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25 results on '"Kelleher, Michelle"'

1. Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial

Author

Palfi, Stéphane, Gurruchaga, Jean Marc, Ralph, G Scott, Lepetit, Helene, Lavisse, Sonia, Buttery, Philip C, Watts, Colin, Miskin, James, Kelleher, Michelle, Deeley, Sarah, Iwamuro, Hirokazu, Lefaucheur, Jean Pascal, Thiriez, Claire, Fenelon, Gilles, Lucas, Cherry, Brugières, Pierre, Gabriel, Inanna, Abhay, Kou, Drouot, Xavier, Tani, Naoki, Kas, Aurelie, Ghaleh, Bijan, Le Corvoisier, Philippe, Dolphin, Patrice, Breen, David P, Mason, Sarah, Guzman, Natalie Valle, Mazarakis, Nicholas D, Radcliffe, Pippa A, Harrop, Richard, Kingsman, Susan M, Rascol, Olivier, Naylor, Stuart, Barker, Roger A, Hantraye, Philippe, Remy, Philippe, Cesaro, Pierre, and Mitrophanous, Kyriacos A

Published
2014
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5. Gene Therapy for Parkinson’s Disease: Preclinical Evaluation of Optimally Configured TH:CH1 Fusion for Maximal Dopamine Synthesis

Author

Badin, Romina Aron, primary, Binley, Katie, additional, Van Camp, Nadja, additional, Jan, Caroline, additional, Gourlay, Jeanne, additional, Robert, Camille, additional, Gipchtein, Pauline, additional, Fayard, Audrey, additional, Stewart, Hannah, additional, Ralph, G. Scott, additional, Lad, Yatish, additional, Kelleher, Michelle, additional, Loader, Julie, additional, Hosomi, Koichi, additional, Palfi, Stéphane, additional, Mitrophanous, Kyriacos A., additional, and Hantraye, Philippe, additional

Published
2019
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9. 200. Advancing a State of the Art Gene Therapy for Parkinson's Disease

Author

Aron Badin, Romina, primary, Binley, Katie, additional, Van Camp, Nadja, additional, Jan, Caroline, additional, Gourlay, Jean, additional, Stewart, Hannah, additional, Ralph, Scott, additional, Lad, Yatish, additional, Kelleher, Michelle, additional, Loader, Julie, additional, Hosomi, Koichi, additional, Palfi, Stephane, additional, Hantraye, Phillippe, additional, and Mitrophanous, Kyriacos, additional

Published
2015
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10. EIAV-Based Retinal Gene Therapy in the shaker1 Mouse Model for Usher Syndrome Type 1B: Development of UshStat

Author

Zallocchi, Marisa, primary, Binley, Katie, additional, Lad, Yatish, additional, Ellis, Scott, additional, Widdowson, Peter, additional, Iqball, Sharifah, additional, Scripps, Vicky, additional, Kelleher, Michelle, additional, Loader, Julie, additional, Miskin, James, additional, Peng, You-Wei, additional, Wang, Wei-Min, additional, Cheung, Linda, additional, Delimont, Duane, additional, Mitrophanous, Kyriacos A., additional, and Cosgrove, Dominic, additional

Published
2014
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11. Transduction of Photoreceptors With Equine Infectious Anemia Virus Lentiviral Vectors: Safety and Biodistribution of StarGen for Stargardt Disease

Author

Binley, Katie, primary, Widdowson, Peter, additional, Loader, Julie, additional, Kelleher, Michelle, additional, Iqball, Sharifah, additional, Ferrige, Georgina, additional, de Belin, Jackie, additional, Carlucci, Marie, additional, Angell-Manning, Diana, additional, Hurst, Felicity, additional, Ellis, Scott, additional, Miskin, James, additional, Fernandes, Alcides, additional, Wong, Paul, additional, Allikmets, Rando, additional, Bergstrom, Christopher, additional, Aaberg, Thomas, additional, Yan, Jiong, additional, Kong, Jian, additional, Gouras, Peter, additional, Prefontaine, Annick, additional, Vezina, Mark, additional, Bussieres, Martin, additional, Naylor, Stuart, additional, and Mitrophanous, Kyriacos A., additional

Published
2013
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12. Safety and Biodistribution of an Equine Infectious Anemia Virus-Based Gene Therapy, RetinoStat®, for Age-Related Macular Degeneration

Author

Binley, Katie, primary, Widdowson, Peter S., additional, Kelleher, Michelle, additional, de Belin, Jackie, additional, Loader, Julie, additional, Ferrige, Georgina, additional, Carlucci, Marie, additional, Esapa, Margaret, additional, Chipchase, Daniel, additional, Angell-Manning, Diana, additional, Ellis, Scott, additional, Mitrophanous, Kyriacos, additional, Miskin, James, additional, Bantseev, Vlad, additional, Nork, T. Michael, additional, Miller, Paul, additional, and Naylor, Stuart, additional

Published
2012
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13. A Stable Producer Cell Line for the Manufacture of a Lentiviral Vector for Gene Therapy of Parkinson's Disease

Author

Stewart, Hannah J., primary, Fong-Wong, Liang, additional, Strickland, Iain, additional, Chipchase, Daniel, additional, Kelleher, Michelle, additional, Stevenson, Laura, additional, Thoree, Vinay, additional, McCarthy, Janine, additional, Ralph, G. Scott, additional, Mitrophanous, Kyriacos A., additional, and Radcliffe, Pippa A., additional

Published
2011
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17. Vaccination Against the Tumour Associated Antigen 5T4 Induces Immune Responses in Advanced Colorectal Cancer Patients: Results Phase I/II Clinical Trial with Recombinant Modified Vaccinia Virus Ankara(TroVax)

Author

Hawkins, Robert, primary, Connolly, Noel, additional, Harrop, Richard, additional, Redchenko, Irina, additional, Ryan, Matthew, additional, Drury, Noel, additional, Kelleher, Michelle, additional, Kingsman, Sue, additional, and Carroll, Miles, additional

Published
2004
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19. EIAV-Based Retinal Gene Therapy in the shaker1 Mouse Model for Usher Syndrome Type 1B: Development of UshStat.

