Gounis, Matthew J.

A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system

Alterman, J. F., Godinho, B. M. D. C., Hassler, M. R., Ferguson, C. M., Echeverria, D., Sapp, E., Haraszti, R. A., Coles, A. H., Conroy, F., Miller, R., et al. (2019) · Nature Biotechnology

RNA Interference and Gene DeliveryDNA and Nucleic Acid Chemistry

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AAV gene therapy for Tay-Sachs disease

Flotte, T. R., Cataltepe, O., Puri, A., Batista, A. R., Moser, R., McKenna-Yasek, D., Douthwright, C., Gernoux, G., Blackwood, M., Mueller, C., et al. (2022) · Nature Medicine

Lysosomal Storage Disorders ResearchVirus-based gene therapy research

Cited by 143

Direct Intracranial Injection of AAVrh8 Encoding Monkey β-N-Acetylhexosaminidase Causes Neurotoxicity in the Primate Brain

Golebiowski, D., van der Bom, I. M. J., Kwon, C.-S., Miller, A. D., Petrosky, K., Bradbury, A. M., Maitland, S., Kühn, A. L., Bishop, N., Curran, E., et al. (2017) · Human Gene Therapy

Lysosomal Storage Disorders ResearchVirus-based gene therapy research

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Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep

Ferguson, C. M., Godinho, B. M. D. C., Alterman, J. F., Coles, A. H., Hassler, M., Echeverria, D., Gilbert, J. W., Knox, E. G., Caiazzi, J., Haraszti, R. A., et al. (2021) · JCI Insight

RNA Interference and Gene DeliveryBarrier Structure and Function Studies

Cited by 27

Registration of 2D x-ray images to 3D MRI by generating pseudo-CT data

van der Bom, M. J., Pluim, J. P. W., Gounis, M. J., van de Kraats, E. B., Sprinkhuizen, S. M., Timmer, J., Homan, R., & Bartels, L. W. (2011) · Physics in Medicine and Biology

Medical Image Segmentation TechniquesAdvanced Radiotherapy Techniques

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AAV Gene Therapy Strategies for Lysosomal Storage Disorders with Central Nervous System Involvement

Golebiowski, D., Bradbury, A. M., Kwon, C.-S., van der Bom, I. M. J., Stoica, L., Johnson, A. K., Wilson, D. U., Gray-Edwards, H. L., Hudson, J. A., Johnson, J. A., et al. (2015) · Neuromethods

Virus-based gene therapy researchLysosomal Storage Disorders Research

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