Search
4 articles for “hematopoietic stem cell transplantation”
-
Wiskott-Aldrich Syndrome: A Comprehensive Guide
Abstract: Wiskott-Aldrich Syndrome (WAS) is an uncommon genetic disorder inherited through the X chromosome, marked by a combination of immune system deficiencies, eczema, and low platelet counts. This syndrome primarily affects males, leading to significant morbidity and mortality due to recurrent infections, bleeding complications, and autoimmune diseases. Mutations in the WAS gene disrupt the production of the Wiskott-Aldrich Syndrome protein (WASp), crucial for the functioning of immune cells and platelet formation. …
Published in Research and Reviews : A Journal of Immunology · Vol. 14, Issue 2, 2024 · pp. 39–46 Read article
-
Prime Editing and Base Editing in Human Hematopoietic Stem Cells Toward Scarless Correction of Monogenic Blood Disorders
Abstract: Monogenic hematological disorders – such as sickle cell disease (SCD), β-thalassemia, and X-linked chronic granulomatous disease (X-CGD) – affect >400, 000 newborns worldwide each year, with a considerable burden in low-resource settings . Although donor-derived allogeneic hematopoietic stem-cell transplantation (HSCT) is widely regarded as curative, its application is limited due to issues of donor availability and graft-versus-host disease (GVHD) and conditioning-related toxicity. DSB-based conventional CRISPR-Cas9 strategies are limited by inefficient …
Published in International Journal of Genetic Modifications and Recombinations · Vol. 4, Issue 1, 2026 Read article
-
An Overview of Fanconi Anemia: A Genetic Disorder
Abstract: Fanconi Anemia (FA) is a rare genetic disorder caused by DNA repair defects due to pathogenic variants in one of 21 genes. It is the primary cause of inherited bone marrow failure, marked by pancytopenia. Clinically, Fanconi Anemia (FA) is associated with congenital malformations in multiple systems, resulting in progressive bone marrow failure and heightened cancer risk, especially in the urogenital region and the head and neck. There are approximately …
Published in International Journal of Oncological Nursing and Practices · Vol. 2, Issue 2, 2024 · pp. 19–25 Read article
-
Revolutionizing Blood Cancer Treatment: Emerging Therapies and Future Perspectives
Abstract: Blood cancer inclusive of leukemia, lymphoma and multiple myeloma poses a significant threat in the world today because of its multifactorial process and its non-amenable to standard therapies. Some of such approaches are immunotherapy, gene-editing technology and targeted small molecule inhibitors that have altered the nature of treatments. CAR T cell therapy is a molecular targeted therapy for cancers and several clinical studies have reported a huge success. Also, the …
Published in International Journal of Biomedical Innovations and Engineering · Vol. 3, Issue 2, 2025 Read article