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020 _a1493972995
_q(electronic bk.)
020 _a9781493972999
_q(electronic bk.)
020 _z9781493972975
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050 4 _aRC641.7.H35
_b2017 EB
066 _c(S
245 0 0 _aGene and cell therapies for beta-globinopathies
_cPunam Malik, John Tisdale, editors.
264 1 _aNew York, NY
_bSpringer International Publishing New York
_c2017
300 _a1 recurso en línea (254 páginas)
336 _aTexto
_btxt
_2rdacontent
337 _aelectrónico
_bc
_2rdamedia
338 _arecurso electrónico
_bcr
_2rdacarrier
347 _atext file
_bPDF
490 0 _aAdvances in experimental medicine and biology
_vv. 1013
490 0 _aAmerican Society of Gene & Cell Therapy
500 _a"" Medical and Surgical Management of Patients with Thalassemia""
500 _aIncludes index.
500 _a
505 0 _a""Preface""; ""Contents""; ""Chapter 1: Clinical Features of β-Thalassemia and Sickle Cell Disease""; "" Introduction""; "" Hemoglobin: Structure and Function""; "" Types of Hemoglobin""; "" Classifying the Hemoglobin Disorders""; "" β-Thalassemia""; "" Overview and Historical Perspective""; "" Epidemiology and Global Burden""; "" Molecular Basis and Pathophysiology""; "" Iron and β-Thalassemia""; "" Classification and Clinical Manifestations of Î-Thalassemia""; "" β-Thalassemia Major""; "" Hematological Manifestations""; "" Skeletal Manifestations""
505 8 _a"" Dominantly Inherited β-Thalassemia"""" Unusual Causes of β-Thalassemia""; "" Genetics of Sickle Cell Disease""; "" Genetic Modifiers of β-Hemoglobinopathies""; "" Update on the Genetic Control of Fetal Hemoglobin (HbF)""; "" Genetic Modifiers of Sickle Cell Disease""; "" Impact of HbF in SCD""; "" Impact of α-Thalassemia on SCD Phenotype""; "" Secondary Modifiers of Sub-phenotypes and Complications""; "" Genetic Modifiers of β-Thalassemia""; "" Effect of the Primary Modifiers: HbF Quantitative Trait Loci and α-Globin Genotype""
505 8 _a"" Endocrine Manifestations"""" Hepatic Manifestations""; "" Cardiac Manifestations""; "" β-Thalassemia Intermedia""; "" β-Thalassemia Minor""; "" Sickle Cell Disease""; "" Overview and Historical Perspective""; "" Epidemiology and Global Burden""; "" Molecular Basis and Pathophysiology""; "" Classification of Sickle Cell Disease""; "" Clinical Manifestations of Sickle Cell Disease""; "" Hematologic Manifestations""; "" Vaso-Occlusive Manifestations""; "" Infectious Manifestations""; "" Neurological Manifestations""; "" Cardiac Manifestations""; "" Pulmonary Manifestations""
505 8 _a"" Secondary Modifiers of Complications of β-Thalassemia""""References""; ""Chapter 3: Current Standards of Care and Long Term Outcomes for Thalassemia and Sickle Cell Disease""; "" Introduction""; "" Thalassemia""; "" Molecular Basis and Classification""; "" Clinical Presentation""; "" Diagnosis""; "" Preventive Care and Screening Measures (Table 3.3)""; "" Growth and Development""; "" Endocrine Studies""; "" Iron Monitoring""; "" Cardiac Studies""; "" Infection""; "" Bone Mineral Density""; "" Ophthalmologic and Auditory Screening""
505 8 _a"" Splenic Manifestations"""" Hepatobiliary Manifestations""; "" Renal Complications""; "" Skeletal Complications""; "" Other Complications""; "" Sickle Cell Disease Treatment Options""; "" Summary""; ""References""; ""Chapter 2: Genetic Basis and Genetic Modifiers of β-Thalassemia and Sickle Cell Disease""; "" Introduction""; "" The β-Globin Gene (HBB) and Normal Expression""; "" Genetics of β-Thalassemia""; "" Non-deletion β-Thalassemia""; "" Transcriptional Mutations""; "" Mutations Affecting RNA Processing""; "" Translational Mutations""; "" Deletions Causing β-Thalassemia""
520 3 _aThis book provides a comprehensive review of gene and cell therapy approaches for hemoglobinopathies. It covers the progress and current state of the field in hematopoietic stem cell transplantation for hemoglobinopathies and genetic correction of autologous hematopoietic stem cells. Chapters focus on such topics as the current standards of care and long term outcomes of b-thalassemias and sickle cell disease, the ability to generate autologous embryonic stem cell-like cells from primary somatic cells, and genetic strategies to reactivate fetal hemoglobin production. In addition to offering a thorough review of the field in its present state, this book also sheds light on the major changes expected in coming years.   Gene and Cell Therapies for Beta-Globinopathies is part of the American Society of Gene and Cell Therapy sub-series of the highly successful Advances in Experimental Medicine and Biology series. It is essential reading for graduate students, clinicians, and researchers interested in gene and cell therapy.
588 0 _aVersión impresa
988 _aEBOOK, EBSPRINGER_2017
650 7 _aHemoglobina
_2embne
_9144141
700 1 _aMalik, Punam.
700 1 _aTisdale, John.
856 4 0 _uhttps://go.openathens.net/redirector/universidadeuropea.es?url=http://link.springer.com/10.1007/978-1-4939-7299-9
_zAcceso a este recurso digital (usuarios Universidad Europea de Madrid)
880 0 _6505-00
_a1. Clinical features of β-thalassemia and sickle cell disease -- 2. Genetic basis and genetic modifiers of β-thalassemia and sickle cell disease -- 3. Current standards of care and long term outcomes for thalassemia and sickle cell disease -- 4. Allogeneic/matched related transplantation for β-thalassemia and sickle cell anemia -- 5. Alternative donor/unrelated donor transplants for the β-thalassemia and sickle cell disease -- 6. Gene addition strategies for β-thalassemia and sickle cell anemia -- 7. Reactivation of fetal hemoglobin for treating β-thalassemia and sickle cell disease -- 8. Genome editing for β-hemoglobinopathies -- 9. Gene and cell therapy for β-thalassemia and sickle cell disease with induced pluripotent stem cells (iPSCs): the next frontier -- Index.
880 3 _6246-00
_aGene and cell therapies for β-globinopathies
942 _2lcc
_cLE
998 _b02/2018
_dz
_e-
_zSI