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12010.
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Long-term expression and safety of administration of AAVrh.10hCLN2 to the brain of rats and nonhuman primates for the treatment of late infantile neuronal ceroid lipofuscinosis. [electronic resource] by
- Sondhi, Dolan
- Johnson, Linda
- Purpura, Keith
- Monette, Sebastien
- Souweidane, Mark M
- Kaplitt, Michael G
- Kosofsky, Barry
- Yohay, Kaleb
- Ballon, Douglas
- Dyke, Jonathan
- Kaminksy, Stephen M
- Hackett, Neil R
- Crystal, Ronald G
Producer: 20130718
In:
Human gene therapy methods vol. 23
Availability: No items available.
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12011.
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A Primate-Specific Isoform of PLEKHG6 Regulates Neurogenesis and Neuronal Migration. [electronic resource] by
- O'Neill, Adam C
- Kyrousi, Christina
- Klaus, Johannes
- Leventer, Richard J
- Kirk, Edwin P
- Fry, Andrew
- Pilz, Daniela T
- Morgan, Tim
- Jenkins, Zandra A
- Drukker, Micha
- Berkovic, Samuel F
- Scheffer, Ingrid E
- Guerrini, Renzo
- Markie, David M
- Götz, Magdalena
- Cappello, Silvia
- Robertson, Stephen P
Producer: 20191113
In:
Cell reports vol. 25
Availability: No items available.
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12012.
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Translational studies of intravenous and intracerebroventricular routes of administration for CNS cellular biodistribution for BMN 250, an enzyme replacement therapy for the treatment of Sanfilippo type B. [electronic resource] by
- Grover, Anita
- Crippen-Harmon, Danielle
- Nave, Lacey
- Vincelette, Jon
- Wait, Jill C M
- Melton, Andrew C
- Lawrence, Roger
- Brown, Jillian R
- Webster, Katherine A
- Yip, Bryan K
- Baridon, Brian
- Vitelli, Catherine
- Rigney, Sara
- Christianson, Terri M
- Tiger, Pascale M N
- Lo, Melanie J
- Holtzinger, John
- Shaywitz, Adam J
- Crawford, Brett E
- Fitzpatrick, Paul A
- LeBowitz, Jonathan H
- Bullens, Sherry
- Aoyagi-Scharber, Mika
- Bunting, Stuart
- O'Neill, Charles A
- Pinkstaff, Jason
- Bagri, Anil
Producer: 20201119
In:
Drug delivery and translational research vol. 10
Availability: No items available.
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