Cystic Fibrosis Leukopak

LeukoCore Cystic Fibrosis Leukopak for CFTR and Inflammation Research

LeukoCore Cystic Fibrosis Leukopak biospecimens are collected from IRB-consented donors clinically diagnosed with cystic fibrosis through IRB-approved apheresis procedures. Cystic fibrosis is a monogenic autosomal recessive disease caused by biallelic pathogenic variants in CFTR, which encodes a cAMP-regulated chloride and bicarbonate channel; loss of channel function dehydrates airway surface liquid, impairs mucociliary clearance and establishes a cycle of chronic infection and neutrophil-dominated inflammation. Each leukopak contains large quantities of peripheral blood mononuclear cells, making it suited to high-cell-number work in immunology, gene correction and drug development.

Why a Leukopak for Cystic Fibrosis Research

• Cystic fibrosis is defined by a specific protein defect rather than by autoimmunity. F508del is the most common pathogenic variant, and variant class determines both the trafficking or gating defect involved and eligibility for CFTR modulator therapy — making donor genotype the single most important selection criterion.
• CFTR is expressed in myeloid cells including monocytes, macrophages and neutrophils, and cell-intrinsic contributions of CFTR dysfunction to impaired phagocyte function and dysregulated inflammatory signalling have been described. A leukopak supplies enough starting material to interrogate this directly.
• Monocyte yields from a single leukopak support differentiation into large numbers of monocyte-derived macrophages, the workhorse cell type for phagocytosis, killing and inflammatory response assays in cystic fibrosis.
• Gene editing and gene correction programmes targeting CFTR require cell numbers well beyond what a standard venipuncture provides. Leukopaks are the practical source for CRISPR and base-editing optimisation in patient-derived cells.
• Patient-derived mononuclear cells are a common starting point for iPSC reprogramming, enabling isogenic corrected and uncorrected lines from the same donor genotype.
• The systemic inflammatory phenotype of cystic fibrosis — including elevated inflammatory cytokines and altered neutrophil biology — is best characterised with sufficient cells for parallel functional, transcriptomic and proteomic readouts from one donor.

Donor Stratification Available

• CFTR genotype where available, including F508del homozygous, F508del heterozygous, and other variant combinations
• CFTR modulator treatment status: on elexacaftor/tezacaftor/ivacaftor or other modulator, modulator-naive, or modulator-ineligible by genotype
• Lung function by FEV1 percent predicted where reported
• Pancreatic sufficiency versus insufficiency
• Comorbidity including cystic fibrosis-related diabetes and hepatobiliary involvement
• Chronic airway colonisation status and age group
• Matched healthy controls available

Product Features

• Research Use Only (RUO) human leukopaks
• Clinically confirmed cystic fibrosis donors
• High cell yield for large-scale studies
• Processed and shipped within 24 hours of collection
• Fresh or cryopreserved options available
• IRB-approved and ethically sourced
• Infectious disease testing
• HLA-A typing
• Screen donors with LeukoLot™

De-identified Donor Data

• Verified cystic fibrosis diagnosis from healthcare providers
• Donor demographics: age, sex assigned at birth, race/ethnicity
• Self-reported medication history and allergies
• CFTR genotype and modulator therapy where available
• Relevant clinical data (if available)

Applications

• Monocyte-derived macrophage differentiation and functional assays
• Phagocytosis, bacterial killing and inflammatory signalling studies in CFTR-deficient myeloid cells
• CFTR gene editing, base editing and gene correction optimisation in patient-derived cells
• iPSC reprogramming from donor mononuclear cells for isogenic disease modelling
• CFTR modulator pharmacology and response research in immune cells
• Cytokine profiling and cell-based assays of systemic cystic fibrosis inflammation
• Multi-omics studies including single-cell RNA sequencing and ATAC-seq
• Immune cell characterisation and immunophenotyping in cystic fibrosis

Other Cystic Fibrosis Specimen Types

• Cystic Fibrosis PBMC
• Cystic Fibrosis Plasma
• Cystic Fibrosis Serum
• Cystic Fibrosis Whole Blood

Compliance and Quality Assurance

• Collected under IRB oversight with donor e-consent (21 CFR Part 11 compliant)
• HIPAA-compliant handling of donor information
• Rigorous donor screening and documentation

Ordering & Customization

Cystic Fibrosis Peripheral Blood Leukopak samples are available fresh or cryopreserved. For pricing, current availability, or to discuss a genotype-stratified cohort, contact learnmore@sanguinebio.com. Need a different condition, or want to check current availability across our full leukopak inventory? Visit our human leukopak page to browse other disease-state options and live stock status. For international shipping or regulatory documentation, please contact us to confirm regional compliance and shipping availability.