Malignancy and lymphoid proliferation in primary immune deficiencies; hard to define, hard to treat

2019 ◽  
Vol 67 (2) ◽  
Author(s):  
Ayca Kiykim ◽  
Nursah Eker ◽  
Ozlem Surekli ◽  
Ercan Nain ◽  
Nurhan Kasap ◽  
...  
Keyword(s):  
Immune Deficiencies ◽  
Lymphoid Proliferation

Analysis of the practicing physicians' awareness about primary immune deficiencies

2018 ◽  
pp. 21-25
Author(s):  
M. Kinash ◽  
◽  
O. Boyarchuk ◽  
O. Levandovich-Ushinskaya ◽  
N. Haliyash ◽  
...  
Keyword(s):  
Immune Deficiencies ◽  
Practicing Physicians

Case Commentary: Severe Combined Immune Deficiencies

Authorea ◽  
2017 ◽  
Author(s):  
Daniel Wells
Keyword(s):  
Immune Deficiencies

Severe combined immune deficiencies due to defects of the common ? chain-JAK3 signaling pathway

1998 ◽  
Vol 19 (4) ◽  
pp. 401-415 ◽  
Author(s):  
Fabio Candotti ◽  
John J. O'Shea ◽  
Anna Villa
Keyword(s):  
Signaling Pathway ◽  
Immune Deficiencies ◽  
The Common

scholarly journals CXCR4 in Central and Peripheral Lymphoid Niches – Physiology, Pathology and Therapeutic Perspectives in Immune Deficiencies and Malignancies

10.5772/58699 ◽  
2014 ◽  
Author(s):  
Christelle Freitas ◽  
Alexandre Bignon ◽  
Karl Balabanian ◽  
Ali Dalloul
Keyword(s):  
Immune Deficiencies

scholarly journals Preclinical Development of Autologous Hematopoietic Stem Cell-Based Gene Therapy for Immune Deficiencies: A Journey from Mouse Cage to Bed Side

Pharmaceutics ◽  
2020 ◽  
Vol 12 (6) ◽  
pp. 549
Author(s):  
Laura Garcia-Perez ◽  
Anita Ordas ◽  
Kirsten Canté-Barrett ◽  
Pauline Meij ◽  
Karin Pike-Overzet ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Severe Combined Immunodeficiency ◽  
Good Manufacturing Practice ◽  
Proof Of Concept ◽  
Preclinical Development ◽  
Hematopoietic Stem ◽  
Immune Deficiencies ◽  
Suitable Vector

Recent clinical trials using patient’s own corrected hematopoietic stem cells (HSCs), such as for primary immunodeficiencies (Adenosine deaminase (ADA) deficiency, X-linked Severe Combined Immunodeficiency (SCID), X-linked chronic granulomatous disease (CGD), Wiskott–Aldrich Syndrome (WAS)), have yielded promising results in the clinic; endorsing gene therapy to become standard therapy for a number of diseases. However, the journey to achieve such a successful therapy is not easy, and several challenges have to be overcome. In this review, we will address several different challenges in the development of gene therapy for immune deficiencies using our own experience with Recombinase-activating gene 1 (RAG1) SCID as an example. We will discuss product development (targeting of the therapeutic cells and choice of a suitable vector and delivery method), the proof-of-concept (in vitro and in vivo efficacy, toxicology, and safety), and the final release steps to the clinic (scaling up, good manufacturing practice (GMP) procedures/protocols and regulatory hurdles).

Flow Cytometry as a Diagnostic Tool in Primary and Secondary Immune Deficiencies

2019 ◽  
Vol 39 (4) ◽  
pp. 591-607 ◽  
Author(s):  
Jocelyn R. Farmer ◽  
Michelle DeLelys
Keyword(s):  
Flow Cytometry ◽  
Diagnostic Tool ◽  
Immune Deficiencies

scholarly journals Experience with a Reduced Toxicity Busulfan, Fludarabine Regimen in Children Undergoing Allogeneic HCT for Non-CGD Primary Immune Deficiencies Requiring Myeloablation

