MSCs can be modified to express missing or defective genes, such as clotting factors (Factor VIII for hemophilia A, Factor IX for hemophilia B), lysosomal enzymes (iduronidase for Hurler syndrome, glucocerebrosidase for Gaucher disease), or dystrophin for muscular dystrophy. After systemic or local administration, MSCs home to target tissues and secrete the therapeutic protein, providing sustained local therapy and potentially correcting the underlying metabolic defect.

Design MSCs to secrete high levels of trophic factors, anti-inflammatory cytokines, or growth hormones to promote regeneration and modulate immune responses. Examples include VEGF and bFGF for angiogenesis in ischemic diseases; BDNF, GDNF, and NT-3 for neuroprotection in Parkinson's, Alzheimer's, and spinal cord injury; IL-10, TGF-β, and PGE2 for immunomodulation in autoimmune disorders like rheumatoid arthritis and type 1 diabetes; and BMP-2, BMP-7 for bone regeneration. The sustained local delivery by MSCs often proves more effective and safer than systemic administration of recombinant proteins.

MSCs loaded with TRAIL (TNF-related apoptosis-inducing ligand) selectively induce apoptosis in cancer cells while sparing normal cells. Similarly, MSCs expressing interferons (IFN-α, IFN-β) or interleukins (IL-12, IL-18) can activate local antitumor immunity. Prodrug-converting enzymes such as cytosine deaminase (converts 5-fluorocytosine to 5-fluorouracil) or herpes simplex virus thymidine kinase (converts ganciclovir to cytotoxic triphosphate) can be delivered by MSCs to tumor sites, where they convert systemically administered non-toxic prodrugs into active chemotherapeutics, minimizing systemic toxicity. Preclinical studies have shown efficacy in models of breast cancer, pancreatic cancer, glioma, and lung metastases.

Engineered MSCs can cross the blood-brain barrier and infiltrate the invasive front of GBM, delivering cytotoxic genes (TRAIL, PTEN, p53) or oncolytic viruses (conditionally replicative adenoviruses) directly to tumor cells. MSC-based delivery has been shown to significantly prolong survival in orthotopic GBM models and is currently being evaluated in early-phase clinical trials.

Intravenous MSCs naturally accumulate in lung tissue due to their size and surface adhesion molecules, making them ideal vehicles for treating lung metastases from breast cancer, melanoma, osteosarcoma, and other primaries. MSCs expressing anti-angiogenic factors (endostatin, angiostatin) or immunostimulatory cytokines (GM-CSF, IL-12) have demonstrated potent suppression of metastatic growth in murine models.

High transduction efficiency (often >90%) in MSCs with transient, high-level expression. Ideal for short-term applications where sustained expression is not required, such as in vitro differentiation studies or acute in vivo treatments. We offer first-generation (E1/E3 deleted) and helper-dependent (gutless) adenoviral vectors with reduced immunogenicity and larger cargo capacity (up to 36 kb).

Non-pathogenic, low immunogenicity, and long-term expression in both dividing and non-dividing cells. Multiple serotypes (AAV1-AAV9, rh10, DJ) are available to optimize tropism for MSCs and target tissues. AAV vectors are particularly suited for in vivo delivery where integration into the host genome is not desired; they persist as episomal concatemers. Cargo capacity is limited to ~4.7 kb, but dual-vector strategies can overcome this.

Integrates into the host genome, providing stable and heritable transgene expression. Ideal for generating MSC lines with sustained therapeutic output for long-term studies or potential cell therapy products. We use third-generation, self-inactivating (SIN) lentiviral systems to enhance safety. Pseudotyping with VSV-G, measles virus (MV), or other envelopes allows efficient transduction of a wide range of MSC types. Titers routinely exceed 10⁸ TU/mL after concentration.

High-efficiency transient transfection suitable for plasmids, mRNA, siRNA, or CRISPR RNP complexes. We have optimized electroporation protocols to achieve >70% transfection efficiency in MSCs while maintaining >80% viability. This method is particularly useful for delivering gene-editing tools without viral integration.

Cationic lipid-based reagents or polymer-based reagents (polyethylenimine, PEI) offer scalable transfection with low cytotoxicity. We can optimize reagent-to-DNA ratios and delivery conditions for your specific MSC source and culture format (adherent vs. suspension, 2D vs. 3D).

Inorganic nanoparticles (gold, silica) and magnetic nanoparticles (MNPs) enable targeted delivery and controlled release of genetic material. Surface functionalization with targeting ligands can further enhance MSC-specific uptake. We offer custom formulation and characterization of nanoparticles for both in vitro and in vivo applications.

Bone marrow (BM-MSC), adipose tissue (AD-MSC), umbilical cord (UC-MSC, including Wharton's jelly and cord blood), placenta, amniotic fluid, dental pulp, and menstrual blood. Each source has distinct characteristics in terms of proliferation rate, differentiation bias, immunomodulatory potency, and secretome profile. We can advise on the most suitable source for your application.

All MSCs are characterized according to ISCT (International Society for Cellular Therapy) minimal criteria: (1) adherence to plastic; (2) surface marker expression: ≥95% positive for CD73, CD90, CD105, and ≤2% positive for CD34, CD45, CD14, CD19, HLA-DR; (3) trilineage differentiation potential into osteoblasts, chondrocytes, and adipocytes confirmed by specific staining. Additional characterization includes immunomodulatory assays (MLR suppression), cytokine profiling, and genomic stability (karyotyping).

Scalable culture systems using serum-free, xeno-free, or defined media to maintain MSC properties. We offer cryopreservation services with validated freezing media and protocols to ensure high post-thaw viability (>80%) and retained functionality. Master cell banks and working cell banks can be established for long-term studies.

CRISPR/Cas9-mediated gene knockout to study gene function or eliminate undesirable genes (e.g., MHC molecules to create hypoimmunogenic universal donor MSCs). We design and deliver sgRNA/Cas9 as plasmid, mRNA, or RNP complex, followed by single-cell cloning and sequencing validation.

Precise insertion of therapeutic genes into safe harbor loci (AAVS1, CCR5, ROSA26) using homology-directed repair (HDR) or non-homologous end joining (NHEJ) strategies. This ensures stable, predictable expression without disrupting endogenous genes. We provide donor templates (ssODN, plasmid, or AAV) and assist with selection and screening.

Introduce disease-associated mutations into healthy MSCs or correct mutations in patient-derived MSCs to generate isogenic controls for drug screening and mechanistic studies. Base editing and prime editing allow single-nucleotide changes without double-strand breaks.