Clinical and Molecular Features of Early Infantile Niemann Pick Type C Disease

por Seker Yilmaz, Berna, Baruteau, Julien, Rahim, Ahad A., Gissen, Paul
Publicado 2020

Using Stem Cells to Grow Artificial Tissue for Peripheral Nerve Repair

por Bhangra, Kulraj Singh, Busuttil, Francesca, Phillips, James B., Rahim, Ahad A.
Publicado 2016

Neuroprotective Effects of Diabetes Drugs for the Treatment of Neonatal Hypoxia-Ischemia Encephalopathy

por Poupon-Bejuit, Laura, Rocha-Ferreira, Eridan, Thornton, Claire, Hagberg, Henrik, Rahim, Ahad A.
Publicado 2020

Immediate Remote Ischemic Postconditioning Reduces Brain Nitrotyrosine Formation in a Piglet Asphyxia Model

por Rocha-Ferreira, Eridan, Rudge, Brogan, Hughes, Michael P., Rahim, Ahad A., Hristova, Mariya, Robertson, Nicola J.
Publicado 2016

Inhibition of Mitochondrial Complex I Impairs Release of α-Galactosidase by Jurkat Cells

por Lambert, Jonathan R. A., Howe, Steven J., Rahim, Ahad A., Burke, Derek G., Heales, Simon J. R.
Publicado 2019

Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development

por Massaro, Giulia, Geard, Amy F., Liu, Wenfei, Coombe-Tennant, Oliver, Waddington, Simon N., Baruteau, Julien, Gissen, Paul, Rahim, Ahad A.
Publicado 2021

Neuroprotective exendin-4 enhances hypothermia therapy in a model of hypoxic-ischaemic encephalopathy

por Rocha-Ferreira, Eridan, Poupon, Laura, Zelco, Aura, Leverin, Anna-Lena, Nair, Syam, Jonsdotter, Andrea, Carlsson, Ylva, Thornton, Claire, Hagberg, Henrik, Rahim, Ahad A
Publicado 2018

Systemic AAV9 gene therapy using the synapsin I promoter rescues a mouse model of neuronopathic Gaucher disease but with limited cross-correction potential to astrocytes

por Massaro, Giulia, Hughes, Michael P, Whaler, Sammie M, Wallom, Kerri-Lee, Priestman, David A, Platt, Frances M, Waddington, Simon N, Rahim, Ahad A
Publicado 2020

Perinatal Gene Transfer to the Liver

por McKay, Tristan R, Rahim, Ahad A, Buckley, Suzanne M.K, Ward, Natalie J, Chan, Jerry K.Y, Howe, Steven J, Waddington, Simon N
Publicado 2011

Evidence for Contribution of CD4+CD25+ Regulatory T Cells in Maintaining Immune Tolerance to Human Factor IX following Perinatal Adenovirus Vector Delivery

por Nivsarkar, Megha S., Buckley, Suzanne M. K., Parker, Alan L., Perocheau, Dany, McKay, Tristan R., Rahim, Ahad A., Howe, Steven J., Waddington, Simon N.
Publicado 2015

Impaired cellular bioenergetics caused by GBA1 depletion sensitizes neurons to calcium overload

por Plotegher, Nicoletta, Perocheau, Dany, Ferrazza, Ruggero, Massaro, Giulia, Bhosale, Gauri, Zambon, Federico, Rahim, Ahad A., Guella, Graziano, Waddington, Simon N., Szabadkai, Gyorgy, Duchen, Michael R.
Publicado 2019

Correction: Impaired cellular bioenergetics caused by GBA1 depletion sensitizes neurons to calcium overload

por Plotegher, Nicoletta, Perocheau, Dany, Ferrazza, Ruggero, Massaro, Giulia, Bhosale, Gauri, Zambon, Federico, Rahim, Ahad A., Guella, Graziano, Waddington, Simon N., Szabadkai, Gyorgy, Duchen, Michael R.
Publicado 2020

