Publications by authors named "Y Lajat"
Article Synopsis
- Severe deficiency in β-glucuronidase (β-glu) causes mucopolysaccharidosis (MPS) VII, leading to various symptoms, particularly in early childhood, including neurological issues due to abnormal accumulation of glycosaminoglycans (GAGs) and gangliosides.
- The study investigates a gene therapy approach using a helper-dependent canine adenovirus (CAV-2) vector with a human GUSB expression cassette to treat MPS VII in dogs, aiming to correct the neurological defects associated with the disease.
- Results showed that the CAV-2 vector effectively targeted neurons, facilitated transport within the brain, and corrected neuropathology in treated areas, suggesting potential for treating MPS VII and similar
Recent trials in patients with neurodegenerative diseases documented the safety of gene therapy based on adeno-associated virus (AAV) vectors deposited into the brain. Inborn errors of the metabolism are the most frequent causes of neurodegeneration in pre-adulthood. In Sanfilippo syndrome, a lysosomal storage disease in which heparan sulfate oligosaccharides accumulate, the onset of clinical manifestation is before 5 years.
View Article and Find Full Text PDFObject: Deep brain stimulation (DBS) has been shown to be an effective treatment for various types of movement disorders. High-frequency stimulation is applied to specific brain targets through an implanted quadripolar lead connected to a pulse generator. These leads can be used for creating lesions in the brain.
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