Gene therapy restores vision in rd1 mice after removal of a confounding mutation in Gpr179
Nishiguchi, Koji M., Carvalho, Livia S., Rizzi, Matteo, Powell, Kate, Holthaus, Sophia-Martha kleine, Azam, Selina A., Duran, Yanai, Ribeiro, Joana, Luhmann, Ulrich F. O., Bainbridge, James W. B., Smith, Alexander J., Ali, Robin R.
Published in Nature communications (23.01.2015)
Published in Nature communications (23.01.2015)
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Long-term preservation of cones and improvement in visual function following gene therapy in a mouse model of leber congenital amaurosis caused by guanylate cyclase-1 deficiency
Mihelec, Marija, Pearson, Rachael A, Robbie, Scott J, Buch, Prateek K, Azam, Selina A, Bainbridge, James W B, Smith, Alexander J, Ali, Robin R
Published in Human gene therapy (01.10.2011)
Published in Human gene therapy (01.10.2011)
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Successful gene therapy in older Rpe65-deficient dogs following subretinal injection of an adeno-associated vector expressing RPE65
Annear, Matthew J, Mowat, Freya M, Bartoe, Joshua T, Querubin, Janice, Azam, Selina A, Basche, Mark, Curran, Paul G, Smith, Alexander J, Bainbridge, James W B, Ali, Robin R, Petersen-Jones, Simon M
Published in Human gene therapy (01.10.2013)
Published in Human gene therapy (01.10.2013)
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Purification of recombinant adeno-associated virus type 8 vectors by ion exchange chromatography generates clinical grade vector stock
Davidoff, Andrew M., Ng, Catherine Y.C., Sleep, Susan, Gray, John, Azam, Selina, Zhao, Yuan, McIntosh, Jenny H., Karimipoor, Morteza, Nathwani, Amit C.
Published in Journal of virological methods (01.11.2004)
Published in Journal of virological methods (01.11.2004)
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Prevention of Photoreceptor Cell Loss in a Cln6 Mouse Model of Batten Disease Requires CLN6 Gene Transfer to Bipolar Cells
kleine Holthaus, Sophia-Martha, Ribeiro, Joana, Abelleira-Hervas, Laura, Pearson, Rachael A., Duran, Yanai, Georgiadis, Anastasios, Sampson, Robert D., Rizzi, Matteo, Hoke, Justin, Maswood, Ryea, Azam, Selina, Luhmann, Ulrich F.O., Smith, Alexander J., Mole, Sara E., Ali, Robin R.
Published in Molecular therapy (02.05.2018)
Published in Molecular therapy (02.05.2018)
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Prevention of Photoreceptor Cell Loss in a Cln6 nclf Mouse Model of Batten Disease Requires CLN6 Gene Transfer to Bipolar Cells
Kleine Holthaus, Sophia-Martha, Ribeiro, Joana, Abelleira-Hervas, Laura, Pearson, Rachael A, Duran, Yanai, Georgiadis, Anastasios, Sampson, Robert D, Rizzi, Matteo, Hoke, Justin, Maswood, Ryea, Azam, Selina, Luhmann, Ulrich F O, Smith, Alexander J, Mole, Sara E, Ali, Robin R
Published in Molecular therapy (02.05.2018)
Published in Molecular therapy (02.05.2018)
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Prevention of Photoreceptor Cell Loss in a Cln6nclf Mouse Model of Batten Disease Requires CLN6 Gene Transfer to Bipolar Cells
kleine Holthaus, Sophia-Martha, Ribeiro, Joana, Abelleira-Hervas, Laura, Pearson, Rachael A., Duran, Yanai, Georgiadis, Anastasios, Sampson, Robert D., Rizzi, Matteo, Hoke, Justin, Maswood, Ryea, Azam, Selina, Luhmann, Ulrich F.O., Smith, Alexander J., Mole, Sara E., Ali, Robin R.
Published in Molecular therapy (02.05.2018)
Published in Molecular therapy (02.05.2018)
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461. Evaluation of In Vivo Model Systems for Safety Assessment of HIV-1 Based Lentiviral Vectors
Zhao, Yuan, Azam, Selina, Keating, Kenneth, Thorpe, Robin
Published in Molecular therapy (01.05.2006)
Published in Molecular therapy (01.05.2006)
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