Generation and characterization of induced pluripotent stem cell line (IGIBi001-A) from a sickle cell anemia patient with homozygous β-globin mutation

Publication date: Available online 14 June 2019Source: Stem Cell ResearchAuthor(s): Nupur Bhargava, Shashank Jaitly, Sangam Giri Goswami, Suman Jain, Debojyoti Chakraborty, Sivaprakash RamalingamAbstractSickle cell disease (SCD) is an autosomal recessive disorder caused by a mutation in β-globin (HBB) gene. We have generated an induced pluripotent stem cell (iPSC) line, IGIBi001-A from an Indian sickle cell patient with a homozygous HBB gene mutation using Sendai virus reprogramming system. Characterization of IGIBi001-A showed that these iPSCs are transgene-free and expressed pluripotent stem cell markers. They had a normal karyotype and were able to differentiate into all three germ layers. This new SCD-iPSC line will contribute to better understanding of the disease biology of sickle cell anemia and for screening of small molecule drugs.
Source: Stem Cell Research - Category: Stem Cells Source Type: research

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Publication date: Available online 25 June 2019Source: Life SciencesAuthor(s): Mehdi Derakhshani, Hossein Abbaszadeh, Ali Akbar Movassaghpour, Amir Mehdizadeh, Majid Ebrahimi-Warkiani, Mehdi YousefiAbstractHematopoietic stem cells (HSCs) are a rare cell population in adult bone marrow, mobilized peripheral blood, and umbilical cord blood possessing self-renewal and differentiation capability into a full spectrum of blood cells. Bone marrow HSC transplantation has been considered as an ideal option for certain disorders treatment including hematologic diseases, leukemia, immunodeficiency, bone marrow failure syndrome, genet...
Source: Life Sciences - Category: Biology Source Type: research
This article is protected by copyright. All rights reserved. PMID: 31231794 [PubMed - as supplied by publisher]
Source: Clinical Genetics - Category: Genetics & Stem Cells Authors: Tags: Clin Genet Source Type: research
ckman E, Poirot C, Socié G, Société Française de Greffe de Moelle et de Thérapie Cellulaire Abstract Allogeneic stem cell transplantation remains the only curative treatment for sickle-cell anemia, but the place of myeloablative conditioning remains to be defined. The aim of the present study was to analyze long-term outcomes, including chimerism, sickle-cell anemia-related events and biological data (hemoglobin, reticulocytes, HbS%), and fertility, in a French series of 234 SCA-patients younger than 30 years who received (1988-2012) a matched-sibling-donor stem cell transplantat...
Source: Haematologica - Category: Hematology Authors: Tags: Haematologica Source Type: research
Discussion This case demonstrates successful cure of pre-B-ALL complicating XLA by alloSCT with restoration of B-cell development and functional antibody response. We are aware of only one previous case of pre-B-ALL in an XLA patient (21), which suggests that human BTK deficiency in itself does not predispose to pre-B-ALL. However, there are data to suggest that BTK may act as a tumor suppressor, and BTK deficiency may predispose to tumor development following a “second hit.” Mice with a genetic deficiency in Slp65, a gene encoding an adaptor protein that functions together with BTK, have a block in progenito...
Source: Frontiers in Immunology - Category: Allergy & Immunology Source Type: research
A popular New Jersey high school principal died Sunday after donating bone marrow to a young boy in France, his family confirmed to news outlets. Derrick Nelson, who was the principal at New Jersey’s Westfield High School, never recovered after falling into a monthlong coma following a bone marrow donation procedure at a Bergen County hospital, his father told NJ.com. Nelson died Sunday at age 44, though the exact cause of his death was unclear, his father said; he told ABC News that his son may have had a heart attack. Nelson was donating bone marrow to supply stem cells to a 14-year-old boy in France with an undisc...
Source: TIME: Health - Category: Consumer Health News Authors: Tags: Uncategorized Education New Jersey onetime Source Type: news
We describe our 5-year experience in generation of hiPSC-lines from human bone marrow-derived mesenchymal stromal cells (BM-MSCs) using synthetic mRNAs encoding reprogramming factors. We generated hiPSC-lines from pediatric patients with β-Thalassemia, Sickle Cell Anemia, Blackfan-Diamond Anemia, Severe Aplastic Anemia, DOCK8 Immunodeficiency and 1 healthy control. After optimization of the reprogramming procedure, average reprogramming efficiency of BM-MSCs was 0.29% (range 0.25–0.4). The complete reprogramming process lasted 14–16 days. Three to five hiPSC-colonies per sample were selected, expanded to...
Source: Blood Cells, Molecules, and Diseases - Category: Hematology Source Type: research
We describe our 5-year experience in generation of hiPSC-lines from human bone marrow-derived mesenchymal stromal cells (BM-MSCs) using synthetic mRNAs encoding reprogramming factors. We generated hiPSC-lines from pediatric patients with β-Thalassemia, Sickle Cell Anemia, Blackfan-Diamond Anemia, Severe Aplastic Anemia, DOCK8 Immunodeficiency and 1 healthy control. After optimization of the reprogramming procedure, average reprogramming efficiency of BM-MSCs was 0.29% (range 0.25-0.4). The complete reprogramming process lasted 14-16 days. Three to five hiPSC-colonies per sample were selected, expanded to 5 culture p...
Source: Blood Cells, Molecules and Diseases - Category: Hematology Authors: Tags: Blood Cells Mol Dis Source Type: research
Matched sibling donor hematopoietic stem cell transplantation (MSD-HSCT) is a promising option for children with sickle cell anemia (SCA) requiring chronic transfusion due to elevated transcranial Doppler velocity (TCD), a study from France suggests.Reuters Health Information
Source: Medscape Medical News Headlines - Category: Consumer Health News Tags: Hematology-Oncology News Source Type: news
This nonrandomized controlled intervention study compared the effect of matched sibling donor hematopoietic stem cell transplantation (HSCT) vs standard care comprising transfusions and hydroxyurea on transcranial Doppler velocities, a measure of cerebral vasculopathy, at 1 year in children with sickle cell anemia requiring chronic transfusion.
Source: JAMA - Category: General Medicine Source Type: research
Stroke is one of the most devastating complications of sickle cell anemia (SCA), and risk of stroke starts very early in life. Before screening techniques were available, 11% of children with homozygous SCA had ischemic stroke by their 20th birthday. Although most children with SCA will have neurologic recovery after stroke, approximately one-third will have long-term neurologic sequelae, including motor impairment. Moreover, children with SCA who have had a stroke often have neurocognitive impairment and difficulties with academic performance.
Source: JAMA - Category: General Medicine Source Type: research
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