Matches in SemOpenAlex for { <https://semopenalex.org/work/W2105596705> ?p ?o ?g. }
- W2105596705 endingPage "1024" @default.
- W2105596705 startingPage "1011" @default.
- W2105596705 abstract "Introduction: Glycogen storage disease (GSD) type Ia and Ib are disorders of impaired glucose homeostasis affecting the liver and kidney. GSD-Ib also affects neutrophils. Current dietary therapies cannot prevent long-term complications. In animal studies, recombinant adeno-associated virus (rAAV) vector-mediated gene therapy can correct or minimize multiple aspects of the disorders, offering hope for human gene therapy. Areas covered: A summary of recent progress in rAAV-mediated gene therapy for GSD-I; strategies to improve rAAV-mediated gene delivery, transduction efficiency and immune avoidance; and vector refinements that improve expression. Expert opinion: rAAV-mediated gene delivery to the liver can restore glucose homeostasis in preclinical models of GSD-I, but some long-term complications of the liver and kidney remain. Gene therapy for GSD-Ib is less advanced than for GSD-Ia and only transient correction of myeloid dysfunction has been achieved. A question remains as to whether a single rAAV vector can meet the expression efficiency and tropism required to treat all aspects of GSD-I, or if a multi-pronged approach is needed. An understanding of the strengths and weaknesses of rAAV vectors in the context of strategies to achieve efficient transduction of the liver, kidney and hematopoietic stem cells is required for treating GSD-I." @default.
- W2105596705 created "2016-06-24" @default.
- W2105596705 creator A5027546016 @default.
- W2105596705 creator A5033006726 @default.
- W2105596705 date "2011-04-20" @default.
- W2105596705 modified "2023-09-26" @default.
- W2105596705 title "Recombinant AAV-directed gene therapy for type I glycogen storage diseases" @default.
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