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Stem Cells for Cell-Based Therapies
The world of stem cells We know the human body comprises many cell types (e.g., blood cells, skin cells, cervical cells), but we often forget to appreciate that all of these different cell types arose from a single cell—the fertilized egg. A host of sequential, awe-inspiring events occur between the fertilization of an egg and the formation of a new individual: Embryonic stem (ES) cells are also called totipotent cells. The first steps involve making more cells by simple cell division: one cell becomes two cells; two cells become four cells, etc. Each cell of early development is undifferentiated; that is, it is...
Source: ActionBioscience - December 28, 2012 Category: Science Authors: Ali Hochberg Source Type: news

Hydroxycarbamide versus chronic transfusion for maintenance of transcranial doppler flow velocities in children with sickle cell anaemia—TCD With Transfusions Changing to Hydroxyurea (TWiTCH): a multicentre, open-label, phase 3, non-inferiority trial
This study is registered with ClinicalTrials.gov, number NCT01425307. Findings Between Sept 20, 2011, and April 17, 2013, 159 patients consented and enrolled in TWiTCH. 121 participants passed screening and were then randomly assigned to treatment (61 to transfusions and 60 to hydroxycarbamide). At the first scheduled interim analysis, non-inferiority was shown and the sponsor terminated the study. Final model-based TCD velocities were 143 cm/s (95% CI 140–146) in children who received standard transfusions and 138 cm/s (135–142) in those who received hydroxycarbamide, with a difference of 4·54 (0·10–8·98). Non-i...
Source: The Lancet - December 7, 2015 Category: Journals (General) Source Type: research

Probability of TCD-Normalization in the "Drepagreffe" Trial Comparing Transplantation to Chronic Transfusion in Sickle Cell Anemia Children with Abnormal-Transcranial Doppler Is Associated with Lower Ang-2 and BDNF Plasma Levels
This study confirms the association between high levels of BDNF and high velocities, and suggests that transplantation increases the likelihood of TCD-normalization compared to transfusion, and is associated with reduced Ang-2 expression in plasma, which may reflect improved brain oxygenation.DisclosuresThuret: Addmedica: Research Funding; bluebird bio: Research Funding; Novartis: Research Funding. Pondarré: Addmedica: Membership on an entity's Board of Directors or advisory committees; Novartis: Honoraria; Blue Bird Bio: Honoraria. Bernaudin: AddMedica: Honoraria; Pierre fabre: Research Funding; Cordons de Vie: Res...
Source: Blood - November 21, 2018 Category: Hematology Authors: Bernaudin, M., Khelif, Y., Chadebech, P., Bodivit, G., Jouard, A., Verlhac, S., Paillard, C., Thuret, I., Guitton, C., Runel, C., Missud, F., Arnaud, C., Kamdem, A., Pondarre, C., Pirenne, F., Bernaudin, F. Tags: 114. Hemoglobinopathies, Excluding Thalassemia-Clinical: Novel or Improved Approaches To Treating Sickle Cell Disease Source Type: research

Current Results of Lentiglobin Gene Therapy in Patients with Severe Sickle Cell Disease Treated Under a Refined Protocol in the Phase 1 Hgb-206 Study
Backgroundβ-globin gene transfer has the potential for substantial clinical benefit in patients with sickle cell disease (SCD). LentiGlobin Drug Product (DP) contains autologous CD34+ hematopoietic stem cells (HSCs) transduced with the BB305 lentiviral vector (LVV), encoding β-globin with an anti-sickling substitution (T87Q). The safety and efficacy of LentiGlobin gene therapy is being evaluated in the ongoing Phase 1 HGB-206 study (NCT02140554). Results in the initial 7 patients treated with LentiGlobin DP from steady state bone marrow harvested (BMH) HSCs using original DP manufacturing process (Group A) demons...
Source: Blood - November 21, 2018 Category: Hematology Authors: Tisdale, J. F., Kanter, J., Mapara, M. Y., Kwiatkowski, J. L., Krishnamurti, L., Schmidt, M., Miller, A. L., Pierciey, F. J., Shi, W., Ribeil, J.-A., Asmal, M., Thompson, A. A., Walters, M. C. Tags: 801. Gene Therapy and Transfer: Gene Therapy for Blood Cell Disorders Source Type: research

Regulatory Genetic Variation at the S100B Gene Associates with Vaso-Occlusive Manifestations in Sickle Cell Disease
In sickle cell disease (SCD) polymerization of hemoglobin S under deoxygenated conditions causes vaso-occlusion, which can manifest as acute pain crisis and progressive bone/organ damage. Molecular studies have attributed vaso-occlusion to elevated vascular adhesion and inflammatory responses, whereas the genetic regulation has only recently been assessed.Genomic DNA isolated from peripheral blood mononuclear cells (PBMCs) was hybridized to Illumina Human 610-Quad SNP array for the PUSH and Walk-PHaSST cohorts and to Affrymetrix SNP 6.0 array for the Howard SCD expression cohort. Single nucleotide polymorphisms (SNPs) for ...
Source: Blood - November 21, 2018 Category: Hematology Authors: Zhang, X., Zhang, W., Saraf, S. L., Nekhai, S., Gladwin, M. T., Machado, R., Gordeuk, V. R. Tags: 113. Hemoglobinopathies, Excluding Thalassemia-Basic and Translational Science: Poster I Source Type: research

Reducing Health Care Disparities in Sickle Cell Disease: A Review.
Authors: Lee L, Smith-Whitley K, Banks S, Puckrein G Abstract Sickle cell disease (SCD) is an inherited blood disorder most common among African American and Hispanic American persons. The disease can cause substantial, long-term, and costly health problems, including infections, stroke, and kidney failure, many of which can reduce life expectancy. Disparities in receiving health care among African Americans and other racial/ethnic minority groups in the United States are well known and directly related to poor outcomes associated with SCD. As an orphan disease-one that affects <200 000 persons nationwide-SCD ...
Source: Public Health Reports - October 11, 2019 Category: International Medicine & Public Health Tags: Public Health Rep Source Type: research