemia induced pluripotent stem cells. Nat Biotechnol. 2010; 29(1):73-8. PMC: 3356916. DOI: 10.1038/nbt.1717. View

11.

Inoue N, Izui-Sarumaru T, Murakami Y, Endo Y, Nishimura J, Kurokawa K . Molecular basis of clonal expansion of hematopoiesis in 2 patients with paroxysmal nocturnal hemoglobinuria (PNH). Blood. 2006; 108(13):4232-6. PMC: 1895453. DOI: 10.1182/blood-2006-05-025148. View

12.

Rotoli B, Luzzatto L . Paroxysmal nocturnal haemoglobinuria. Baillieres Clin Haematol. 1989; 2(1):113-38. DOI: 10.1016/s0950-3536(89)80010-1. View

13.

Visconte V, Raghavachari N, Liu D, Keyvanfar K, Desierto M, Chen J . Phenotypic and functional characterization of a mouse model of targeted Pig-a deletion in hematopoietic cells. Haematologica. 2009; 95(2):214-23. PMC: 2817023. DOI: 10.3324/haematol.2009.011650. View

14.

Cox D, Platt R, Zhang F . Therapeutic genome editing: prospects and challenges. Nat Med. 2015; 21(2):121-31. PMC: 4492683. DOI: 10.1038/nm.3793. View

15.

Ware R, Pickens C, DeCastro C, Howard T . Circulating PIG-A mutant T lymphocytes in healthy adults and patients with bone marrow failure syndromes. Exp Hematol. 2001; 29(12):1403-9. DOI: 10.1016/s0301-472x(01)00746-9. View

16.

Brodsky R . Paroxysmal nocturnal hemoglobinuria. Blood. 2014; 124(18):2804-11. PMC: 4215311. DOI: 10.1182/blood-2014-02-522128. View

17.

Kawagoe K, Kitamura D, Okabe M, Taniuchi I, Ikawa M, Watanabe T . Glycosylphosphatidylinositol-anchor-deficient mice: implications for clonal dominance of mutant cells in paroxysmal nocturnal hemoglobinuria. Blood. 1996; 87(9):3600-6. View

18.

Wu C, Li B, Lu R, Koelle S, Yang Y, Jares A . Clonal tracking of rhesus macaque hematopoiesis highlights a distinct lineage origin for natural killer cells. Cell Stem Cell. 2014; 14(4):486-499. PMC: 3979461. DOI: 10.1016/j.stem.2014.01.020. View

19.

Larochelle A, Dunbar C . Hematopoietic stem cell gene therapy:assessing the relevance of preclinical models. Semin Hematol. 2013; 50(2):101-30. PMC: 3761403. DOI: 10.1053/j.seminhematol.2013.03.025. View

20.

Young N . Paroxysmal nocturnal hemoglobinuria and myelodysplastic syndromes: clonal expansion of PIG-A-mutant hematopoietic cells in bone marrow failure. Haematologica. 2009; 94(1):3-7. PMC: 2625409. DOI: 10.3324/haematol.2008.001297. View