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Generation of nanobodies targeting the human, transcobalamin-mediated vitamin B12 uptake route
Bloch JS, Sequeira JM, Ramírez AS, Quadros EV, Locher KP (2021) Generation of nanobodies targeting the human, transcobalamin-mediated vitamin B12 uptake route. bioRxiv 2021.08.16.456495. doi: 10.1101/2021.08.16.456495
Related Products: Streptavidin-ZAP (Cat. #IT-27)
Autosomal recessive osteopetrosis: mechanisms and treatments
Penna S, Villa A, Capo V (2021) Autosomal recessive osteopetrosis: mechanisms and treatments. Dis Model Mech 14(5):dmm048940. doi: 10.1242/dmm.048940
Summary: Autosomal recessive osteopetrosis (ARO) is a severe inherited bone disease characterized by defective osteoclast resorption or differentiation. Novel therapeutic approaches are needed for ARO patients. The authors review preclinical and proof-of-concept studies, such as gene therapy, systematic administration of deficient protein, in utero Hematopoietic stem cell transplantation (HSCT) and gene editing.
Usage: Efficacy in HSCT conditioning was demonstrated with CD45.2-SAP (biotinylated Anti-CD45 mixed with Streptavidin-ZAP). In mice, CD45.2–SAP preserved normal bone marrow architecture compared to total body irradiation, which instead reduced vascular integrity and bone marrow cellularity. Mice conditioned with CD45.2–SAP rapidly recovered their peripheral myeloid cells and had a survival advantage when exposed to infections (3 mg/kg iv; Palchaudhuri et al.). Additionally, conditioning with CD45.2–SAP resulted in significant chimerism after transplantation, even in a pathological mouse model (3 mg/kg iv; Castiello et al.).
Related Products: Streptavidin-ZAP (Cat. #IT-27)
See Also:
- Palchaudhuri R et al. Non-genotoxic conditioning for hematopoietic stem cell transplantation using a hematopoietic-cell-specific internalizing immunotoxin. Nat Biotechnol 34:738-745, 2016.
- Castiello MC et al. Efficacy and safety of anti-CD45-saporin as conditioning agent for RAG deficiency. J Allergy Clin Immunol 147(1):309-320.e6, 2021.
Activated factor X targeted stored in platelets as an effective gene therapy strategy for both hemophilia A and B
Wang D, Shao X, Wang Q, Pan X, Dai Y, Yao S, Yin T, Wang Z, Zhu J, Xi X, Chen Z, Chen S, Zhang G (2021) Activated factor X targeted stored in platelets as an effective gene therapy strategy for both hemophilia A and B. Clin Transl Med 11(3):e375. doi: 10.1002/ctm2.375
Summary: Treatment of hemophiliacs with inhibitors remains challenging, and new treatments are in urgent need. Coagulation factor X plays a critical role in downstream blood coagulation cascade, which could serve as a bypassing agent for hemophilia therapy. Target expression of the FXa precursor to platelets can generate a storage pool of FXa in platelet α-granules, the platelet-stored FXa is effective in treating HA and HB with inhibitors, suggesting that this could be a novel choice for hemophilia patients with inhibitors.
Usage: A single dose of CD45.2-SAP (biotinylated Anti-CD45 mixed with Streptavidin-ZAP) enabled efficient engraftment of donor cells (> 90%) and full correction of sickle-cell anemia. (3 mg/kg iv; Palchaudhuri et al.).
Related Products: Streptavidin-ZAP (Cat. #IT-27)
Bone marrow-derived myeloid progenitors in the leptomeninges of adult mice
Koeniger T, Bell L, Mifka A, Enders M, Hautmann V, Mekala SR, Kirchner P, Ekici AB, Schulz C, Wörsdörfer P, Mencl S, Kleinschnitz C, Ergün S, Kuerten S (2021) Bone marrow-derived myeloid progenitors in the leptomeninges of adult mice. Stem Cells 39(2):227-239. doi: 10.1002/stem.3311
Summary: This report confirms the presence of myeloid progenitors at the meningeal border of the brain and lays the foundation to unravel their possible functions in CNS surveillance and local immune cell production. Compared to bone marrow transfer after whole-body irradiation, chimerism developed more slowly in the CD45-SAP (biotinylated anti-CD45 mixed with Streptavidin-ZAP) model and only reached around 50% in the blood myeloid compartment 15 weeks after transplantation.
Related Products: Streptavidin-ZAP (Cat. #IT-27)
Efficacy and safety of anti-CD45-saporin as conditioning agent for RAG deficiency.
Castiello MC, Bosticardo M, Sacchetti N, Calzoni E, Fontana E, Yamazaki Y, Draghici E, Corsino C, Bortolomai I, Sereni L, Yu HH, Uva P, Palchaudhuri R, Scadden DT, Villa A, Notarangelo LD (2021) Efficacy and safety of anti-CD45-saporin as conditioning agent for RAG deficiency. J Allergy Clin Immunol 147(1):309-320.e6. doi: 10.1016/j.jaci.2020.04.033
Objective: To improve multi-lineage engraftment using non-genotoxic conditioning with Anti-CD45-Saporin.
