CD22ΔE12 as a molecular target for corrective repair using RNA trans-splicing: Anti-leukemic activity of a rationally designed RNA trans-splicing molecule

Integrative Biology (United Kingdom)

Volumn 7, Issue 2, 2015, Pages 237-249

  Uckun, Fatih M ^a,b,c   Qazi, Sanjive ^a,d   Ma, Hong ^a   Reaman, Gregory H ^e   Mitchell, Lloyd G ^f  

^a CHILDREN S HOSPITAL LOS ANGELES   (United States)

^b UNIVERSITY OF SOUTHERN CALIFORNIA   (United States)

^c UNIVERSITY OF SOUTHERN CALIFORNIA   (United States)

^d GUSTAVUS ADOLPHUS COLLEGE   (United States)

^e CHILDREN'S NATIONAL MEDICAL CENTER   (United States)

^f Retrotherapy LLC   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

MESSENGER RNA PRECURSOR; SIALIC ACID BINDING IMMUNOGLOBULIN LIKE LECTIN; CD22 ANTIGEN; CD22 PROTEIN, HUMAN; MESSENGER RNA; RNA;

ACUTE LYMPHOBLASTIC LEUKEMIA; ANTINEOPLASTIC ACTIVITY; ARTICLE; CD22 GENE; CELL LINEAGE; CLONE; CLONOGENESIS; EXON; GENE; GENE DELETION; GENE EXPRESSION PROFILING; GENETIC DISORDER; GENETIC TRANSFECTION; HUMAN; HUMAN CELL; IN VITRO STUDY; INCIDENCE; INTRON; LEUKEMIA; LEUKEMIA CELL; LYMPHATIC SYSTEM TUMOR; LYMPHOMA; MOLECULARLY TARGETED THERAPY; MUTATION; NONHUMAN; PRIORITY JOURNAL; PROTEIN EXPRESSION; QUANTITATIVE ANALYSIS; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; RNA SPLICING; TRANS SPLICING; XENOGRAFT; ADULT; ANIMAL; CHILD; DRUG SCREENING; GENETICS; METABOLISM; MOLECULAR GENETICS; MOUSE; NONOBESE DIABETIC MOUSE; NUCLEOTIDE SEQUENCE; PRECURSOR B-CELL LYMPHOBLASTIC LEUKEMIA-LYMPHOMA; PROCEDURES; REAL TIME POLYMERASE CHAIN REACTION; SCID MOUSE; TARGETED GENE REPAIR; TUMOR CELL CULTURE;

ADULT; ANIMALS; BASE SEQUENCE; CHILD; EXONS; GENE EXPRESSION PROFILING; HUMANS; MICE; MICE, INBRED NOD; MICE, SCID; MOLECULAR SEQUENCE DATA; PRECURSOR B-CELL LYMPHOBLASTIC LEUKEMIA-LYMPHOMA; REAL-TIME POLYMERASE CHAIN REACTION; RNA, MESSENGER; RNA, NEOPLASM; SEQUENCE DELETION; SIALIC ACID BINDING IG-LIKE LECTIN 2; TARGETED GENE REPAIR; TRANS-SPLICING; TRANSFECTION; TUMOR CELLS, CULTURED; TUMOR STEM CELL ASSAY; XENOGRAFT MODEL ANTITUMOR ASSAYS;

EID: 84923070487     PISSN: 17579694     EISSN: 17579708     Source Type: Journal    
DOI: 10.1039/c4ib00221k     Document Type: Article

References (27)

