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stem cell transplant in SCD

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https://www.readbyqxmd.com/read/30533448/pre-clinical-development-of-a-lentiviral-vector-expressing-the-anti-sickling-%C3%AE-as3-globin-for-gene-therapy-for-sickle-cell-disease
#1
Valentina Poletti, Fabrizia Urbinati, Sabine Charrier, Guillaume Corre, Roger P Hollis, Beatriz Campo Fernandez, Samia Martin, Michael Rothe, Axel Schambach, Donald B Kohn, Fulvio Mavilio
Sickle cell disease (SCD) is caused by a mutation (E6V) in the hemoglobin (Hb) β-chain that induces polymerization of Hb tetramers, red blood cell deformation, ischemia, anemia, and multiple organ damage. Gene therapy is a potential alternative to human leukocyte antigen (HLA)-matched allogeneic hematopoietic stem cell transplantation, available to a minority of patients. We developed a lentiviral vector expressing a β-globin carrying three anti-sickling mutations (T87Q, G16D, and E22A) inhibiting axial and lateral contacts in the HbS polymer, under the control of the β-globin promoter and a reduced version of the β-globin locus-control region...
December 14, 2018: Molecular Therapy. Methods & Clinical Development
https://www.readbyqxmd.com/read/30140714/an-optimized-lentiviral-vector-efficiently-corrects-the-human-sickle-cell-disease-phenotype
#2
Leslie Weber, Valentina Poletti, Elisa Magrin, Chiara Antoniani, Samia Martin, Charles Bayard, Hanem Sadek, Tristan Felix, Vasco Meneghini, Michael N Antoniou, Wassim El-Nemer, Fulvio Mavilio, Marina Cavazzana, Isabelle Andre-Schmutz, Annarita Miccio
Autologous transplantation of hematopoietic stem cells transduced with a lentiviral vector (LV) expressing an anti-sickling HBB variant is a potential treatment for sickle cell disease (SCD). With a clinical trial as our ultimate goal, we generated LV constructs containing an anti-sickling HBB transgene ( HBBAS3 ), a minimal HBB promoter, and different combinations of DNase I hypersensitive sites (HSs) from the locus control region (LCR). Hematopoietic stem progenitor cells (HSPCs) from SCD patients were transduced with LVs containing either HS2 and HS3 (β-AS3) or HS2, HS3, and HS4 (β-AS3 HS4)...
September 21, 2018: Molecular Therapy. Methods & Clinical Development
https://www.readbyqxmd.com/read/30067082/the-potential-role-of-lsd1-inhibitors-in-the-treatment-of-sickle-cell-disease-a-review-of-preclinical-animal-model-data
#3
Angela Rivers, Ramasamy Jagadeeswaran, Donald Lavelle
Sickle cell disease (SCD) is caused by a mutation of the b-globin gene(21) that triggers the polymerization of deoxygenated sickle hemoglobin (HbS). Approximately 100,000 SCD patients in the U.S. and millions worldwide(53) suffer from chronic hemolytic anemia, painful crises, multisystem organ damage, and reduced life expectancy(60, 70). Hematopoietic stem cell (HSC) transplantation can be curative, but the majority of patients do not have a suitable donor (80). Advanced gene editing technologies also offer the possibility of a cure (16, 34), but the likelihood that these strategies can be mobilized to treat the large numbers of patients residing in developing countries is remote...
August 1, 2018: American Journal of Physiology. Regulatory, Integrative and Comparative Physiology
https://www.readbyqxmd.com/read/30001893/haematopoietic-stem-cell-transplantation-in-paediatric-patients-with-%C3%AE-thalassaemia-and-sickle-cell-disease-an-experience-of-the-spanish-working-group-for-bone-marrow-transplantation-in-children-getmon
#4
Laura Alonso, Marta González-Vicent, Cristina Belendez, Isabel Badell, Ana Sastre, Antonia Rodríguez-Villa, Mar Bermúdez-Cortés, Raquel Hladun, Cristina Díaz de Heredia
BACKGROUND AND OBJECTIVES: A recently occurring increase of the prevalence of haemoglobinopathies, β-thalassaemia major (TM) and sickle cell disease (SCD) over the last two decades in our country has generated new needs in terms of medical resources for both prevention and treatment of these patients. Allogeneic haematopoietic stem cell transplant (allo-HSCT) is a curative treatment available for patients who have severe haemoglobinopathies. The main objective of this study was to evaluate the results of allo-HSCT in paediatric patients with TM or SCD performed in paediatric hematopoietic transplant units within the Spanish Group of Bone Marrow Transplantation in Children (GETMON)...
