Publications

Coady, T.H., Shababi, M., Tullis, G.E. and Lorson, C.L. (2007). Restoration of SMN function: delivery of a trans-splicing RNA re-directs SMN2 pre-mRNA splicing. Molecular Therapy. 15: 1471-1478.

Zayed, H., Xia, L., Yerich, A., Yan, S.R., Kay, M. A., Puttaraju, M., McGarrity, G.J., Wiest, D.L., McIvor, S., Tolar, and Blazar, B.R. (2007) Correction of DNA protein kinase deficiency by spliceosome-mediated RNA trans-splicing and sleeping beauty transposon delivery. Molecular Therapy 15: 1273-1279.

Yang, Y. and Walsh, C.E. (2005) Spliceosome mediated RNA trans-splicing. Molecular Therapy

Rodriguez-Martin, T., Garcia-Blanco, M.A., Mansfield, S.G., Grover, A.C., Hutton, M., Yu, Q., Zhou, J., Anderton, B.H. and Gallo, J-M. (2005). Reprogramming of tau alternative splicing by spliceosome-mediated RNA trans-splicing: Implications for tauopathies. Proceedings of the National Academy of Sciences USA 102: 15659-15664.

Garcia-Blanco, M.A. (2005). Making antisense of splicing. (2005). Current Opinions in Molecular Therapeutics 7: 476-482.

Mitchell, L.G. and McGarrity, G.J. (2005). Gene therapy progress and prospects: reprogramming gene expression by trans-splicing. Gene Therapy 12: 1477-1485.

Liu, X., Luo, M., Zhang, L.N., Yan, Z., Zak, R., Ding, W., Mansfield, S.G., Mitchell, L.G. and Engelhardt, J.F. (2005). Spliceosome-mediated RNA trans-splicing with recombinant adeno-associated virus partially restores cystic fibrosis transmembrane conductance regulator function to polarized human cystic fibrosis airway epithelial cells. Human Gene Therapy 16: 1116-1123.

Nakayama, K., Pergolizzi, R.G. and Crystal, R.G. (2005). Gene transfer- mediated pre-mRNA segmental trans-splicing as a strategy to deliver intracellular toxins for cancer therapy. Cancer Research 65: 254-263.
Walsh, C.E. (2004). New paradigms for gene transfer: RNA trans-splicing and small interfering RNA as therapeutic strategies. Seminars in Hematology 41: 297-302.

McGarrity, G.J. (2004). SMaRT RNA therapy. Current Drug Discovery (November): 15-19.

Baumik, S., Wall, Z., Puttaraju, M., Mitchell, L.G. and Gambhir, S.S. (2004).

Real-time Molecular Imaging of Gene Expression in Living Subjects
Spliceosome Mediated RNA Trans-splicing. Proceedings National
Academy Sciences USA 101: 8693-8698.

Tahara, M., Pergolizzi, R.G., Kobayashi, A., Luettich, K., Lesser, M.L. and Crystal, R.G. (2004). Trans-splicing repair of CD40 ligand deficiency results in naturally regulated correction of a mouse model of hyper-IgM X-linked immunodeficiency. Nature Medicine 10: 835-841.

Garcia-Blanco, M.A., Baraniak, A.P. and Lasda, E.L. (2004). Alternative
Splicing in Disease and Therapy. Nature Biotechnology 22: 535-546.

Garcia-Blanco, M.A. (2003) Mending the Message. Nature Biotechnology
21: 1448-1449.

Pergolizzi, R., Ropper, A., Dragos, R., Reid, A., Nakayama, K., Tan, Y., Ehteshami, J., Coleman, S., Silver, R., Hackett, N., Menez, A., and Crystal, R. (2003). In Vivo Trans-splicing of 5’ and 3’ Segments of Pre-mRNA Directed by Corresponding DNA Sequences Delivered by Gene Transfer. Molecular Therapy 8: 999-1008.

