Mucopolysaccharidosis enzyme production by bone marrow and dental pulp derived human mesenchymal stem cells

Molecular Genetics and Metabolism
Matilda JacksonSharon Byers

Abstract

Mucopolysaccharidoses (MPS) are inherited metabolic disorders that arise from a complete loss or a reduction in one of eleven specific lysosomal enzymes. MPS children display pathology in multiple cell types leading to tissue and organ failure and early death. Mesenchymal stem cells (MSCs) give rise to many of the cell types affected in MPS, including those that are refractory to current treatment protocols such as hematopoietic stem cell (HSC) based therapy. In this study we compared multiple MPS enzyme production by bone marrow derived (hBM) and dental pulp derived (hDP) MSCs to enzyme production by HSCs. hBM MSCs produce significantly higher levels of MPS I, II, IIIA, IVA, VI and VII enzyme than HSCs, while hDP MSCs produce significantly higher levels of MPS I, IIIA, IVA, VI and VII enzymes. Higher transfection efficiency was observed in MSCs (89%) compared to HSCs (23%) using a lentiviral vector. Over-expression of four different lysosomal enzymes resulted in up to 9303-fold and up to 5559-fold greater levels in MSC cell layer and media respectively. Stable, persistent transduction of MSCs and sustained over-expression of MPS VII enzyme was observed in vitro. Transduction of MSCs did not affect the ability of the cells to d...Continue Reading

References

Aug 1, 1992·Genomics·H S ScottC P Morris
Jan 1, 1995·Journal of Inherited Metabolic Disease·E G ShapiroW Krivit
Mar 29, 1994·Proceedings of the National Academy of Sciences of the United States of America·P M Challita, D B Kohn
Mar 5, 1996·Proceedings of the National Academy of Sciences of the United States of America·L J FairbairnT M Dexter
Jan 1, 1996·Journal of Inherited Metabolic Disease·E A KarpovaO P van Diggelen
Mar 28, 1998·The Journal of Biological Chemistry·C MiechB Schmidt
Apr 2, 1999·Science·M F PittengerD R Marshak
Feb 24, 2001·The New England Journal of Medicine·R QuartoM Marcacci
Feb 16, 2002·Methods : a Companion to Methods in Enzymology·K J Livak, T D Schmittgen
May 7, 2002·Molecular Therapy : the Journal of the American Society of Gene Therapy·Xian-Yang ZhangJakob Reiser
Jun 22, 2002·Nature·Yuehua JiangCatherine M Verfaillie
Jul 31, 2002·Journal of Dental Research·S GronthosS Shi
Apr 30, 2003·Proceedings of the National Academy of Sciences of the United States of America·Masako MiuraSongtao Shi
Jun 15, 2004·Molecular Therapy : the Journal of the American Society of Gene Therapy·A Alex HoflingMark S Sands
Jul 6, 2004·Molecular Therapy : the Journal of the American Society of Gene Therapy·Shuyuan YaoJames Ellis
Oct 21, 2004·Journal of Cell Science·Fernando Anjos-AfonsoDominique Bonnet
Jun 2, 2007·British Journal of Haematology·James M FoxJim Middleton
Jan 2, 2009·Pediatrics·Joseph MuenzerUNKNOWN International Consensus Panel on Management and Treatment of Mucopolysaccharidosis I
Nov 12, 2009·Proceedings of the National Academy of Sciences of the United States of America·Daren WangDao Pan
Dec 8, 2009·Biotechnology and Applied Biochemistry·Hui QianJing Sun
Jul 7, 2010·Arthritis Research & Therapy·Hans U SchererRené E M Toes
Jan 15, 2011·Cytotherapy·Partow Kebriaei, Simon Robinson

❮ Previous
Next ❯

Citations

Oct 27, 2016·Molecular Genetics and Metabolism·Ainslie L K Derrick-RobertsSharon Byers
Apr 7, 2017·Genetics in Medicine : Official Journal of the American College of Medical Genetics·Matthias ZielonkaMarkus Ries
Oct 17, 2020·Current Osteoporosis Reports·Zhirui JiangLachlan J Smith

❮ Previous
Next ❯

Related Concepts

Related Feeds

Adult Stem Cells

Adult stem cells reside in unique niches that provide vital cues for their survival, self-renewal, and differentiation. They hold great promise for use in tissue repair and regeneration as a novel therapeutic strategies. Here is the latest research.

Cell-Type Specific Viral Vectors

Viral vectors are used in biological research and therapy to deliver genetic material into cells. However, the efficiency of viral vectors varies depending on the cell type. Here is the latest research on cell-type-specific viral vectors.

Aminoglycosides

Aminoglycoside is a medicinal and bacteriologic category of traditional Gram-negative antibacterial medications that inhibit protein synthesis and contain as a portion of the molecule an amino-modified glycoside. Discover the latest research on aminoglycoside here.

Cell-Type-Specific Viral Vectors (ASM)

Viral vectors are used in biological research and therapy to deliver genetic material into cells. However, the efficiency of viral vectors varies depending on the cell type. Here is the latest research on cell-type-specific viral vectors.

Aminoglycosides (ASM)

Aminoglycoside is a medicinal and bacteriologic category of traditional Gram-negative antibacterial medications that inhibit protein synthesis and contain as a portion of the molecule an amino-modified glycoside. Discover the latest research on aminoglycoside here.

Calcium & Bioenergetics

Bioenergetic processes, including cellular respiration and photosynthesis, concern the transformation of energy by cells. Here is the latest research on the role of calcium in bioenergetics.

Blood And Marrow Transplantation

The use of hematopoietic stem cell transplantation or blood and marrow transplantation (bmt) is on the increase worldwide. BMT is used to replace damaged or destroyed bone marrow with healthy bone marrow stem cells. Here is the latest research on bone and marrow transplantation.

Cell-Type-Specific Viral Vectors

Viral vectors are used in biological research and therapy to deliver genetic material into cells. However, the efficiency of viral vectors varies depending on the cell type. Here is the latest research on cell-type-specific viral vectors.