Journal Papers 28 Related Insights 0
Intravenous Delivery of a Gene Therapy Vector that Expresses an Antitransferrin Receptor 1 Nanobody-I2S Fusion Protein Rescued Central Nervous System Lysosomal Burden in Mucopolysaccharidosis II Mice Ruangsiriluk, Wanida; Jacobo, Sarah Melissa P.; Iskenderian, Andrea; Modi, Meera; Chen, Nancy; Felice, Brian; Yuan, Shipeng; Mukherji, Shreya T.; Hernandez, Melody Rivera; Sunkara, Shashank; Macdonald, Amanda; Rajarshi, Girija; Palmieri, Kathleen; Crooker, Robert; Wu, Rui; Dalrymple, Margaret; Chheda, Mihir; Megahid, Donia; Arce, Camila; Shen, Mingfang; Dasari, Bhanu; Zhao, Xiaohui; Yalavarthi, Maneesha; Rompikuntal, Pramod; Ayyadurai, Saravanan; Fang, Josephine; Hozanovic, Alen; Pescatore, Brian; Bassous, Dany; Romashko, Alla; Galbreath, Elizabeth; Maccani, Andreas; Steinkellner, Julia; Ehmann, David E.; Choi, Vivian W.; Islam, Rizwana Share Save
Brain-Directed AAV Gene Therapy Rescues a Mouse Model of the CLN5 Form of Neuronal Ceroid Lipofuscinosis Disease and Normalizes a Blood Plasma Biomarker of Neurodegeneration Liu, Wenfei; Geard, Amy F.; Massaro, Giulia; Hughes, Michael P.; Aristorena, Mikel; Coombe-Tennant, Oliver; Xu, Liangyuan; Semenyuk, Olha; Bush, Reuben; Te Vruchte, Danielle; Priestman, David; Laban, Rhiannon; Veleva, Elena; Heslegrave, Amanda J.; Zetterberg, Henrik; Platt, Frances M.; Smith, Alexander J.; Mole, Sara E.; Ali, Robin R.; Rahim, Ahad A. Share Save
Efficient LAMA1 Gene Activation by Epigenome Editing as a Therapeutic Approach for LAMA2-CMD Qin, Yuanbo; Akbulut, Talha; Mandraju, Rajakumar; Connolly, Keith; Bechill, John; Assadian, Farzaneh; Foster, Claudia; Shottek, Alison; Levy, Seth; Benoit, Jamie; Yamagata, Tetsuya Share Save
In Remembrance of Professor Zelig Eshhar: A Life Committed to CAR-T Debets, Reno; Hawkins, Robert; Gilham, David; Gorochov, Guy; Schumacher, Ton; Uckert, Wolfgang; Blankenstein, Thomas; Crawford, Dorothy; Taylor, Naomi; Szollosi, Janos; Stauss, Hans; Mondino, Anna; Allavena, Paola; Alarcon, Balbino; Buning, Hildegard; Abken, Hinrich Share Save
Multivariate Process Optimization for Fixed-Bed Bioreactor-Based AAV Production Improves Total Batch Yield Selot, Ruchita; Khaparde, Ashish; Babu, G. R. Sharath; Gopinath, Chitra; Panigrahi, Trailokyanath; Sarkar, Subhradeep; Dhinakar, Joy Elvin; Patra, Riya; Panikker, Priyalakshmi; Ghosh, Arkasubhra Share Save
Stable and Predictable Lentiviral Vector Production at Clinical Scale Heinz, Niels; Mosbacher, Laura; Cordie, Lisa C.; Zeller, Katharina; Rohn, Florian; Naeher, Melanie; Hien, Melina; Ohlinger, Stefan; Kuehlcke, Klaus; Loew, Rainer Share Save
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