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ProSavin

From Wikipedia, the free encyclopedia

ProSavin is an experimental drug believed to be of use in the treatment of Parkinson's disease. It is administered to the striatum in the brain, inducing production of dopamine.[1]

It is manufactured by Oxford BioMedica. Results from a Phase I/II clinical trial were published in the Lancet[2] and showed safety, but little efficacy.[3] ProSavin was superseded by AXO-Lenti-PD (OXB-102), an optimized version of the drug.[4]

Mechanism of action

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Prosavin uses Oxford BioMedica's Lentivector delivery system to transfer three genes, aromatic amino acid dopa decarboxylase, tyrosine hydroxylase and GTP-cyclohydrolase 1, to the striatum in the brain, reprogramming transduced cells to secrete dopamine.[5][6]

See also

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References

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  1. ^ Oxford BioMedica. Drug Information Page. Retrieved on March 29, 2007.
  2. ^ Palfi, Stéphane; Gurruchaga, Jean Marc; Ralph, G. Scott; Lepetit, Helene; Lavisse, Sonia; Buttery, Philip C.; Watts, Colin; Miskin, James; Kelleher, Michelle; Deeley, Sarah; Iwamuro, Hirokazu (2014-03-29). "Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial". The Lancet. 383 (9923): 1138–1146. doi:10.1016/S0140-6736(13)61939-X. ISSN 0140-6736. PMID 24412048. S2CID 4993549.
  3. ^ Communications, Maggie Kuhl Director Research & CEO (28 January 2014). "ProSavin Trial Results: Once Again, a Gene Therapy Approach to Parkinson's Yields Encouraging Safety Data but Modest Efficacy | Parkinson's Disease". www.michaeljfox.org. Retrieved 2021-04-09.
  4. ^ Wexler, Marisa (20 August 2019). "Parkinson's Gene Therapy in Clinical Trial, AXO-Lenti-PD, Safe And Effective in Monkey Model of Disease, Study Says". Retrieved 2021-04-09.
  5. ^ "Positive results in Phase I/II trial". OxfordBiomedica. 19 November 2008.
  6. ^ "Prosavin". OxfordBiomedica. Archived from the original on 16 March 2014.