ArticleMolecular therapy : the journal of the American Society of Gene Therapy2010
Neonatal gene therapy of glycogen storage disease type Ia using a feline immunodeficiency virus-based vector.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.
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Who cites it
16 citing papers in PubMed, 26 citations in OpenAlex.
- A triple-blinded crossover study to evaluate the short-term safety of sweet manioc starch for the treatment of glycogen storage disease type Ia.Orphanet journal of rare diseases · 2021Trial
- Review
- Pseudotyped Viruses: A Useful Platform for Pre-Clinical Studies Conducted in a BSL-2 Laboratory Setting.Biomolecules · 2025Review
- Gene therapy for glycogen storage diseases.Journal of inherited metabolic disease · 2024Review
- Glycogen storage disease type I: Genetic etiology, clinical manifestations, and conventional and gene therapies.Pediatric discovery · 2023Article
- Recent development and gene therapy for glycogen storage disease type Ia.Liver research (Beijing, China) · 2017Article
- In vivo bioimaging with tissue-specific transcription factor activated luciferase reporters.Scientific reports · 2015Article
- Preclinical Development of New Therapy for Glycogen Storage Diseases.Current gene therapy · 2015Review
- Transduction of fetal mice with a feline lentiviral vector induces liver tumors which exhibit an E2F activation signature.Molecular therapy : the journal of the American Society of Gene Therapy · 2014Article
- A lentiviral vector-based, herpes simplex virus 1 (HSV-1) glycoprotein B vaccine affords cross-protection against HSV-1 and HSV-2 genital infections.Journal of virology · 2012Article
- Gene transfer to chicks using lentiviral vectors administered via the embryonic chorioallantoic membrane.PloS one · 2012Article
- Treatment of newborn G6pc(-/-) mice with bone marrow-derived myelomonocytes induces liver repair.Journal of hepatology · 2011Article
- Hepatorenal correction in murine glycogen storage disease type I with a double-stranded adeno-associated virus vector.Molecular therapy : the journal of the American Society of Gene Therapy · 2011Article
- Recombinant AAV-directed gene therapy for type I glycogen storage diseases.Expert opinion on biological therapy · 2011Review
- Altering α-dystroglycan receptor affinity of LCMV pseudotyped lentivirus yields unique cell and tissue tropism.Genetic vaccines and therapy · 2011Article
- Perinatal gene transfer to the liver.Current pharmaceutical design · 2011Review
Corrections and comments
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Authors and funding
11 authors at 3 institutions in 3 countries.
Funding
Abstract
Glycogen storage disease type Ia (GSD-Ia), also known as von Gierke disease, is caused by a deficiency of glucose-6-phosphatase-alpha (G6Pase), a key enzyme in glucose homeostasis. From birth, affected individuals cannot maintain normal blood glucose levels and suffer from a variety of metabolic disorders, leading to life-threatening complications. Gene therapy has been proposed as a possible option for treatment of this illness. Vectors have been constructed from feline immunodeficiency virus (FIV), a nonprimate lentivirus, because the wild-type virus does not cause disease in humans. Previously, we have shown that these vectors are capable of integrating stably into hepatocyte cell lines and adult murine livers and lead to long-term transgene expression. In the current work, we have assessed the ability to attenuate disease symptoms in a murine model of GSD-Ia. Single administration of FIV vectors containing the human G6Pase gene to G6Pase-alpha(-/-) mice did not change the biochemical and pathological phenotype. However, a double neonatal administration protocol led to normalized blood glucose levels, significantly extended survival, improved body weight, and decreased accumulation of liver glycogen associated with the disease. This approach shows a promising paradigm for treating GSD-Ia patients early in life thereby avoiding long-term consequences.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.