- Home
- Publications
- Publication Search
- Publication Details
Title
Sickle Cell Disease: From Genetics to Curative Approaches
Authors
Keywords
-
Journal
Annual Review of Genomics and Human Genetics
Volume 24, Issue 1, Pages 255-275
Publisher
Annual Reviews
Online
2023-08-26
DOI
10.1146/annurev-genom-120122-081037
References
Ask authors/readers for more resources
Related references
Note: Only part of the references are listed.- CRISPR-based genome editing through the lens of DNA repair
- (2022) Tarun S. Nambiar et al. MOLECULAR CELL
- Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial
- (2022) Elisa Magrin et al. NATURE MEDICINE
- Identification of novel HPFH-like mutations by CRISPR base editing that elevate the expression of fetal hemoglobin
- (2022) Nithin Sam Ravi et al. eLife
- ON-Target Adverse Events of CRISPR-Cas9 Nuclease: More Chaotic than Expected
- (2022) Julian Boutin et al. CRISPR Journal
- An evaluation of patient-reported outcomes in sickle cell disease within a conceptual model
- (2022) Marsha J. Treadwell et al. QUALITY OF LIFE RESEARCH
- High-level correction of the sickle mutation is amplified in vivo during erythroid differentiation
- (2022) Wendy Magis et al. iScience
- Sickle Cell Disease
- (2022) Patricia L. Kavanagh et al. JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION
- A precise and efficient adenine base editor
- (2022) Tianxiang Tu et al. MOLECULAR THERAPY
- Considerations for Selecting Cognitive Endpoints and Psychological Patient-Reported Outcomes for Clinical Trials in Pediatric Patients With Sickle Cell Disease
- (2022) Anna M. Hood et al. Frontiers in Neurology
- Hematopoietic Stem Cell Gene-Addition/Editing Therapy in Sickle Cell Disease
- (2022) Paula Germino-Watnick et al. Cells
- Real-World data on efficacy of L-glutamine in preventing sickle cell disease-related complications in pediatric and adult patients
- (2022) Narcisse Elenga et al. Frontiers in Medicine
- Choice of template delivery mitigates the genotoxic risk and adverse impact of editing in human hematopoietic stem cells
- (2022) Samuele Ferrari et al. Cell Stem Cell
- Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression
- (2022) Panagiotis Antoniou et al. Nature Communications
- CRISPRthripsis: The Risk of CRISPR/Cas9-induced Chromothripsis in Gene Therapy
- (2022) Mario Amendola et al. Stem Cells Translational Medicine
- Gene therapy for hemoglobinopathies
- (2021) Yvette C. Tanhehco TRANSFUSION AND APHERESIS SCIENCE
- Influence of UGT1A1 promoter polymorphism, α-thalassemia and βs haplotype in bilirubin levels and cholelithiasis in a large sickle cell anemia cohort
- (2021) Jéssica V. G. F. Batista et al. ANNALS OF HEMATOLOGY
- Genome Editing for β-Hemoglobinopathies: Advances and Challenges
- (2021) Giacomo Frati et al. Journal of Clinical Medicine
- Base editing of haematopoietic stem cells rescues sickle cell disease in mice
- (2021) Gregory A. Newby et al. NATURE
- Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease
- (2021) Annalisa Lattanzi et al. Science Translational Medicine
- Prioritizing Health-Sector Interventions for Noncommunicable Diseases and Injuries in Low- and Lower-Middle Income Countries: National NCDI Poverty Commissions
- (2021) Neil Gupta et al. Global Health-Science and Practice
- Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease
- (2021) Julie Kanter et al. NEW ENGLAND JOURNAL OF MEDICINE
- Acute Myeloid Leukemia Case after Gene Therapy for Sickle Cell Disease
- (2021) Sunita Goyal et al. NEW ENGLAND JOURNAL OF MEDICINE
- Comprehensive analysis of prime editing outcomes in human embryonic stem cells
- (2021) Omer Habib et al. NUCLEIC ACIDS RESEARCH
- Sickle cell disease in sub-Saharan Africa: transferable strategies for prevention and care
- (2021) Kevin Esoh et al. Lancet Haematology
- Curative options for sickle cell disease: haploidentical stem cell transplantation or gene therapy?
