about
Treating sickle cell disease by targeting HbS polymerizationFunction and safety of lentivirus-mediated gene transfer for CSF2RA-deficiency.Scalable Lentiviral Vector Production Using Stable HEK293SF Producer Cell Lines.Bone Marrow as a Hematopoietic Stem Cell Source for Gene Therapy in Sickle Cell Disease: Evidence from Rhesus and SCD Patients.Gene Therapy for β-Hemoglobinopathies.Molecular basis of β thalassemia and potential therapeutic targets.A Guide to Approaching Regulatory Considerations for Lentiviral-Mediated Gene Therapies.Sickle cell disease: tipping the balance of genomic research to catalyse discoveries in Africa.Eliminating HIV-1 Packaging Sequences from Lentiviral Vector Proviruses Enhances Safety and Expedites Gene Transfer for Gene Therapy.Therapeutic advances in sickle cell disease in the last decade.Rounding up sickle cells with gene therapy.The changing landscape of gene editing in hematopoietic stem cells: a step towards Cas9 clinical translation.Fetal haemoglobin induction in sickle cell disease.Ex Vivo Selection of Transduced Hematopoietic Stem Cells for Gene Therapy of β-Hemoglobinopathies.Evolution of hemoglobin loci and their regulatory elements.A long noncoding RNA from the HBS1L-MYB intergenic region on chr6q23 regulates human fetal hemoglobin expression.Pathways to pulmonary hypertension in sickle cell disease: the search for prevention and early intervention.Personalized Therapeutics and Pharmacogenomics: Integral to Personalized Health Care.Sickle cell disease: a malady beyond a hemoglobin defect in cerebrovascular disease.Emerging Therapies.Safety and efficacy of plerixafor dose escalation for the mobilization of CD34+ hematopoietic progenitor cells in patients with sickle cell disease: interim results.Plerixafor enables the safe, rapid, efficient mobilization of haematopoietic stem cells in sickle cell disease patients after exchange transfusion.Managing cell and human identity.Haploidentical Peripheral Blood Stem Cell Transplantation Demonstrates Stable Engraftment in Adults with Sickle Cell Disease.Gene Therapy With Regulatory T Cells: A Beneficial Alliance.How I safely transfuse patients with sickle cell disease and manage delayed hemolytic transfusion reactions.Oncostatin M and Kit-Ligand Control Hematopoietic Stem Cell Fate during Zebrafish Embryogenesis.Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined Immunodeficiency.Priming Human Repopulating Hematopoietic Stem and Progenitor Cells for Cas9/sgRNA Gene Targeting.Erythropoiesis: insights into pathophysiology and treatments in 2017.Foamy Virus Vectors Transduce Visceral Organs and Hippocampal Structures following In Vivo Delivery to Neonatal MiceThe past, present and future management of sickle cell retinopathy within an African contextMultiple Integrated Non-clinical Studies Predict the Safety of Lentivirus-Mediated Gene Therapy for β-ThalassemiaGetting the Most: Enhancing Efficacy by Promoting Erythropoiesis and Thrombopoiesis after Gene Therapy in Mice with Hurler SyndromeHepatocyte ALOXE3 is induced during adaptive fasting and enhances insulin sensitivity by activating hepatic PPARγFetal hemoglobin induction in sickle erythroid progenitors using a synthetic zinc finger DNA-binding domainGene Therapy for HemoglobinopathiesAn Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease PhenotypeA ten year review of the sickle cell program in Muhimbili National Hospital, Tanzania
P2860
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P2860
description
2017 nî lūn-bûn
@nan
2017年の論文
@ja
2017年論文
@yue
2017年論文
@zh-hant
2017年論文
@zh-hk
2017年論文
@zh-mo
2017年論文
@zh-tw
2017年论文
@wuu
2017年论文
@zh
2017年论文
@zh-cn
name
Gene Therapy in a Patient with Sickle Cell Disease.
@en
type
label
Gene Therapy in a Patient with Sickle Cell Disease.
@en
prefLabel
Gene Therapy in a Patient with Sickle Cell Disease.
@en
P2093
P50
P921
P356
P1476
Gene Therapy in a Patient with Sickle Cell Disease.
@en
P2093
Benedicte Neven
David Grevent
Gabor Veres
Jean-Antoine Ribeil
Jean-François Meritet
Laura Sandler
Leslie Weber
Mariane de Montalembert
Olivier Negre
Pablo Bartolucci
P304
P356
10.1056/NEJMOA1609677
P407
P50
P577
2017-03-01T00:00:00Z