2020 ◽  
Vol 26 (3) ◽  
pp. S214-S215
Author(s):  
Sharat Chandra ◽  
Shanmuganathan Chandrakasan ◽  
Jack J. Bleesing ◽  
Michael B. Jordan ◽  
Ashish Kumar ◽  
...  
Keyword(s):  
Allogeneic Hct ◽  
Immune Deficiencies ◽  
Reduced Toxicity

VEGF inhibits T-cell development and may contribute to tumor-induced immune suppression

Blood ◽  
2003 ◽  
Vol 101 (12) ◽  
pp. 4878-4886 ◽  
Author(s):  
Joyce E. Ohm ◽  
Dmitry I. Gabrilovich ◽  
Gregory D. Sempowski ◽  
Ekaterina Kisseleva ◽  
Kelly S. Parman ◽  
...  
Keyword(s):  
Bone Marrow ◽  
T Cell ◽  
Cancer Patients ◽  
Thymocyte Development ◽  
Thymic Atrophy ◽  
Fetal Thymic Organ Culture ◽  
Immune Deficiencies ◽  
Thymocyte Apoptosis ◽  
Advanced Stage Cancer ◽  
Endothelial Growth Factor

AbstractT-cell defects and premature thymic atrophy occur in cancer patients and tumor-bearing animals. We demonstrate that exposure of mice to recombinant vascular endothelial growth factor (VEGF) at concentrations similar to those observed in advanced stage cancer patients reproduces this profound thymic atrophy and is highlighted by a dramatic reduction in CD4+/CD8+ thymocytes. We find that VEGF does not induce thymocyte apoptosis, but instead rapidly decreases the number of the earliest observable progenitors in the thymus. VEGF does not inhibit thymocyte development in fetal thymic organ culture, further suggesting a prethymic effect. We also demonstrate that bone marrow progenitors from animals infused with recombinant VEGF and transferred to irradiated untreated animals recolonize the thymus more efficiently than progenitors from control animals. This suggests that VEGF exposure is associated with an increased population of thymus-committed progenitors in the bone marrow. We hypothesize that pathophysiologically relevant concentrations of VEGF may block the differentiation and/or emigration of these progenitors resulting in the observed thymic atrophy. Removal of VEGF via cessation of infusion or adoptive transfer of progenitors to a congenic host induces a preferential commitment of lymphoid progenitors to the T lineage and results in a restoration of the normal composition and cellularity of the thymus. These data demonstrate that at pathophysiologic concentrations, VEGF interferes with the development of T cells from early hematopoetic progenitor cells and this may contribute to tumor-associated immune deficiencies.

scholarly journals Primary immune deficiencies with defects in neutrophil function

Hematology ◽  
2016 ◽  
Vol 2016 (1) ◽  
pp. 43-50 ◽  
Author(s):  
Mary C. Dinauer
Keyword(s):  
Fungal Infections ◽  
Leukocyte Adhesion ◽  
Genetic Disorders ◽  
Clinical Manifestations ◽  
Treatment Strategies ◽  
Adaptor Protein ◽  
Invasive Fungal Disease ◽  
Neutrophil Function ◽  
Microbial Pathogens ◽  
Immune Deficiencies

Abstract Immune deficiencies resulting from inherited defects in neutrophil function have revealed important features of the innate immune response. Although sharing an increased susceptibility to bacterial and fungal infections, these disorders each have distinctive features in their clinical manifestations and characteristic microbial pathogens. This review provides an update on several genetic disorders with impaired neutrophil function, their pathogenesis, and treatment strategies. These include chronic granulomatous disease, which results from inactivating mutations in the superoxide-generating nicotinamide dinucleotide phosphate oxidase. Superoxide-derived oxidants play an important role in the control of certain bacterial and fungal species, and also contribute to the regulation of inflammation. Also briefly summarized are updates on leukocyte adhesion deficiency, including the severe periodontal disease characteristic of this disorder, and a new immune deficiency associated with defects in caspase recruitment domain–containing protein 9, an adaptor protein that regulates signaling in neutrophils and other myeloid cells, leading to invasive fungal disease.

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