Schlesinger, J., Arama, D., Noy, H., Dagash, M., Belinky, P., Gross, G. (2003). In-Cell Generation of Antibody Single-Chain Fv Transcripts by Targeted RNA Trans-Splicing. J. Immunological Methods 282: 175-186.

Garcia-Blanco, M. A. (2003). Messenger RNA Reprogramming by Spliceosome-Mediated RNA Trans-Splicing. Journal of Clinical Investigation. 112: 474-480.

Mansfield, S. G., Hawkins-Clark, R., Puttaraju, M., Kole, J., Cohn, J. A., Mitchell, L. G., and Garcia-Blanco, M. A. (2003). 5’ Exon Replacement and Repair by Spliceosome-Mediated RNA Trans-Splicing. RNA 9: 1290-1297.

Chao, H., Mansfield, S. G., Bartel, R., Hiriyanna, S., Mitchell, L. G., Garcia-Blanco, M. A., and Walsh, C. E. (2003). Phenotype Correction of Hemophilia A Mice by Spliceosome-Mediated RNA Trans-Splicing. Nature Medicine 9: 1015-1019.

Otto, E., Temple, G. F., and McGarrity, G. J. (2003). Re-Programming Gene Expression Using Spliceosome-Mediated RNA Trans-Splicing (SMaRT™). Current Drug Discovery (June): 37-42.

Dallinger, G., Puttaraju, M., Mitchell, L. G., Yancey, K. B., Yee, C., Klausegger, A., Hintner, H., and Bauer, J. W. (2003). Development of Spliceosome-Mediated RNA Trans-Splicing (SMaRT™) for the Correction of Inherited Skin Diseases. Experimental Dermatology 12: 37-46.

Liu, X., Jiang, Q., Mansfield, S. G., Puttaraju, M., Zhang, Y., Zhou, W., Garcia-Blanco, M. A., Mitchell, L. G., and Engelhardt, J. F. (2002). Functional Restoration of CFTR Chloride Conductance in Human CF Epithelia by Spliceosome-Mediated RNA Trans-Splicing. Nature Biotechnology 20(1): 47-52.

Crystal, R.G. (2002). Fooling Mother Nature. Nature Biotechnology 20 (1): 32-33.

Puttaraju, M., Mitchell, L. G., and Garcia-Blanco, M. A. (2001). Restoration of Protein Function by Spliceosome Mediated RNA Trans-splicing of an Endogenous Pre-mRNA Target. Molecular Therapy 4(2): 105-114.

Garcia-Blanco, M. A, Puttaraju, M., Mansfield, S. G., and Mitchell, L. G. (2000). Spliceosome-Mediated RNA Trans-Splicing in Gene Therapy and Genomics. Gene Therapy and Regulation 1(2): 141-163.

Dallinger, G., Puttaraju, M., Mitchell, L. G., Yancey, K. B., Hintner, H., and Bauer, J. W. (2000). Collagen 17A1 Gene Correction Using Spliceosome Mediated RNA Trans-Splicing (SMaRT™) Technology. J. Investigative Dermatology 115(2): 332.

Mansfield, S. G, Kole, J., Puttaraju M., Yang, C. C., Garcia-Blanco, M. A., Cohn, J. A., and Mitchell, L. G. (2000). Repair of the Cystic Fibrosis Transmembrane Conductance Regulator mRNA by Spliceosome Mediated RNA Trans-splicing. Gene Therapy 7: 1885-1895.
Puttaraju, M., Jamison, S. F., Mansfield, S. G., Garcia-Blanco, M. A., and Mitchell, L. G. (1999). Spliceosome Mediated Targeted Trans-splicing, a New Tool for Gene Therapy and RNA Repair. Nature BioTechnology 17(3): 246-252.

Kole, R. (1999). A New Routine in Molecular Gymnastics. Nature Biotechnology. 17(3):223-224.

These publications include work by Intronn personnel and collaborations as well as publications by other non-Intronn scientists.