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Development (Cambridge, England)|March 24, 2006
Early, H+-V-ATPase-dependent proton flux is necessary for consistent left-right patterning of non-mammalian vertebratesDany S Adams, Kenneth R Robinson, Takahiro Fukumoto, et al.
Biomedicines|March 28, 2025
Reversing Pathology in an Aggravated Fabry Mouse Model Using Low-Dose Engineered Human Alpha-Galactosidase A AAV Gene TherapyWanida Ruangsiriluk, Mugdha Deshpande, Natalia Boukharov, et al.
Human Gene Therapy|July 6, 2024
Developing Gene Therapy for Mitigating Multisystemic Pathology in Fabry Disease: Proof of Concept in an Aggravated Mouse ModelNatalia Boukharov, Shipeng Yuan, Wanida Ruangsirluk, et al.
Plos One|October 24, 2025
Volumetric MRI of dorsal root ganglia as a biomarker for disease progression and response to AAV treatment in a mouse model of Fabry diseaseFuqiang Zhao, Shipeng Yuan, Charalambos Kaittanis, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|November 20, 2024
Longitudinal imaging of therapeutic enzyme expression after gene therapy for Fabry disease using positron emission tomography and the radiotracer [18F]AGALCharalambos Kaittanis, Tyler Teceno, Ashley Knight, et al.
Human Gene Therapy|December 29, 2025
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 MiceWanida Ruangsiriluk, Sarah Melissa P Jacobo, Andrea Iskenderian, et al.
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