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Gene therapy

Comparison of neonatal systemic and intracerebroventricular AAV9 gene therapy delivery demonstrating improved behavioral and phenotypic outcomes in a mouse model of Niemann-Pick disease, type C1

Benjamin E. Epstein, Gabrielle M. Soden, [ ... ], William J. Pavan

Assessing the biopotency of the rAAV9 vector In Vitro

Pratikshya Adhikari, Peter G. Nichols, [ ... ], Haiyan Fu

Adeno-associated vector corneal gene therapy reverses corneal clouding in a feline model of mucopolysaccharidosis VI

Brian C. Gilger, Liujiang Song, [ ... ], Matthew L. Hirsch

Synergistic enhancement of AAV gene delivery in 2D cells and 3D organoids using polybrene and hydroxychloroquine

Hyeon-Jin Na, Yongbo Shin, [ ... ], Kyung-Sook Chung

Toward an all-in-one recombinant adeno-associated virus vector for functionally ablating the prion gene using CRISPR-Cas technology

Claire Verkuyl, Ari Belotserkovsky, [ ... ], Gerold Schmitt-Ulms

Enrollment patterns among medicaid beneficiaries with sickle cell disease: Multistate findings from the sickle cell data collection program

Ashima Singh, Mahua Dasgupta, [ ... ], Angela B. Snyder

Volumetric MRI of dorsal root ganglia as a biomarker for disease progression and response to AAV treatment in a mouse model of Fabry disease

Fuqiang Zhao, Shipeng Yuan, [ ... ], Talakad G. Lohith

HMGB1-BoxA gene therapy in reversing cisplatin resistance in non-small cell lung cancer

Nattapong Puthdee, Van-Hieu Mai, [ ... ], Chanida Vinayanuwattikun

Gene editing therapy as a therapeutic approach for cardiovascular diseases in animal models: A scoping review

Quan Duy Vo

Intracerebroventricular administration of a modified hexosaminidase ameliorates late-stage neurodegeneration in a GM2 mouse model

Manuel E. Lopez, Daniel Wendt, [ ... ], Mika Aoyagi-Scharber

Tagitinin A regulates an F-box gene, CPR30, to resist tomato spotted wilt orthotospovirus (TSWV) infection in Nicotiana benthamiana

Jing Li, Xiaoman Ai, [ ... ], Lihua Zhao

Banana fruit (Musa sp.) DNA-magnetite nanoparticles: Synthesis, characterization, and biocompatibility assays on normal and cancerous cells

David Arregui-Almeida, Martín Coronel, [ ... ], Fernanda Pilaquinga

Dynamic BMP gene expression regulation in chick RPE during recovery from short term optical defocus and form-deprivation

Yan Zhang, Qiurong Zhu, [ ... ], Christine F. Wildsoet

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