July 9, 2018: Medicina Clínica
https://www.readbyqxmd.com/read/29963521/allogeneic-hematopoietic-stem-cell-transplantation-in-congenital-hemoglobinopathies-with-myeloablative-conditioning-and-rabbit-anti-thymocyte-globulin
#5
Bo-Kyoung Park, Hyo-Sup Kim, Seongkoo Kim, Jae-Wook Lee, Young Shil Park, Pil-Sang Jang, Nack-Gyun Chung, Dae-Chul Jeong, Bin Cho
Background: Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative therapy for β-thalassemia major (TM) and sickle cell disease (SCD) in children. Graft-versus-host disease (GVHD) and treatment-related mortality (TRM) remain significant challenges to improving survival after HSCT. Here, we analyzed the outcome of TM and SCD patients, who received allogeneic HSCT with myeloablative conditioning at our institution. Methods: Twenty-two patients (15 TM, 7 SCD), with a median age of 9 years (range, 1...
June 2018: Blood Research
https://www.readbyqxmd.com/read/29958563/genetic-therapies-for-sickle-cell-disease
#6
REVIEW
Erica B Esrick, Daniel E Bauer
After decades with few novel therapeutic options for sickle cell disease (SCD), autologous hematopoietic stem cell (HSC) based genetic therapies including lentiviral gene therapy (GT), and genome editing (GE) now appear imminent. Lentiviral GT has advanced considerably in the past decade with promising clinical trial results in multiple disorders. For β-hemoglobinopathies, GT strategies of gene addition and fetal hemoglobin induction through BCL11A regulation are both being evaluated in open clinical trials...
April 2018: Seminars in Hematology
https://www.readbyqxmd.com/read/29883237/stem-cell-transplantation-in-sickle-cell-disease-therapeutic-potential-and-challenges-faced
#7
Alexis Leonard, John F Tisdale
Sickle cell disease (SCD) is the most common inherited hemoglobinopathy worldwide, and is a life-limiting disease with limited therapeutic options to reduce disease severity. Despite being a monogenic disorder, the clinical phenotypes of SCD are variable, with few reliable predictors of disease severity easily identifying patients where the benefits of curative therapy outweigh the risks. Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative option, though significant advances in gene therapy raise the promise for additional curative methods...
July 2018: Expert Review of Hematology
https://www.readbyqxmd.com/read/29876866/definitive-erythropoiesis-from-pluripotent-stem-cells-recent-advances-and-perspectives
#8
Selami Demirci, John F Tisdale
Derivation of functional and mature red blood cells (RBCs) with adult globin expression from renewable source such as induced pluripotent stem cells (iPSCs) is of importance from the clinical point of view. Definitive RBC generation can only be succeeded through production of true hematopoietic stem cells (HSCs). There has been a great effort to obtain definitive engraftable HSCs from iPSCs but the results were mostly unsatisfactory due to low, short-term and linage-biased engraftment in mouse models. Moreover, ex vivo differentiation approaches ended up with RBCs with mostly embryonic and fetal globin expression...
June 7, 2018: Advances in Experimental Medicine and Biology
https://www.readbyqxmd.com/read/29859773/gene-therapy-for-sickle-cell-disease-an-update
#9
REVIEW
Selami Demirci, Naoya Uchida, John F Tisdale
Sickle cell disease (SCD) is one of the most common life-threatening monogenic diseases affecting millions of people worldwide. Allogenic hematopietic stem cell transplantation is the only known cure for the disease with high success rates, but the limited availability of matched sibling donors and the high risk of transplantation-related side effects force the scientific community to envision additional therapies. Ex vivo gene therapy through globin gene addition has been investigated extensively and is currently being tested in clinical trials that have begun reporting encouraging data...
July 2018: Cytotherapy
https://www.readbyqxmd.com/read/29807062/hemoglobin-disorders-lentiviral-gene-therapy-in-the-starting-blocks-to-enter-clinical-practice
#10
Karine Sii-Felice, Marie Giorgi, Philippe Leboulch, Emmanuel Payen
The β-hemoglobinopathies, transfusion-dependent β-thalassemia and sickle cell disease, are the most prevalent inherited disorders worldwide and affect millions of people. Many of these patients have a shortened life expectancy and suffer from severe morbidity despite supportive therapies, which impose an enormous financial burden to societies. The only available curative therapy is allogeneic hematopoietic stem cell transplantation, although most patients do not have an HLA-matched sibling donor, and those who do still risk life-threatening complications...
