Cheng, Seng H.

Sustained Therapeutic Reversal of Huntington's Disease by Transient Repression of Huntingtin Synthesis

Kordasiewicz, H. B., Stanek, L. M., Wancewicz, E. V., Mazur, C., McAlonis, M. M., Pytel, K. A., Artates, J. W., Weiss, A., Cheng, S. H., Shihabuddin, L. S., et al. (2012) · Neuron

Genetic Neurodegenerative DiseasesRNA Interference and Gene Delivery

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Hypoxia-inducible factor-1 mediates activation of cultured vascular endothelial cells by inducing multiple angiogenic factors

Yamakawa, M., Liu, L. X., Date, T., Belanger, A. J., Vincent, K. A., Akita, G. Y., Kuriyama, T., Cheng, S. H., Gregory, R. J., & Jiang, C. (2003) · Circulation Research

Angiogenesis and VEGF in CancerCancer, Hypoxia, and Metabolism

Cited by 327

CRISPR/Cas9-Mediated Genome Editing as a Therapeutic Approach for Leber Congenital Amaurosis 10

Ruan, G.-X., Barry, E., Yu, D., Lukason, M., Cheng, S. H., & Scaria, A. (2017) · Molecular Therapy

CRISPR and Genetic EngineeringRetinal Development and Disorders

Cited by 270

Intravitreous injection of AAV2-sFLT01 in patients with advanced neovascular age-related macular degeneration: a phase 1, open-label trial

Heier, J. S., Kherani, S., Desai, S., Dugel, P., Kaushal, S., Cheng, S. H., Delacono, C., Purvis, A., Richards, S., Le-Halpere, A., et al. (2017) · The Lancet

Retinal Diseases and TreatmentsRetinal Development and Disorders

Cited by 229

Glucosylceramide synthase inhibition alleviates aberrations in synucleinopathy models

Sardi, S. P., Viel, C., Clarke, J., Treleaven, C. M., Richards, A. M., Park, H., Olszewski, M. A., Dodge, J. C., Marshall, J., Makino, E., et al. (2017) · Proceedings of the National Academy of Sciences

Lysosomal Storage Disorders ResearchParkinson's Disease Mechanisms and Treatments

Cited by 203

Analytical Ultracentrifugation as an Approach to Characterize Recombinant Adeno-Associated Viral Vectors

Burnham, B., Nass, S., Kong, E., Mattingly, M., Woodcock, D., Song, A., Wadsworth, S., Cheng, S. H., Scaria, A., & O'Riordan, C. R. (2015) · Human Gene Therapy Methods

Virus-based gene therapy researchProtein purification and stability

Cited by 150

Universal Method for the Purification of Recombinant AAV Vectors of Differing Serotypes

Nass, S. A., Mattingly, M. A., Woodcock, D. A., Burnham, B. L., Ardinger, J. A., Osmond, S. E., Frederick, A. M., Scaria, A., Cheng, S. H., & O’Riordan, C. R. (2018) · Molecular Therapy - Methods & Clinical Development

Virus-based gene therapy researchProtein purification and stability

Cited by 146

Optimization of Plasmid Vectors for High-Level Expression in Lung Epithelial Cells

Yew, N. S., Wysokenski, D. M., Wang, K. X., Ziegler, R. J., Marshall, J., McNeilly, D., Cherry, M., Osburn, W., & Cheng, S. H. (1997) · Human Gene Therapy

RNA Interference and Gene DeliveryVirus-based gene therapy research

Cited by 113

Contribution of Toll-like Receptor 9 Signaling to the Acute Inflammatory Response to Nonviral Vectors

Zhao, H., Hemmi, H., Akira, S., Cheng, S. H., Scheule, R. K., & Yew, N. S. (2004) · Molecular Therapy

RNA Interference and Gene DeliveryImmune Response and Inflammation

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Rationally designed AAV2 and AAVrh8R capsids provide improved transduction in the retina and brain

Sullivan, J. A., Stanek, L. M., Lukason, M. J., Bu, J., Osmond, S. R., Barry, E. A., O’Riordan, C. R., Shihabuddin, L. S., Cheng, S. H., & Scaria, A. (2018) · Gene Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

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Gene transfer to the CNS is efficacious in immune-primed mice harboring physiologically relevant titers of anti-AAV antibodies

Treleaven, C. M., Tamsett, T. J., Bu, J., Fidler, J. A., Sardi, S. P., Hurlbut, G. D., Woodworth, L. A., Cheng, S. H., Passini, M. A., Shihabuddin, L. S., et al. (2012) · Molecular Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

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