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Muscular Dystrophy Cell Model Products

Introduction Types Advantages Applications FAQs Related Product Sections Product List

Introduction

The development of effective therapies for devastating Muscular Dystrophies (MD), like DMD and FSHD, is hampered by traditional preclinical models that are ill-suited for today's advanced treatments, such as ASOs and CRISPR gene editing. Consequently, the industry urgently needs scalable, reproducible, and highly human-relevant platforms to improve screening accuracy.

As a leading life sciences provider, Creative Biolabs addresses this critical bottleneck. Our comprehensive therapeutic discovery platform integrates advanced technologies, from human pluripotent stem cells (hPSCs) to precision gene editing, offering global clients an accelerated and more reliable path to innovative drug development. Our team of scientific experts is available to understand your specific needs and design a solution that works for you. Contact Our Team for More Information and to Discuss Your Project.

For a detailed enumeration of our specific offerings, please consult our comprehensive Product List.

Types of Models We Offer

We provide a portfolio of validated cell models and the capability to create new ones on demand.

Types Description
Duchenne Muscular Dystrophy (DMD) Models Featuring common or custom deletions/mutations in the DMD gene.
Facioscapulohumeral Dystrophy (FSHD) Models hPSC-derived models exhibiting aberrant DUX4 expression and associated cellular phenotypes.
Becker Muscular Dystrophy (BMD) Models Models expressing truncated but partially functional dystrophin protein.
Custom-Generated Models Tell us the gene, mutation, and cell type, and we will build it for you.

The Creative Biolabs Advantage: De-risking the Path to Clinic

Unprecedented Mechanistic Insight

Use our hPSC platform to understand not just if your drug works, but how it impacts the early stages of human muscle development and disease.

Quantitative Accuracy

Move beyond ambiguous, semi-quantitative data. Our immortalized models enable robust, reproducible quantification of dystrophin and utrophin, providing you with clear, actionable results.

Proven Predictive Power

Results generated in our immortalized cell models have shown a strong correlation with in vivo outcomes in humanized DMD mouse models. This gives you unprecedented confidence that the candidates you advance to more complex and expensive animal studies have the highest potential for success.

Comprehensive Validation

Every model we provide undergoes a series of rigorous characterization analyses to confirm its genetic integrity, myogenic potential, and target protein expression.

Speed and Accessibility:

We eliminate the time and logistical hurdles required to source and validate primary cells. Your team will receive a validated, scalable tool ready for immediate use.

Applications

Our Muscular Dystrophy Cell Models are powerful tools for a wide range of research applications:

Applications Description
High-Throughput Screening (HTS) Screen large compound libraries to identify novel therapeutic hits with confidence.
Lead Optimization Characterize and rank your most promising candidates based on efficacy and potency in a human-relevant system.
Mechanism of Action (MoA) Studies Elucidate the precise biological pathways your compounds are modulating.
Gene Therapy & ASO Validation Test the efficacy and specificity of genetic medicines, including antisense oligonucleotides (ASOs) for exon skipping, in a controlled environment.
Biomarker Discovery Identify and validate novel biomarkers that correlate with disease pathology or therapeutic response.
Toxicity Screening Assess potential off-target or cytotoxic effects early in the development process.
A picture that presents Model for necroptosis involvement in Duchenne muscular dystrophy. (Bencze, et al., 2022) (OA Literature)Fig.1 Model for necroptosis involvement in Duchenne muscular dystrophy. 1

FAQs

  • Can you create a model for a very rare mutation?
    Yes. Our CRISPR/Cas9 platform allows us to engineer virtually any genetic mutation, including rare or novel variants, into either a healthy or patient-derived genetic background.
  • What's the main difference between your immortalized and iPSC-derived models?
    Immortalized models are excellent for robust, scalable screening of myoblasts. iPSC-derived models are ideal for studying developmental biology and early disease onset, as they can be differentiated into mature, functional myotubes that recapitulate later-stage disease phenotypes.
  • What is the lead time for a custom model?
    Lead times vary based on complexity, but typically range from 12-16 weeks. We provide regular updates and milestones throughout the process.
  • Can you model muscular dystrophies other than DMD and FSHD?
    Yes. We are constantly expanding our portfolio. We can generate custom models for Limb-Girdle, Myotonic, Congenital, and other muscular dystrophies. Please inquire about your specific disease of interest.
  • How do I get started with a project or place an order?
    Simply click the contact link on this page or email our scientific team. A PhD-level specialist will connect with you to discuss your technical requirements and prepare a detailed project proposal and quote.

The advancement of muscular dystrophy therapeutics necessitates a paradigm shift from traditional models to more predictive, human-relevant systems. Leveraging robust in vitro platforms is paramount for de-risking preclinical pipelines and ensuring only candidates with the highest probability of success advance to in vivo studies.

Creative Biolabs provides these critical cellular tools. Our platforms enable unparalleled mechanistic insight and validation of therapeutic strategies. Contact our scientific team to discuss how our multi-platform DMD modeling can accelerate your program's translation to the clinic.

Related Product Sections

For more categories of Neuromuscular & Genetic Disease related Research Tools, please visit the following link:

Reference

  1. Bencze, Maximilien. "Mechanisms of myofibre death in muscular dystrophies: the emergence of the regulated forms of necrosis in myology." International Journal of Molecular Sciences 24.1 (2022): 362. DOI: 10.3390/ijms24010362. Use under Open Access license CC BY 4.0, without modification.