Robot-assisted intrathalamic infusion for gene therapy in young children: surgical considerations
In this study, the surgical technique used in the first in-human intrathalamic drug infusion gene therapy trial in young children with GM2 gangliosidosis was reviewed. The innovative stereotactic setup used robotic surgical assistance and ensured high-precision targeting and secure cannula placement during prolonged infusions, even in infants as young as 6 months of age. Post-infusion MRI confirmed that the infusate remained well contained within the thalamus. The mean Vd/Vi ratio of 2.0 is consistent with the literature. Overall, bilateral thalamic delivery of AAV transgene in children was safe and well tolerated.
Authors
- Terence R. Flotte (ORCID: https://orcid.org/0000-0002-8255-0588)
- Mohammed Salman Shazeeb (ORCID: https://orcid.org/0000-0002-3175-8098)
- Richard P. Moser (ORCID: https://orcid.org/0000-0001-6884-0653)
- Oğuz Çataltepe (ORCID: https://orcid.org/0000-0003-4414-5386)
- Anna Luisa Kühn (ORCID: https://orcid.org/0000-0001-9512-9461)
- Brittany Owusu‐Adjei (ORCID: https://orcid.org/0009-0009-3695-3173)
- Spiro G. Spanakis
- Julia Parzych
- Rrita Daci (ORCID: https://orcid.org/0000-0003-3767-2284)
- R King (ORCID: https://orcid.org/0000-0002-5144-9110)
Institutions
- University of Massachusetts Chan Medical School (US)
- UMass Memorial Medical Center (US)
Publication Details
- Journal
- Journal of Neurosurgery Pediatrics
- Published
- 2025-08-16
- DOI
- https://doi.org/10.3171/2025.4.peds2569
- Citations
- 2
- Primary Topic
- Fetal and Pediatric Neurological Disorders
- Type
- article
- Field-Weighted Citation Impact
- 6.99