Targeted Delivery and Tolerability of MRI-Guided CED Infusion into the Cerebellum of Nonhuman Primates.
| Autor: | Salegio EA; 1 Valley Biosystems, Inc. , West Sacramento, CA., Campagna MV; 1 Valley Biosystems, Inc. , West Sacramento, CA., Allen PC; 1 Valley Biosystems, Inc. , West Sacramento, CA., Stockinger DE; 1 Valley Biosystems, Inc. , West Sacramento, CA., Song Y; 2 Raymond G. Perelman Center for Cellular and Molecular Therapeutics , The Children's Hospital of Philadelphia, Philadelphia, PA.; 3 Department of Pathology and Laboratory Medicine, University of Pennsylvania , Philadelphia, PA., Hwa GGC; 1 Valley Biosystems, Inc. , West Sacramento, CA. |
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| Jazyk: | angličtina |
| Zdroj: | Human gene therapy methods [Hum Gene Ther Methods] 2018 Aug; Vol. 29 (4), pp. 169-176. Date of Electronic Publication: 2018 Aug 13. |
| DOI: | 10.1089/hgtb.2018.049 |
| Abstrakt: | This study explored the feasibility of intraparenchymal delivery (gadoteridol and/or Serotype 5 Adeno-Associated Viral Vector-enhanced Green Fluorescent Protein [AAV5-eGFP]) into the cerebellum of nonhuman primates using real-time magnetic resonance imaging-guided convection enhanced delivery (MRI-CED) technology. All animals tolerated the neurosurgical procedure without any clinical sequela. Gene expression was detected within the cerebellar parenchyma at the site of infusion and resulted in transduction of neuronal cell bodies and fibers. Histopathology indicated localized damage along the stem of the cannula tract. These findings demonstrate the potential of real-time MRI-CED to deliver therapeutics into the cerebellum, which has extensive reciprocal connections and may be used as a target for the treatment of neurological disorders. |
| Databáze: | MEDLINE |
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