August 2018: Experimental Hematology
https://www.readbyqxmd.com/read/29728697/hematopoietic-stem-cell-transplantation-for-adult-sickle-cell-disease-in-the-era-of-universal-donor-availibility
#11
Hafiz Muhammad Aslam, Said Yousuf, Adetola Kassim, Shumaila Muhammad Iqbal, Shahrukh K Hashmi
Current projections estimate that the number of newborns with sickle cell disease (SCD) globally will exceed 400,000 by 2050. Over the last three decades, increased newborn screening, supportive care, and use of hydroxyurea therapy, have decreased early childhood mortality among individuals affected with SCD. Despite hematopoietic cell transplantation (HCT) being curative in SCD, its impact on disease free survival remains unknown, especially in adults, partly due to previous limitations in donor options and perceived mortality in adults using myeloablative conditioning...
May 4, 2018: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/29684562/bone-marrow-transplantation-after-nonmyeloablative-treosulfan-conditioning-is-curative-in-a-murine-model-of-sickle-cell-disease
#12
Divya Devadasan, Chiao-Wang Sun, Erik R Westin, Li-Chen Wu, Kevin M Pawlik, Tim M Townes, Frederick D Goldman
Allogeneic hematopoietic stem cell transplantation (HSCT) can be curative for patients with sickle cell disease (SCD). However, morbidity associated with myeloablative conditioning and graft-versus-host disease has limited its utility. To this end, autologous HSCT for SCD using lentiviral gene-modified bone marrow (BM) or peripheral blood stem cells has been undertaken, although toxicities of fully ablative conditioning with busulfan and incomplete engraftment have been encountered. Treosulfan, a busulfan analog with a low extramedullary toxicity profile, has been used successfully as part of a myeloablative conditioning regimen in the allogeneic setting in SCD...
August 2018: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/29656137/haploidentical-peripheral-blood-stem-cell-transplantation-demonstrates-stable-engraftment-in-adults-with-sickle-cell-disease
#13
Santosh L Saraf, Annie L Oh, Pritesh R Patel, Karen Sweiss, Matthew Koshy, Sally Campbell-Lee, Michel Gowhari, Shivi Jain, David Peace, John G Quigley, Irum Khan, Robert E Molokie, Nadim Mahmud, Victor R Gordeuk, Damiano Rondelli
We report on the screening and development of haploidentical hematopoietic stem cell transplantation (HSCT) for adult patients with clinically aggressive sickle cell disease (SCD) at our institution. Of 50 adult SCD patients referred for HSCT between January 2014 and March 2017, 20% were denied by insurance. Of 41 patients initially screened, 10% lacked an available haploidentical donor, 29% had elevated donor-specific antibodies (DSAs), and 34% declined to proceed to HSCT. All 10 patients who were transplanted received peripheral blood stem cells...
August 2018: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/29653206/late-effects-screening-guidelines-after-hematopoietic-cell-transplantation-hct-for-hemoglobinopathy-consensus-statement-from-the-second-pediatric-blood-and-marrow-transplant-consortium-international-conference-on-late-effects-after-pediatric-hct
#14
Shalini Shenoy, Javid Gaziev, Emanuele Angelucci, Allison King, Monica Bhatia, Angela Smith, Dorine Bresters, Anne E Haight, Christine N Duncan, Josu de la Fuente, Andrew C Dietz, K Scott Baker, Michael A Pulsipher, Mark C Walters
Allogeneic hematopoietic cell transplantation (HCT) can halt organ damage and eliminate symptoms in hemoglobin disorders, including sickle cell disease (SCD) and thalassemia major. Managing the residual manifestations of pre-HCT disease complications and the long-term effects of HCT requires systematic monitoring, follow-up and intervention when indicated. Late complications vary with age and disease status at HCT and with transplant variables such as preparative regimen, donor source and compatibility, and immune reconstitution...
July 2018: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/29624126/health-related-quality-of-life-and-personal-life-goals-of-adults-with-sickle-cell-disease-after-hematopoietic-stem-cell-transplantation
#15
Agatha M Gallo, Crystal Patil, Tokunbo Adeniyi, Lewis L Hsu, Damiano Rondelli, Santosh Saraf
Allogeneic hematopoietic stem cell transplantation (HSCT) from a human leukocyte antigen (HLA)-matched sibling donor offers a unique therapy to reverse SCD. This mixed-methods study explores recipients' perception of HSCT success, personal life goals, and associated health-related quality of life (HRQOL) more than 1 year after HSCT. Recipients completed the Short Form-36, version 1 (SF-36v1) HRQOL survey followed by a 60- to 90-min face-to-face or telephone audio-recorded interview. Eleven of 15 eligible recipients participated in the study...
