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Molecular Therapy : the Journal of the American Society of Gene Therapy|June 1, 2005
Genetic analysis of the antibody response to AAV2 and factor IXHuang-Ge Zhang, Katherine A High, Qi Wu, et al.Plos One|July 12, 2013
IL12-mediated liver inflammation reduces the formation of AAV transcriptionally active forms but has no effect over preexisting AAV transgene expressionIrene Gil-Fariña, Marianna Di Scala, Lucia Vanrell, et al.EMBO Molecular Medicine|October 10, 2013
Effective gene therapy for haemophilic mice with pathogenic factor IX antibodiesDavid M Markusic, Brad E Hoffman, George Q Perrin, et al.Journal of Virology|July 16, 2004
Disparate regions of envelope protein regulate syncytium formation versus spongiform encephalopathy in neurological disease induced by murine leukemia virus TRSamuel L Murphy, Marek J Honczarenko, Natalie V Dugger, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|July 16, 2009
Impact of the underlying mutation and the route of vector administration on immune responses to factor IX in gene therapy for hemophilia BOu Cao, Brad E Hoffman, Babak Moghimi, et al.The Journal of Clinical Investigation|May 3, 2003
Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transferFederico Mingozzi, Yi-Lin Liu, Eric Dobrzynski, et al.Blood|October 14, 2004
Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal modelValder R Arruda, Hansell H Stedman, Timothy C Nichols, et al.Blood|August 25, 2012
The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapyJonathan D Finn, Timothy C Nichols, Nikolaos Svoronos, et al.Nature Neuroscience|November 18, 2025
Roadmap for direct and indirect translation of optogenetics into discoveries and therapies for humansChristian Lüscher, Valentina Emiliani, Nita Farahany, et al.Clinical & Experimental Ophthalmology|July 12, 2017
Novel mobility test to assess functional vision in patients with inherited retinal dystrophiesDaniel C Chung, Sarah McCague, Zi-Fan Yu, et al.Pageof 15