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Items: 24


Spliceosome-Mediated Pre-mRNA trans-Splicing Can Repair CEP290 mRNA.

Dooley SJ, McDougald DS, Fisher KJ, Bennicelli JL, Mitchell LG, Bennett J.

Mol Ther Nucleic Acids. 2018 Sep 7;12:294-308. doi: 10.1016/j.omtn.2018.05.014. Epub 2018 Jun 27.


Amelioration of Neurosensory Structure and Function in Animal and Cellular Models of a Congenital Blindness.

Song JY, Aravand P, Nikonov S, Leo L, Lyubarsky A, Bennicelli JL, Pan J, Wei Z, Shpylchak I, Herrera P, Bennett DJ, Commins N, Maguire AM, Pham J, den Hollander AI, Cremers FPM, Koenekoop RK, Roepman R, Nishina P, Zhou S, Pan W, Ying GS, Aleman TS, de Melo J, McNamara I, Sun J, Mills J, Bennett J.

Mol Ther. 2018 Jun 6;26(6):1581-1593. doi: 10.1016/j.ymthe.2018.03.015. Epub 2018 Mar 21.


Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial.

Bennett J, Wellman J, Marshall KA, McCague S, Ashtari M, DiStefano-Pappas J, Elci OU, Chung DC, Sun J, Wright JF, Cross DR, Aravand P, Cyckowski LL, Bennicelli JL, Mingozzi F, Auricchio A, Pierce EA, Ruggiero J, Leroy BP, Simonelli F, High KA, Maguire AM.

Lancet. 2016 Aug 13;388(10045):661-72. doi: 10.1016/S0140-6736(16)30371-3. Epub 2016 Jun 30.


Correction: AAV-Mediated Gene Therapy for Choroideremia: Preclinical Studies in Personalized Models.

Vasireddy V, Mills JA, Gaddameedi R, Basner-Tschakarjan E, Kohnke M, Black AD, Alexandrov K, Zhou S, Maguire AM, Chung DC, Mac H, Sullivan L, Gadue P, Bennicelli JL, French DL, Bennett J.

PLoS One. 2015 Jun 19;10(6):e0129982. doi: 10.1371/journal.pone.0129982. eCollection 2015. No abstract available.


Stem cells set their sights on retinitis pigmentosa.

Bennicelli JL, Bennett J.

Elife. 2013 Aug 27;2:e01291. doi: 10.7554/eLife.01291.


AAV-mediated gene therapy for choroideremia: preclinical studies in personalized models.

Vasireddy V, Mills JA, Gaddameedi R, Basner-Tschakarjan E, Kohnke M, Black AD, Alexandrov K, Zhou S, Maguire AM, Chung DC, Mac H, Sullivan L, Gadue P, Bennicelli JL, French DL, Bennett J.

PLoS One. 2013 May 7;8(5):e61396. doi: 10.1371/journal.pone.0061396. Print 2013. Erratum in: PLoS One. 2015;10(6):e0129982.


AAV2 gene therapy readministration in three adults with congenital blindness.

Bennett J, Ashtari M, Wellman J, Marshall KA, Cyckowski LL, Chung DC, McCague S, Pierce EA, Chen Y, Bennicelli JL, Zhu X, Ying GS, Sun J, Wright JF, Auricchio A, Simonelli F, Shindler KS, Mingozzi F, High KA, Maguire AM.

Sci Transl Med. 2012 Feb 8;4(120):120ra15. doi: 10.1126/scitranslmed.3002865.


Safety and efficacy of subretinal readministration of a viral vector in large animals to treat congenital blindness.

Amado D, Mingozzi F, Hui D, Bennicelli JL, Wei Z, Chen Y, Bote E, Grant RL, Golden JA, Narfstrom K, Syed NA, Orlin SE, High KA, Maguire AM, Bennett J.

Sci Transl Med. 2010 Mar 3;2(21):21ra16. doi: 10.1126/scitranslmed.3000659.


Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration.