Author

Zallocchi, Marisa, Binley, Katie, Lad, Yatish, Ellis, Scott, Widdowson, Peter, Iqball, Sharifah, Scripps, Vicky, Kelleher, Michelle, Loader, Julie, Miskin, James, Peng, You-Wei, Wang, Wei-Min, Cheung, Linda, Delimont, Duane, Mitrophanous, Kyriacos A., and Cosgrove, Dominic

Subjects
GENE therapy, TREATMENT of eye diseases, RETINAL diseases, LABORATORY mice, USHER'S syndrome, RHODOPSIN, MICROBIOLOGY, MOLECULAR biology
Abstract

Usher syndrome type 1B is a combined deaf-blindness condition caused by mutations in the MYO7A gene. Loss of functional myosin VIIa in the retinal pigment epithelia (RPE) and/or photoreceptors leads to blindness. We evaluated the impact of subretinally delivered UshStat, a recombinant EIAV-based lentiviral vector expressing human MYO7A, on photoreceptor function in the shaker1 mouse model for Usher type 1B that lacks a functional Myo7A gene. Subretinal injections of EIAV-CMV-GFP, EIAV-RK-GFP (photoreceptor specific), EIAV-CMV-MYO7A (UshStat) or EIAV-CMV-Null (control) vectors were performed in shaker1 mice. GFP and myosin VIIa expression was evaluated histologically. Photoreceptor function in EIAV-CMV-MYO7A treated eyes was determined by evaluating α-transducin translocation in photoreceptors in response to low light intensity levels, and protection from light induced photoreceptor degeneration was measured. The safety and tolerability of subretinally delivered UshStat was evaluated in macaques. Expression of GFP and myosin VIIa was confirmed in the RPE and photoreceptors in shaker1 mice following subretinal delivery of the EIAV-CMV-GFP/MYO7A vectors. The EIAV-CMV-MYO7A vector protected the shaker1 mouse photoreceptors from acute and chronic intensity light damage, indicated by a significant reduction in photoreceptor cell loss, and restoration of the α-transducin translocation threshold in the photoreceptors. Safety studies in the macaques demonstrated that subretinal delivery of UshStat is safe and well-tolerated. Subretinal delivery of EIAV-CMV-MYO7A (UshStat) rescues photoreceptor phenotypes in the shaker1 mouse. In addition, subretinally delivered UshStat is safe and well-tolerated in macaque safety studies These data support the clinical development of UshStat to treat Usher type 1B syndrome. [ABSTRACT FROM AUTHOR]

Published
2014
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24. Is tweet little Niall set to wear down Glenda's resolve?

Author

Lynne Kelleher ;Michelle O'Keeffe

Abstract

NIALL Horan might have the angelic good looks, but he certainly has an eye for the ladies as well - and still has Glenda Gilson in his sights. [ABSTRACT FROM PUBLISHER]

Published
2012

25. Gene Therapy for Parkinson's Disease: Preclinical Evaluation of Optimally Configured TH:CH1 Fusion for Maximal Dopamine Synthesis.

Author

Badin RA, Binley K, Van Camp N, Jan C, Gourlay J, Robert C, Gipchtein P, Fayard A, Stewart H, Ralph GS, Lad Y, Kelleher M, Loader J, Hosomi K, Palfi S, Mitrophanous KA, and Hantraye P

Abstract

A recent phase I-II, open-label trial of ProSavin, a lentiviral vector delivering the key enzymes in the dopamine biosynthetic pathway to non-dopaminergic striatal neurons, demonstrated safety and improved motor function in parkinsonian patients. However, the magnitude of the effect suggested that optimal levels of dopamine replacement may not have been achieved. OXB-102, a lentiviral vector with an optimized expression cassette for dopamine biosynthesis, has been shown to achieve a significantly higher dopamine yield than ProSavin. We assessed the efficacy of OXB-102 in the MPTP macaque model of Parkinson's disease (PD). At 6 months post-vector administration, all treated animals showed significant improvements in clinical scores and spontaneous locomotor activity compared to controls, with the highest recovery observed in the OXB-102 high-dose (HD) group. Positron emission tomography quantification of 6-[ 18 F]-fluoro-L-m-tyrosine uptake showed a significant increase in amino acid decarboxylase activity for all treated animals, compared with controls, where the OXB-102 HD group showed the highest level of dopaminergic activity. A toxicology study in macaques demonstrated that the vector was safe and well tolerated, with no associated clinical or behavioral abnormalities and no immune response mounted against any transgene products. Overall, these data support the further clinical development of OXB-102 for the treatment of PD.

Published
2019
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