1. T. F. Tedder J. C. Poe K. M. Haas CD22: a multifunctional receptor that regulates B lymphocyte survival and signal transduction Adv. Immunol. 2005 88 1 50
2. J. C. Poe T. F. Tedder CD22 and Siglec-G in B cell function and tolerance Trends Immunol. 2012 33 8 413 420
3. J. Muller L. Nitschke The role of CD22 and Siglec-G in B-cell tolerance and autoimmune disease Nat. Rev. Rheumatol. 2014 10 7 422 428 10.1038/nrrheum.2014.54
4. C. H. Pui C. G. Mullighan W. E. Evans M. V. Relling Pediatric acute lymphoblastic leukemia: where are we going and how do we get there? Blood 2012 120 1165 1174 10.1182/blood-2012-05-378943
5. F. M. Uckun P. Goodman H. Ma I. Dibirdik S. Qazi CD22 exon 12 Deletion as a Novel Pathogenic Mechanism of Human B-Precursor Leukemia Proc. Natl. Acad. Sci. U. S. A. 2010 107 16852 16857
6. H. Ma S. Qazi Z. Ozer P. Gaynon G. H. Reaman F. M. Uckun CD22 exon 12 deletion is a characteristic genetic defect of therapy-refractory clones in paediatric acute lymphoblastic leukaemia Br. J. Haematol. 2014 156 1 89 98 10.1111/j.1365-2141.2011.08901
7. F. M. Uckun S. Qazi H. Ma L. Yin J. J. Cheng A rationally designed nanoparticle for RNA interference therapy in B-lineage lymphoid maligancies EBioMedicine 2014 10.1016/j.ebiom.2014.10.013
8. F. M. Uckun H. Ma J. Zhang Z. Ozer S. Dovat C. Mao R. Ishkhanian P. Goodman S. Qazi Serine phosphorylation by SYK is critical for nuclear localization and transcription factor function of Ikaros Proc. Natl. Acad. Sci. U. S. A. 2012 109 18072 18077 10.1073/pnas.1209828109
9. L. G. Mitchell G. McGarrity Progress & Prospects: Reprograming Gene Expression by Trans-splicing Gene Ther. 2005 12 20 1477 1485
10. M. Tahara R. G. Pergolizzi H. Kobayashi A. Krause K. Luettich M. L. Lesser R. G. Crystal Trans-splicing repair of CD40 ligand deficiency results in naturally regulated correction of a mouse model of hyper-IgM X-linked immunodeficiency Nat. Med. 2004 10 835 841
11. V. Wally A. Klausegger U. Koller H. Lochmüller S. Krause G. Wiche L. G. Mitchell H. Hintner J. W. Bauer 5′ Trans-Splicing Repair of the PLEC1 Gene J. Invest. Dermatol. 2008 128 3 568 574
12. X. Liu Q. Jiang S. G. Mansfield M. Puttaraju Y. Zhang W. Zhou M. A. Garcia-Blanco L. G. Mitchell J. F. Engelhardt Functional Restoration of CFTR Chloride Conductance in Human CF Epithelia by Spliceosome-mediated RNA Trans-splicing Nat. Biotechnol. 2002 20 1 47 52
13. H. Chao S. G. Mansfield R. C. Bartel S. Hiriyanna L. G. Mitchell M. A. Garcia-Blanco C. E. Walsh Phenotype correction of hemophilia A mice by spliceosome-mediated RNA trans-splicing Nat. Med. 2003 9 1015 1019
14. T. H. Coady M. Shababi G. E. Tullis C. L. Lorson Restoration of SMN function: Delivery of a trans-splicing RNA re-directs SMN2 pre-mRNA splicing Mol. Ther. 2007 15 8 1471 1478
15. H. Y. Chen P. Kathirvel W. C. Yee P. S. Lai Correction of dystrophia myotonica type 1 pre-mRNA transcripts by artificial trans-splicing Gene Ther. 2009 16 2 211 217
16. G. Dallinger M. Puttaraju L. G. Mitchell K. B. Yancey C. Yee A. Klausegger H. Hintner J. W. Bauer Development of spliceosome-mediated RNA trans-splicing (SMaRT) for the correction of inherited skin diseases Exp. Dermatol. 2003 12 1 37 46
17. M. Puttaraju S. F. Jamison S. G. Mansfield M. A. Garcia-Blanco L. G. Mitchell Spliceosome mediated targeted Trans-splicing, a new tool for gene therapy and RNA repair Nat. Biotechnol. 1999 17 3 246 252
18. U. Koller V. Wally J. W. Bauer E. M. Murauer Considerations for a Successful RNA Trans-splicing Repair of Genetic Disorders Mol. Ther.-Nucleic Acids 2014 3 e157 10.1038/mtna.2014.10
19. J. P. Venables Aberrant and alternative splicing in cancer Cancer Res. 2004 64 7647 7654
20. D. Baralle M. Baralle Splicing in action: assessing disease causing sequence changes J. Med. Genet. 2005 42 737 748
21. Z. M. Zheng C. C. Baker Papilloma genome structure, expression and post-transcriptional regulation Front. Biosci. 2006 11 2286 2302
22. E. M. Murauer Y. Gache I. K. Gratz A. Klausegger W. Muss C. Gruber G. Meneguzzi H. Hintner J. W. Bauer Functional Correction of Type VII Collagen Expression in Dystrophic Epidermolysis Bullosa J. Invest. Dermatol. 2011 131 1 74 83
23. M. N. Kierlin-Duncan B. A. Sullenger Using 5′ PTMs to repair mutant β-globin transcripts RNA 2007 13 1 11
24. M. E. Avale T. Rodríguez-Martín J. M. Gallo Trans-splicing correction of tau isoform imbalance in a mouse model of tau mis-splicing Hum. Mol. Genet. 2013 22 13 2603 2611 10.1093/hmg/ddt108
25. E. A. Raetz M. S. Cairo M. J. Borowitz X. Lu M. Devidas J. M. Reid et al., Reinduction chemoimmunotherapy with epratuzumab in relapsed acute lymphoblastic leukemia (ALL) in children, adolescents and young adults: results from Children's Oncology Group (COG) study ADVL04P2 Blood 2013 122 355
26. H. Kantarjian D. Thomas J. Jorgensen E. Jabbour P. Kebriaei M. Rytting et al., Inotuzumab ozogamicin, an anti-CD22-calecheamicin conjugate, for refractory and relapsed acute lymphocytic leukaemia: a phase 2 study Lancet Oncol. 2012 13 403 411 10.1016/s1470-2045(11)70386-2
27. O. J. D'Cruz and F. M. Uckun, Novel mAb-based therapies for leukemia, in Monoclonal Antibodies in Oncology, ed., F. M. Uckun, Future Medicine, Future Medicine Ltd, London, 2013, pp. 54-77, 10.2217/ebo.13.218