The TransLab offers a comprehensive battery of cellular and molecular assays to help guide personalized therapy or support patient monitoring. We can assist with simple, routine processes as well as complex, non-routine laboratory developed tests. Our goal is to support clinical research needs whatever they may be. We have 19 years of experience and expertise covering the fields of cancer immunotherapy (CAR-T cell trials), regenerative medicine and gene therapy (lentiviral, retroviral or gene editing). Most notably, the TransLab has played a key role in the development of clinical trials at Boston Children's Hospital for several genetic deficiencies:
- X-linked Severe Combined ImmunoDeficiency (X-SCID)
- Wiskott-Aldrich Syndrome (WAS),
- X-linked Chronic Granulomatous Disease (X-CGD)
- Childhood Cerebral Adrenoleukodystrophy (CCALD)
- Sickle Cell Disease (SCD)
- Hemophilia A
- Diamon-Blackfan Anemia (DBA)
Examples of processes and assays the TransLab has used to support IND-enabling studies or phase I/II clinical trials (drug product characterization and patient monitoring):
- Blood and bone marrow processing for isolation & cryopreservation of mononuclear cells
- Large-scale cell selection (CliniMACS®) from mobilized peripheral blood or apheresis products
- Nucleic acid extraction
- Flow cytometry-based immunophenotyping, cell population isolation and functional assays (cytotoxic assay, proliferation, apoptosis)
- Molecular assays: transgene identity assay by PCR, vector copy number assay by qPCR or ddPCR, detection of gene edits using drop-off assays, gene expression by RT-qPCR, ddPCR or QuantiGeneä RNA assay, DNA libraries
- Biomarkers analysis using plate-bound immunoassays: ELISA or multiplex immunoassay (Luminex MAGPIX®)
Additionally, the TransLab has great expertise in complex cell culture and provide supports for genetic testing on skin biopsy-derived fibroblasts and screening of therapeutic candidates antisense oligonucleotides.