An article published this year in “SCIENTIFIC REPORTS” using our Cell
Fixation & Permeabilization IntraCell Kit, by our customers from
Division of Immunology, University Children’s Hospital and Children’s Research
Center, Zurich, Switzerland, in the study of how CRISPR/Cas9-generated
p47phox-deficient cell line for Chronic Granulomatous Disease gene therapy
vector development. Congrats and Thanks.
Summary:
Development of gene therapy
vectors requires cellular models reflecting the genetic background of a disease
thus allowing for robust preclinical vector testing. For human
p47phox-deficient chronic granulomatous disease (CGD) vector testing we
generated a cellular model using clustered regularly interspaced short
palindromic repeats (CRISPR)/Cas9 to introduce a GT-dinucleotide deletion (ΔGT)
mutation in p47phox encoding NCF1 gene in the human acute myeloid leukemia PLB-985
cell line. CGD is a group of hereditary immunodeficiencies characterized by
impaired respiratory burst activity in phagocytes due to a defective phagocytic
nicotinamide adenine dinucleotide phosphate (NADPH) oxidase. In Western
countries autosomal-recessive p47phox-subunit deficiency represents the second
largest CGD patient cohort with unique genetics, as the vast majority of
p47phox CGD patients carries ΔGT deletion in exon two of the NCF1 gene. The
established PLB-985 NCF1 ΔGT cell line reflects the most frequent form of
p47phox-deficient CGD genetically and functionally. It can be differentiated to
granulocytes efficiently, what creates an attractive alternative to currently
used iPSC models for rapid testing of novel gene therapy approaches.
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