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Myelodysplastic Syndrome (MDS)

Myelodysplastic syndrome (MDS) affects blood stem cells. With our pioneering efforts in MDS research, we are at the forefront of developing cutting-edge diagnostic tools and therapeutics to facilitate the effective management of MDS. As your reliable partner in MDS research, we offer unmatched support to fulfill your scientific research needs.

Introduction to MDS

MDS is a group of blood disorders characterized by ineffective production of blood cells in the bone marrow. MDS is caused by abnormalities in blood stem cells, resulting in a reduced number of healthy blood cells. Individuals affected by MDS commonly exhibit symptoms like anemia, frequent infections, and prolonged bleeding. In certain instances, MDS may advance to acute myeloid leukemia (AML), which is a more aggressive type of blood cancer. MDS is very rare, with an annual incidence rate of around 4 new cases per 100,000 individuals.

Myelodysplastic syndrome originates from a clonal hematopoietic stem cell.Fig. 1 Myelodysplastic syndrome originates from a clonal hematopoietic stem cell. (Fontenay, Michaela, et al., 2021)

Pathogenesis of MDS

The occurrence of MDS is primarily associated with a combination of risk factors encountered in daily activities and genetic factors.

Risk Factors

Risk factors include exposure to substances that can cause DNA damage.

  • Chemotherapy or radiation therapy
  • Carcinogens, such as tobacco smoke, pesticides and solvents such as benzene
  • Heavy metals, such as mercury or lead

Genetic Factors

Gene mutations and chromosomal abnormalities play key roles in the pathogenesis of MDS.

  • Gene mutations such as TP53, NRAS, RUNX1, SF3B1, TET2, ASXL1 and DNMT3A
  • Chromosomal abnormalities, such as deletions, duplications, or translocations

Targets of MDS Therapy

Target Description
Epigenetic Modifications Abnormal DNA methylation and histone modifications contribute to the dysregulation of gene expression in MDS. Drugs that modulate these epigenetic marks, such as hypomethylating agents like azacitidine and decitabine, have shown efficacy in the therapeutics of MDS.
Genetic Targets Gene mutations found in MDS may serve as targets for drug development. For instance, SF3B1-targeting inhibitors have shown promising results in MDS individuals with SF3B1 mutations. These inhibitors can correct abnormal splicing, restore normal gene expression, and enhance blood cell production.

Our Services

As a frontrunner in the field of biological research and CRO services, our company is dedicated to offering holistic solutions for the diagnostics development and therapy research of MDS. Through collaborations with industry experts, we have established an all-encompassing platform for rare diseases to facilitate MDS therapy development.

Platforms of MDS Therapy Development

Recognizing the significance of dependable animal models in MDS disease research, our company is committed to offering animal model development services that facilitate preclinical research and aid in drug discovery endeavors.

Animal Models of MDS

Xenograft Models
Xenograft models are used to establish human tumors in immunodeficient animals and are effective tools for studying malignant diseases. Establishing xenograft models typically involves transplanting an immortal human cell line (skm-1) established from MDS individuals or cells obtained directly from MDS individuals.
Induced Models
At our company, we excel at inducing MDS animal models using physicochemical and biological agents. Our scientists induce MDS by administering carcinogenic chemicals (such as benzene and alkylating agents) or exposing animals to ionizing radiation (such as gamma rays). These inducible models are low-maintenance and closely replicate key processes in MDS, making them valuable for establishing tumor models.
Genetically Engineered Models
Our company focuses on gene targeting and transgenic technology to knock out MDS-related genes. By employing complex gene positioning and DNA fragment modification, our scientists have obtained mouse embryonic stem cells carrying homologous recombination genes, resulting in mouse models exhibiting human MDS traits.
Optional Models
  • NUP98-HOXD13 (NHD13) Transgenic Model
  • NPM1+/− Model
  • APC+/− Model
  • NRAS Mutation Model
  • RUNX1 Mutation Model
  • TET2 Mutation Model
Optional Species Mice, Rats, Zebrafish, Non-human Primates (Baboons and Macaques), Others

With complete animal species resources, we can meet your diversified preclinical research including drug safety evaluation and pharmacokinetic analysis. If you are interested in our services, please feel free to contact us for more details and quotation information for related services.

References

  • Fontenay, Michaela, Batoul Farhat, and Ismael Boussaid. "Pathophysiology of myelodysplastic syndromes." Hemato 2.3 (2021): 477-495.
  • Li, Weisha, et al. "Summary of animal models of myelodysplastic syndrome." Animal Models and Experimental Medicine 4.1 (2021): 71-76.

All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.

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