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Putaminal Recombinant Glucocerebrosidase Delivery with Magnetic Resonance-Guided Focused Ultrasound in Parkinson's Disease: A Phase I Study.

Movement disorders : official journal of the Movement Disorder Society - 1 Oct 2022

Meng Ying, Pople Christopher B, Huang Yuexi, Jones Ryan M, Ottoy Julie, Goubran Maged, Oliveira Lais M, Davidson Benjamin, Lawrence Liam S P, Lau Angus Z, Bethune Allison, Maralani Pejman, Abrahao Agessandro, Hamani Clement, Hynynen Kullervo, Kalia Suneil K, Lipsman Nir, Kalia Lorraine V

Abstract excerpt

BACKGROUND: GBA1 mutation is the most common genetic risk factor for Parkinson's disease (PD). Replacement of the lysosomal enzyme glucocerebrosidase (GCase) slows neurodegeneration in PD models and may be a promising disease-modifying therapy in patients with PD. However, recombinant GCase has limited penetration through the blood-brain barrier (BBB). Microbubble-mediated magnetic resonance-guided focused...

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PMID: 36089809
DOI: 10.1002/mds.29190

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