Evaluation of neuroretina following i.v. or intra-CSF AAV9 gene replacement in mice with MPS IIIA, a childhood dementia.
Helen BeardLeanne K WinnerAndrew ShoubridgeEmma Parkinson-LawrenceAdeline A LauSiti N MubarokahTabitha-Rose LanceBarbara KingWilliam ScottMarten F SnelPaul J TrimKim M HemsleyPublished in: CNS neuroscience & therapeutics (2024)
Confirmatory studies are needed in adult mice; however, these data indicate that i.v. AAV9-sulfamidase infusion leads to superior outcomes in neuroretina, and cerebrospinal fluid-delivered AAV9 may need to be supplemented with another therapeutic approach for optimal patient quality of life.
Keyphrases
- cerebrospinal fluid
- gene therapy
- high fat diet induced
- mild cognitive impairment
- low dose
- case report
- big data
- childhood cancer
- type diabetes
- copy number
- gene expression
- genome wide
- metabolic syndrome
- wild type
- skeletal muscle
- transcription factor
- young adults
- deep learning
- genome wide identification
- adipose tissue
- artificial intelligence
- glycemic control