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Primary immunodeficiency diseases are representative of potential genetic disorders for new therapeutic approaches. Utilizing autologous CD34+ hematopoietic stem cells, our laboratory is creating new drugs for patients with PID. The collection of peripheral blood CD34+ cells from patients with these immunodeficiency disorders could have dramatic effects on these disorders. Objectives: To develop granulocyte colony stimulating factor mobilized peripheral blood CD34+ hematopoietic stem cells for laboratory research studies into DOCK8 deficiency, LAD-1, and GATA2 Deficiency. Patients 18-40 years old with DOCK8 deficiency, LAD-1, and GATA2 Deficiency who meet the eligibility requirements will be considered for this procedure. Patients 18-40 years old with DOCK8 deficiency, LAD-1, and GATA2 deficiency, with G-filgrastim will be given five days of G-filgrastim followed by a single apheresis. Cell Processing Section of the Department of Transfusion Medicine will select and freeze in aliquots for CD34+ cells.
Source link: https://clinicaltrials.gov/ct2/show/NCT01212055
An autosomal recessive disorder of leukocyte function is classified as a factor in autosomal recessive disorder type 1. It is a monoclonal antibody directed against human interleukins IL-12 and IL-23's p40 subunit. Ustekinumab has medical consequences by interfering with the TH1 and TH17 cytokine pathways. Blockade of IL-17, which is highly present in the gingiva of people with LAD1, is a common condition in gingival disease, and can reduce bacterial load and resolve inflammatory gingival disease, according to previous research. Our aim is to investigate the possibility of ustekinumab as a treatment for LAD1 inflammatory disease. In ten patients with LAD1, the goal of this open-label, proof-of-concept study is to determine the safety and tolerability of ustekinumab. Over the course of 1 year, participants will receive 5 doses of ustekinumab via subcutaneous injection.
Source link: https://clinicaltrials.gov/ct2/show/NCT03366142
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