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The Lancet. Neurology|December 15, 2023
Safety and efficacy of cerliponase alfa in children with neuronal ceroid lipofuscinosis type 2 (CLN2 disease): an open-label extension studyAngela Schulz, Nicola Specchio, Emily de Los Reyes, et al.
Human Gene Therapy|August 25, 2023
Positron Emission Tomography Quantitative Assessment of Off-Target Whole-Body Biodistribution of I-124-Labeled Adeno-Associated Virus Capsids Administered to Cerebral Spinal FluidJonathan B Rosenberg, Edward K Fung, Jonathan P Dyke, et al.
Human Gene Therapy. Clinical Development|February 8, 2018
AAVrh.10-Mediated APOE2 Central Nervous System Gene Therapy for APOE4-Associated Alzheimer's DiseaseJonathan B Rosenberg, Michael G Kaplitt, Bishnu P De, et al.
Human Gene Therapy|September 10, 2005
Confronting the issues of therapeutic misconception, enrollment decisions, and personal motives in genetic medicine-based clinical research studies for fatal disordersLisa M Arkin, Dolan Sondhi, Stefan Worgall, et al.
Scientific Reports|June 20, 2024
In vivo brain estrogen receptor density by neuroendocrine aging and relationships with cognition and symptomatologyLisa Mosconi, Matilde Nerattini, Dawn C Matthews, et al.
Biorxiv : the Preprint Server for Biology|July 28, 2023
Bidirectional Regulation of Motor Circuits Using Magnetogenetic Gene Therapy Short: Magnetogenetic Regulation of Motor CircuitsSantiago R Unda, Lisa E Pomeranz, Roberta Marongiu, et al.
Science Advances|October 9, 2024
Bidirectional regulation of motor circuits using magnetogenetic gene therapySantiago R Unda, Lisa E Pomeranz, Roberta Marongiu, et al.
Frontiers in Neuroimaging|March 6, 2023
Tau PET following acute TBI: Off-target binding to blood products, tauopathy, or both?Tracy Butler, Gloria C Chiang, Sumit Narayan Niogi, et al.
Scientific Reports|April 14, 2025
Divergent neurodegeneration associations with choroid plexus volume and degree of calcification in cognitively normal APOE ε4 carriers and non-carriersIlker Ozsahin, Xiuyuan Wang, Liangdong Zhou, et al.
Science Translational Medicine|December 3, 2020
Slowing late infantile Batten disease by direct brain parenchymal administration of a rh.10 adeno-associated virus expressing <i>CLN2</i>Dolan Sondhi, Stephen M Kaminsky, Neil R Hackett, et al.
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