Summary: Conditioning with Anti-CD45 antibody-drug conjugates may represent a novel and safe conditioning regimen for patients with RAG deficiency and other inborn errors of immunity.
Usage: Intravenous injection of Anti-CD45-SAP (3 mg/kg).
Related Products: Streptavidin-ZAP (Cat. #IT-27)
Pseudomonas Exotoxin A based toxins targeting epidermal growth factor receptor for the treatment of prostate cancer
Fischer A, Wolf I, Fuchs H, Masilamani AP, Wolf P (2020) Pseudomonas Exotoxin A based toxins targeting epidermal growth factor receptor for the treatment of prostate cancer. Toxins (Basel) 12(12):753. doi: 10.3390/toxins12120753
Summary: Refers to chimeric murine-human mAb cetuximab bound to Streptavidin-ZAP.
Related Products: Streptavidin-ZAP (Cat. #IT-27)
Antibody-drug conjugates targeting CD45 plus Janus kinase inhibitors effectively condition for allogeneic hematopoietic stem cell transplantation
Persaud SP, Ritchey JK, Choi J, Ruminski PG, Cooper ML, Rettig MP, DiPersio JF (2020) Antibody-drug conjugates targeting CD45 plus Janus kinase inhibitors effectively condition for allogeneic hematopoietic stem cell transplantation. bioRxiv 2020.10.02.324475. doi: 10.1101/2020.10.02.324475
Related Products: Streptavidin-ZAP (Cat. #IT-27), Blank-Streptavidin-SAP (Cat. #IT-27B)
ALPPL2 is a highly specific and targetable tumor cell surface antigen
Su Y, Zhang X, Bidlingmaier S, Behrens CR, Lee NK, Liu B (2020) ALPPL2 is a highly specific and targetable tumor cell surface antigen. Cancer Res 80(20):4552-4564. doi: 10.1158/0008-5472.CAN-20-1418 PMID: 32868383
Objective: To evaluate therapeutic potential of ALPPL2 targeting.
Summary: Exquisite tissue specificity and broad tumor type coverage suggest that ALPPL2 could be an excellent cell surface target for therapeutic development against mesothelioma.
Usage: Biotinylated M25 IgG1 and Streptavidin-ZAP were mixed at a molar ratio of 1:1.
Related Products: Streptavidin-ZAP (Cat. #IT-27)
Chlorotoxin conjugated with saporin reduces viability of ML-1 thyroid cancer cells in vitro.
Rizvanovic H, Pinheiro AD, Kim K, Thomas J (2019) Chlorotoxin conjugated with saporin reduces viability of ML-1 thyroid cancer cells in vitro. bioRxiv 885483. doi: 10.1101/2019.12.20.885483
Objective: To determine whether Chlorotoxin-conjugated Saporin (CTX-SAP) would inhibit the growth of aggressive thyroid cancer cell lines expressing MMP-2.
Summary: This in vitro study demonstrated the efficacy of a CTX-SAP conjugate in reducing the viability of ML-1 thyroid cancer cells in a dose dependent manner.
Usage: There was a statistically significant reduction in cell viability with increasing concentrations of the CTX-SAP conjugate (biotinylated Chlorotoxin mixed with Streptavidin-ZAP). The cell viability of ML-1 cells was decreased by 49.77% with the treatment of 600 nM of CTX-SAP (F=44.24). Unconjugated Chlorotoxin or Saporin had no significant effect on cell viability a using similar assay.
Related Products: Streptavidin-ZAP (Cat. #IT-27), , Saporin (Cat. #PR-01), Chlorotoxin-SAP (Cat. #IT-86)
Nongenotoxic antibody-drug conjugate conditioning enables safe and effective platelet gene therapy of hemophilia A mice.
Gao C, Schroeder JA, Xue F, Jing W, Cai Y, Scheck A, Subramaniam S, Rao S, Weiler H, Czechowicz A, Shi Q (2019) Nongenotoxic antibody-drug conjugate conditioning enables safe and effective platelet gene therapy of hemophilia A mice. Blood Adv 3(18):2700-2711. doi: 10.1182/bloodadvances.2019000516
Objective: To determine whether hematopoietic cell–targeted ADC preconditioning is effective for engraftments that are genetically manipulated by 2bF8 lentivirus (2bF8LV) and whether sustained therapeutic platelet FVIII expression is attainable in platelet-specific gene therapy utilizing ADC-based preconditioning.
Summary: The authors describe targeted nongenotoxic preconditioning for 2bF8 gene therapy utilizing a hematopoietic cell–specific antibody-drug conjugate (ADC), which consists of saporin conjugated to CD45.2- and CD117-targeting antibodies.
Usage: ADCs were prepared by combining biotinylated antibody with Streptavidin-ZAP. The combination of CD45.2-ADC (3 mg/kg) plus CD117-ADC (0.5 mg/kg), with or without additional CD4-ADC (0.5 mg/kg) or CD8-ADC (0.5 mg/kg), was administered IV to 5- to 6-week-old FVIIInull/CD45.2 recipients 2 days before transplantation.
Related Products: Streptavidin-ZAP (Cat. #IT-27)