ArticleMolecular therapy : the journal of the American Society of Gene Therapy2009
Hepatocyte transplantation improves phenotype and extends survival in a murine model of intermediate maple syrup urine disease.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
21 citing papers in PubMed, 31 citations in OpenAlex.
- Article
- Review
- Hyperactivation of mTOR and AKT in a cardiac hypertrophy animal model of Friedreich ataxia.Heliyon · 2022Article
- Liver transplantation for pediatric inherited metabolic liver diseases.World journal of hepatology · 2021Review
- Clinical perspective on the use of human amniotic epithelial cells to treat congenital metabolic diseases with a focus on maple syrup urine disease.Stem cells translational medicine · 2021Article
- [Maple syrup urine disease and gene mutations in twin neonates].Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics · 2016Article
- Overexpression of transcription factor Foxa2 and Hnf1α induced rat bone mesenchymal stem cells into hepatocytes.Cytotechnology · 2016Article
- Metformin inhibits Branched Chain Amino Acid (BCAA) derived ketoacidosis and promotes metabolic homeostasis in MSUD.Scientific reports · 2016Article
- Dynamic Arginine Methylation of Tumor Necrosis Factor (TNF) Receptor-associated Factor 6 Regulates Toll-like Receptor Signaling.The Journal of biological chemistry · 2015Article
- The history and use of human hepatocytes for the treatment of liver diseases: the first 100 patients.Current protocols in toxicology · 2014Article
- Branched-chain amino acid metabolism: from rare Mendelian diseases to more common disorders.Human molecular genetics · 2014Review
- Benzothiophene carboxylate derivatives as novel allosteric inhibitors of branched-chain α-ketoacid dehydrogenase kinase.The Journal of biological chemistry · 2014Article
- Brain-blood amino acid correlates following protein restriction in murine maple syrup urine disease.Orphanet journal of rare diseases · 2014Article
- Therapeutic hepatocyte transplant for inherited metabolic disorders: functional considerations, recent outcomes and future prospects.Journal of inherited metabolic disease · 2014Review
- Placental stem cell correction of murine intermediate maple syrup urine disease.Hepatology (Baltimore, Md.) · 2013Article
- Mutation of zebrafish dihydrolipoamide branched-chain transacylase E2 results in motor dysfunction and models maple syrup urine disease.Disease models & mechanisms · 2012Article
- Advances and challenges in the treatment of branched-chain amino/keto acid metabolic defects.Journal of inherited metabolic disease · 2012Review
- Therapeutic liver repopulation for phenylketonuria.Journal of inherited metabolic disease · 2010Review
- Chimeric mice with humanized liver: tools for the study of drug metabolism, excretion, and toxicity.Methods in molecular biology (Clifton, N.J.) · 2010Review
- Hepatocyte transplantation (HTx) corrects selected neurometabolic abnormalities in murine intermediate maple syrup urine disease (iMSUD).Biochimica et biophysica acta · 2009Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
10 authors at 1 institution in 2 countries.
Funding
Abstract
Maple syrup urine disease (MSUD; OMIM 248600) is an inborn error of metabolism of the branched chain alpha-ketoacid dehydrogenase (BCKDH) complex that is treated primarily by dietary manipulation of branched-chain amino acids (BCAA). Dietary restriction is lifelong and compliance is difficult. Liver transplantation significantly improves outcomes; however, alternative therapies are needed. To test novel therapies such as hepatocyte transplantation (HTx), we previously created a murine model of intermediate MSUD (iMSUD), which closely mimics human iMSUD. LacZ-positive murine donor hepatocytes were harvested and directly injected (10(5) cells/50 microl) into liver of iMSUD mice (two injections at 1-10 days of age). Donor hepatocytes engrafted into iMSUD recipient liver, increased liver BCKDH activity, improved blood total BCAA/alanine ratio, increased body weight at weaning, and extended the lifespan of HTx-treated iMSUD mice compared to phosphate-buffered saline (PBS)-treated and untreated iMSUD mice. Based on these data demonstrating partial metabolic correction of iMSUD in a murine model, coupled to the fact that multiple transplants are possible to enhance these results, we suggest that HTx represents a promising therapeutic intervention for MSUD that warrants further investigation.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
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.