Publications by authors named "Oguz I Cataltepe"
Article Synopsis
- - Neurological diseases caused by single gene defects can be treated through AAV-mediated gene therapy, but delivering this therapy to the brain is difficult because of the blood-brain barrier.
- - Advanced techniques, like convection-enhanced delivery and image-guided methods to cerebrospinal fluid spaces, allow for precise gene therapy delivery to target specific brain areas.
- - Neuroimaging methods, including MRI and fMRI, are crucial for both delivering AAV vectors and monitoring the effectiveness of the gene therapy over time.
Thalamic infusion of adeno-associated viral (AAV) vectors has been shown to have therapeutic effects in neuronopathic lysosomal storage diseases. Preclinical studies in sheep model of Tay-Sachs disease demonstrated that bilateral thalamic injections of AAV gene therapy are required for maximal benefit. Translation of thalamic injection to patients carries risks in that (1) it has never been done in humans, and (2) dosing scale-up based on brain weight from animals to humans requires injection of larger volumes.
View Article and Find Full Text PDFGlobal gene delivery to the CNS has therapeutic importance for the treatment of neurological disorders that affect the entire CNS. Due to direct contact with the CNS, cerebrospinal fluid (CSF) is an attractive route for CNS gene delivery. A safe and effective route to achieve global gene distribution in the CNS is needed, and administration of genes through the cisterna magna (CM) via a suboccipital puncture results in broad distribution in the brain and spinal cord.
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