Simonelli F, Maguire AM, Testa F, Pierce EA, Mingozzi F, Bennicelli JL, Rossi S, Marshall K, Banfi S, Surace EM, Sun J, Redmond TM, Zhu X, Shindler KS, Ying GS, Ziviello C, Acerra C, Wright JF, McDonnell JW, High KA, Bennett J, Auricchio A.

Mol Ther. 2010 Mar;18(3):643-50. doi: 10.1038/mt.2009.277. Epub 2009 Dec 1.


Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial.

Maguire AM, High KA, Auricchio A, Wright JF, Pierce EA, Testa F, Mingozzi F, Bennicelli JL, Ying GS, Rossi S, Fulton A, Marshall KA, Banfi S, Chung DC, Morgan JI, Hauck B, Zelenaia O, Zhu X, Raffini L, Coppieters F, De Baere E, Shindler KS, Volpe NJ, Surace EM, Acerra C, Lyubarsky A, Redmond TM, Stone E, Sun J, McDonnell JW, Leroy BP, Simonelli F, Bennett J.

Lancet. 2009 Nov 7;374(9701):1597-605. doi: 10.1016/S0140-6736(09)61836-5. Epub 2009 Oct 23. Erratum in: Lancet. 2010 Jan 2;375(9708):30.


Inducible short-term and stable long-term cell culture systems reveal that the PAX3-FKHR fusion oncoprotein regulates CXCR4, PAX3, and PAX7 expression.

Tomescu O, Xia SJ, Strezlecki D, Bennicelli JL, Ginsberg J, Pawel B, Barr FG.

Lab Invest. 2004 Aug;84(8):1060-70.


Chromosomal translocations and sarcomas.

Bennicelli JL, Barr FG.

Curr Opin Oncol. 2002 Jul;14(4):412-9. Review.


Predominant expression of alternative PAX3 and PAX7 forms in myogenic and neural tumor cell lines.

Barr FG, Fitzgerald JC, Ginsberg JP, Vanella ML, Davis RJ, Bennicelli JL.

Cancer Res. 1999 Nov 1;59(21):5443-8.


Genetics and the biologic basis of sarcomas.

Bennicelli JL, Barr FG.

Curr Opin Oncol. 1999 Jul;11(4):267-74. Review.


Up-regulation of MET but not neural cell adhesion molecule expression by the PAX3-FKHR fusion protein in alveolar rhabdomyosarcoma.

Ginsberg JP, Davis RJ, Bennicelli JL, Nauta LE, Barr FG.

Cancer Res. 1998 Aug 15;58(16):3542-6.


Mechanism for transcriptional gain of function resulting from chromosomal translocation in alveolar rhabdomyosarcoma.

Bennicelli JL, Edwards RH, Barr FG.

Proc Natl Acad Sci U S A. 1996 May 28;93(11):5455-9.


Structural characterization of the FKHR gene and its rearrangement in alveolar rhabdomyosarcoma.

Davis RJ, Bennicelli JL, Macina RA, Nycum LM, Biegel JA, Barr FG.

Hum Mol Genet. 1995 Dec;4(12):2355-62.


Production of multiple cytokines by cultured human melanomas.

Bennicelli JL, Guerry D 4th.

Exp Dermatol. 1993 Aug;2(4):186-90.


Characterization of tenascin secreted by human melanoma cells.

Herlyn M, Graeven U, Speicher D, Sela BA, Bennicelli JL, Kath R, Guerry D 4th.

Cancer Res. 1991 Sep 15;51(18):4853-8.


Human melanoma cells transcribe interleukin 1 genes identical to those of monocytes.

Shabon U, Bennicelli JL, Guerry DP 4th, Koprowski H, Ricciardi RP.

Cancer Res. 1991 Jun 15;51(12):3334-5.


Production of interleukin 1 activity by cultured human melanoma cells.

Bennicelli JL, Elias J, Kern J, Guerry D 4th.

Cancer Res. 1989 Feb 15;49(4):930-5.


Characteristics of cultured human melanocytes isolated from different stages of tumor progression.

Herlyn M, Thurin J, Balaban G, Bennicelli JL, Herlyn D, Elder DE, Bondi E, Guerry D, Nowell P, Clark WH, et al.

Cancer Res. 1985 Nov;45(11 Pt 2):5670-6.

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