Mitochondria and Quality Control Defects in a Mouse Model of Gaucher Disease—Links to Parkinson’s Disease

por Osellame, Laura D., Rahim, Ahad A., Hargreaves, Iain P., Gegg, Matthew E., Richard-Londt, Angela, Brandner, Sebastian, Waddington, Simon N., Schapira, Anthony H.V., Duchen, Michael R.
Publicado 2013

AAV9 intracerebroventricular gene therapy improves lifespan, locomotor function and pathology in a mouse model of Niemann–Pick type C1 disease

por Hughes, Michael P, Smith, Dave A, Morris, Lauren, Fletcher, Claire, Colaco, Alexandria, Huebecker, Mylene, Tordo, Julie, Palomar, Nuria, Massaro, Giulia, Henckaerts, Els, Waddington, Simon N, Platt, Frances M, Rahim, Ahad A
Publicado 2018

Systemic gene delivery following intravenous administration of AAV9 to fetal and neonatal mice and late-gestation nonhuman primates

por Mattar, Citra N., Wong, Andrew M. S., Hoefer, Klemens, Alonso-Ferrero, Maria E., Buckley, Suzanne M. K., Howe, Steven J., Cooper, Jonathan D., Waddington, Simon N., Chan, Jerry K. Y., Rahim, Ahad A.
Publicado 2015

Epilepsy Gene Therapy Using an Engineered Potassium Channel

por Snowball, Albert, Chabrol, Elodie, Wykes, Robert C., Shekh-Ahmad, Tawfeeq, Cornford, Jonathan H., Lieb, Andreas, Hughes, Michael P., Massaro, Giulia, Rahim, Ahad A., Hashemi, Kevan S., Kullmann, Dimitri M., Walker, Matthew C., Schorge, Stephanie
Publicado 2019

Perinatal systemic gene delivery using adeno-associated viral vectors

por Karda, Rajvinder, Buckley, Suzanne M. K., Mattar, Citra N., Ng, Joanne, Massaro, Giulia, Hughes, Michael P., Kurian, Manju A., Baruteau, Julien, Gissen, Paul, Chan, Jerry K. Y., Bacchelli, Chiara, Waddington, Simon N., Rahim, Ahad A.
Publicado 2014

In vivo bioimaging with tissue-specific transcription factor activated luciferase reporters

por Buckley, Suzanne M. K., Delhove, Juliette M. K. M., Perocheau, Dany P., Karda, Rajvinder, Rahim, Ahad A., Howe, Steven J., Ward, Natalie J., Birrell, Mark A., Belvisi, Maria G., Arbuthnot, Patrick, Johnson, Mark R., Waddington, Simon N., McKay, Tristan R.
Publicado 2015

Foamy Virus Vectors Transduce Visceral Organs and Hippocampal Structures following In Vivo Delivery to Neonatal Mice

por Counsell, John R., Karda, Rajvinder, Diaz, Juan Antinao, Carey, Louise, Wiktorowicz, Tatiana, Buckley, Suzanne M.K., Ameri, Shima, Ng, Joanne, Baruteau, Julien, Almeida, Filipa, de Silva, Rohan, Simone, Roberto, Lugarà, Eleonora, Lignani, Gabriele, Lindemann, Dirk, Rethwilm, Axel, Rahim, Ahad A., Waddington, Simon N., Howe, Steven J.
Publicado 2018

Generation of light-producing somatic-transgenic mice using adeno-associated virus vectors

por Karda, Rajvinder, Rahim, Ahad A., Wong, Andrew M. S., Suff, Natalie, Diaz, Juan Antinao, Perocheau, Dany P., Tijani, Maha, Ng, Joanne, Baruteau, Julien, Martin, Nuria Palomar, Hughes, Michael, Delhove, Juliette M. K. M., Counsell, John R., Cooper, Jonathan D., Henckaerts, Els, Mckay, Tristan R., Buckley, Suzanne M. K., Waddington, Simon N.
Publicado 2020