Molecular Transfer and Therapy

IBE Department

Molecular Transfer and Therapy

The Molecular Transfer and Therapy research group led by Dr. Elizabeth Rosado Balmayor focuses on developing innovative technologies to efficiently deliver genes, transcripts and other biomolecules inside cells. Our group is interested in the combination of complex, multiphasic biomaterials and morphogens to better target specific cell populations. The ultimate goal is to guide cellular differentiation towards pathways promoting musculoskeletal homeostasis and regeneration.

Our main research interests include 1) development of novel particulate systems, dendrimers, lipids and micelles for non-viral gene and transcript delivery, 2) development of gene- and transcript-activated matrices (GAMs and TAMs) and 3) fabrication of 3D multiphasic scaffolds that mimic native tissue interfaces and their functionalization and incorporation of biomolecules. Our group has a strong background in nano-/micro-particle fabrication, functionalization and biomolecule loading. We also develop magnetic and superparamagnetic systems for specific cell separation and cell target applications. Dendrimers and lipid systems have been extensively used by our group as well.

We directly test our gene- and transcript-activated materials for cell internalization, efficient transfection and protein production. Our group has developed several ex vivo models for gene and transcript transfer for more translational investigation. In vivo, we have optimized several rodent models for gene and transcript delivery to bone, tendon and bone-to-tendon interface.

If you are interested in reading more on RNA therapeutics in tissue engineering, have a look at this special focus issue on "RNA Therapeutics for Tissue Engineering" (guest editors: Elizabeth R. Balmayor and Christopher H. Evans).

Main collaborations

Funding sources

U.S. National Institutes of Health – NIH
Use of Chemically Modified RNA to Enhance Bone Healing
September 1, 2019 - August 31, 2024, Mayo clinic, USA

NIH logo


European Union - Horizon 2020

cmRNAbone – 3D Printed-Matrix Assisted Chemically Modified RNAs Bone Regenerative Therapy for Trauma and Osteoporotic Patients 
January 1, 2020 - December 31, 2023

cmRNAbone

Selected publications

  • Balmayor, E. R. Targeted delivery as key for the success of small osteoinductive molecules. Advanced Drug Delivery Reviews. 2015;94: 13-27, https://www.ncbi.nlm.nih.gov/pubmed/25959428
  • Balmayor, E. R.; Geiger, J. P.; Aneja, M. K.; Berezhanskyy, T.; Utzinger, M.; Mykhaylyk, O.; Rudolph, C.; Plank, C. Chemically modified RNA induces osteogenesis of stem cells and human tissue explants as well as accelerates bone healing in rats. Biomaterials. 2016;87: 131-146, https://www.ncbi.nlm.nih.gov/pubmed/26923361
  • Mueller, C. W.; Hildebrandt, K.; Gerich, T.; Krettek, C.; van Griensven, M.; Balmayor, E. R. BMP-2 transduced human bone marrow stemcells enhance neo-bone formation in a rat critical size femur defect. Journal of Tissue Engineering and Regenerative Medicine. 2017;11(4): 1122-1131, https://www.ncbi.nlm.nih.gov/pubmed/25783748
  • Balmayor, E.R.; Geiger, J.P.; Koch, C.; Aneja, M.K.; van Griensven M.; Rudolph, C.; Plank, C. Modified mRNA for BMP 2 in combination with biomaterials serves as a transcript activated matrix for effectively inducing osteogenic pathways in stem cells. Stem Cells and Development. 2017;26(1): 25-34, https://www.ncbi.nlm.nih.gov/pubmed/27676276
  • Zhang, W.; De La Vega, R. E.; Coenen, M. J.; Peniche Silva, M. J.; Aneja, M. K.; Plank, C.; van Griensven, M.; Evans, C. H.; Balmayor, E. R. An improved, chemically modified RNA encoding BMP-2 enhances osteogenesis in vitro and in vivo. Tissue Engineering Part A. 2019;25(1-2): 131-144, https://www.ncbi.nlm.nih.gov/pubmed/30009674

Image

Chemically modified mRNAs (cmRNAs) encoding therapeutic proteins are loaded into a 3D scaffold to produce Transcript-Activated Matrixes termed TAMs. TAMs deliver loaded cmRNAs to target cells that will then produce and secrete relevant proteins. An example of such proteins may be TGF-beta or BMP-2. TGF-beta is important to stimulate stem cells and other progenitor cells towards tenocyte differentiation. BMP-2, on the other hand, will stimulate stem cells and/or osteoprogenitor to osteogenic differentiation. The ultimate goal is to guide cellular differentiation towards pathways promoting musculoskeletal homeostasis and regeneration.Chemically modified mRNAs (cmRNAs) encoding therapeutic proteins are loaded into a 3D scaffold to produce Transcript-Activated Matrixes termed TAMs. TAMs deliver loaded cmRNAs to target cells that will then produce and secrete relevant proteins. An example of such proteins may be TGF-beta or BMP-2. TGF-beta is important to stimulate stem cells and other progenitor cells towards tenocyte differentiation. BMP-2, on the other hand, will stimulate stem cells and/or osteoprogenitor to osteogenic differentiation. The ultimate goal is to guide cellular differentiation towards pathways promoting musculoskeletal homeostasis and regeneration.

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