April 1, 2018: Western Journal of Nursing Research
https://www.readbyqxmd.com/read/29614637/inflammation-in-sickle-cell-disease
#16
Nicola Conran, John D Belcher
The primary β-globin gene mutation that causes sickle cell disease (SCD) has significant pathophysiological consequences that result in hemolytic events and the induction of the inflammatory processes that ultimately lead to vaso-occlusion. In addition to their role in the initiation of the acute painful vaso-occlusive episodes that are characteristic of SCD, inflammatory processes are also key components of many of the complications of the disease including autosplenectomy, acute chest syndrome, pulmonary hypertension, leg ulcers, nephropathy and stroke...
2018: Clinical Hemorheology and Microcirculation
https://www.readbyqxmd.com/read/29542687/sickle-cell-disease
#17
REVIEW
Gregory J Kato, Frédéric B Piel, Clarice D Reid, Marilyn H Gaston, Kwaku Ohene-Frempong, Lakshmanan Krishnamurti, Wally R Smith, Julie A Panepinto, David J Weatherall, Fernando F Costa, Elliott P Vichinsky
Sickle cell disease (SCD) is a group of inherited disorders caused by mutations in HBB, which encodes haemoglobin subunit β. The incidence is estimated to be between 300,000 and 400,000 neonates globally each year, the majority in sub-Saharan Africa. Haemoglobin molecules that include mutant sickle β-globin subunits can polymerize; erythrocytes that contain mostly haemoglobin polymers assume a sickled form and are prone to haemolysis. Other pathophysiological mechanisms that contribute to the SCD phenotype are vaso-occlusion and activation of the immune system...
March 15, 2018: Nature Reviews. Disease Primers
https://www.readbyqxmd.com/read/29535106/toward-dual-hematopoietic-stem-cell-transplantation-and-solid-organ-transplantation-for-sickle-cell-disease
#18
REVIEW
Hitomi Hosoya, Jeffrey Levine, Peter Abt, David Henry, David L Porter, Saar Gill
Sickle-cell disease (SCD) leads to recurrent vaso-occlusive crises, chronic end-organ damage, and resultant physical, psychological, and social disabilities. Although hematopoietic stem-cell transplantation (HSCT) is potentially curative for SCD, this procedure is associated with well-recognized morbidity and mortality and thus is ideally offered only to patients at high risk of significant complications. However, it is difficult to identify patients at high risk before significant complications have occurred, and once patients experience significant organ damage, they are considered poor candidates for HSCT...
March 13, 2018: Blood Advances
https://www.readbyqxmd.com/read/29419693/organ-damage-mitigation-with-the-baskent-sickle-cell-medical-care-development-program-bascare
#19
Hakan Ozdogu, Can Boga, Suheyl Asma, Ilknur Kozanoglu, Cigdem Gereklioglu, Mahmut Yeral, Nurhilal Turgut Buyukkurt, Soner Solmaz, Aslı Korur, Pelin Aytan, Erkan Maytalman, Mutlu Kasar
The Eastern Mediterranean is among the regions where sickle cell disease (SCD) is common. The morbidity and mortality of this disease can be postponed to adulthood through therapies implemented in childhood. The present study focuses on the organ damage-reducing effects of the Baskent Sickle Cell Medical Care Development Program (BASCARE), which was developed by a team who lives in this region and has approximately 25 years of experience. The deliverables of the program included the development of an electronic health recording system (PRANA) and electronic vaccination system; the use of low citrate infusion in routine prophylactic automatic erythrocyte exchange (ARCE) programs including pregnant women; the use of leukocyte-filtered and irradiated blood for transfusion; the use of magnetic resonance imaging methods (T2) for the management of transfusion-related hemosiderosis; and the implementation of an allogeneic hematopoietic stem cell transplantation protocol for adult patients...
February 2018: Medicine (Baltimore)
https://www.readbyqxmd.com/read/29403023/allogenic-peripheral-stem-cell-transplantation-from-hla-matched-related-donors-for-adult-sickle-cell-disease-remarkable-outcomes-from-a-single-center-trial
#20
H Ozdogu, C Boga, M Yeral, I Kozanoglu, C Gereklioglu, P Aytan, M Kasar, S Asma, N Buyukkurt, S Solmaz, A Korur, C Sariturk
Adult patients with sickle cell disease (SCD) are highly susceptible to stem cell transplant complications, including drug toxicity, graft versus host disease (GVHD), and graft rejection due to SCD-related tissue damage, endothelial activation, and inflammation. The scarcity of compatible stem cells for transplantation further limits treatment options, with only 43 cases of adult allogeneic peripheral blood stem cell transplantation (allo-PSCT) from human leukocyte antigen (HLA)-identical sibling donors reported in the international registry for the period 1986-2013...
July 2018: Bone Marrow Transplantation
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