- (2020) Alexis Leonard et al. BRITISH JOURNAL OF HAEMATOLOGY
- The promise and challenge of therapeutic genome editing
- (2020) Jennifer A. Doudna NATURE
- Evaluation and minimization of Cas9-independent off-target DNA editing by cytosine base editors
- (2020) Jordan L. Doman et al. NATURE BIOTECHNOLOGY
- Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype
- (2020) Leslie Weber et al. Science Advances
- Directed evolution of adenine base editors with increased activity and therapeutic application
- (2020) Nicole M. Gaudelli et al. NATURE BIOTECHNOLOGY
- Therapeutic base editing of human hematopoietic stem cells
- (2020) Jing Zeng et al. NATURE MEDICINE
- A rationally engineered cytosine base editor retains high on-target activity while reducing both DNA and RNA off-target effects
- (2020) Erwei Zuo et al. NATURE METHODS
- Cost of health care for paediatric patients with sickle cell disease: An analysis of resource use and costs in a European country
- (2020) Frederick W. Thielen et al. PEDIATRIC BLOOD & CANCER
- Myelodysplastic syndrome unrelated to lentiviral vector in a patient treated with gene therapy for sickle cell disease
- (2020) Matthew M. Hsieh et al. Blood Advances
- Base editing: advances and therapeutic opportunities
- (2020) Elizabeth M. Porto et al. NATURE REVIEWS DRUG DISCOVERY
- Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease
- (2020) Erica B. Esrick et al. NEW ENGLAND JOURNAL OF MEDICINE
- CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia
- (2020) Haydar Frangoul et al. NEW ENGLAND JOURNAL OF MEDICINE
- Cytosine base editor generates substantial off-target single-nucleotide variants in mouse embryos
- (2019) Erwei Zuo et al. SCIENCE
- Cytosine, but not adenine, base editors induce genome-wide off-target mutations in rice
- (2019) Shuai Jin et al. SCIENCE
- Innovative Curative Treatment of Beta Thalassemia: Cost-Efficacy Analysis of Gene Therapy Versus Allogenic Hematopoietic Stem-Cell Transplantation
- (2019) Séverine Coquerelle et al. HUMAN GENE THERAPY
- Precise Gene Editing Preserves Hematopoietic Stem Cell Function following Transient p53-Mediated DNA Damage Response
- (2019) Giulia Schiroli et al. Cell Stem Cell
- Risk factors and outcomes according to age at transplantation with an HLA-identical sibling for sickle cell disease
- (2019) Barbara Cappelli et al. HAEMATOLOGICA
- Transcriptome-wide off-target RNA editing induced by CRISPR-guided DNA base editors
- (2019) Julian Grünewald et al. NATURE
- Off-target RNA mutation induced by DNA base editing and its elimination by mutagenesis
- (2019) Changyang Zhou et al. NATURE
- A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease
- (2019) Elliott Vichinsky et al. NEW ENGLAND JOURNAL OF MEDICINE
- Search-and-replace genome editing without double-strand breaks or donor DNA
- (2019) Andrew V. Anzalone et al. NATURE
- The epidemiology of sickle cell disease in children recruited in infancy in Kilifi, Kenya: a prospective cohort study
- (2019) Sophie Uyoga et al. Lancet Global Health
- Direct Promoter Repression by BCL11A Controls the Fetal to Adult Hemoglobin Switch
- (2018) Nan Liu et al. CELL
- Plerixafor enables safe, rapid, efficient mobilization of hematopoietic stem cells in sickle cell disease patients after exchange transfusion
- (2018) Chantal Lagresle-Peyrou et al. HAEMATOLOGICA
- Global Transcriptional Response to CRISPR/Cas9-AAV6-Based Genome Editing in CD34 + Hematopoietic Stem and Progenitor Cells
- (2018) M. Kyle Cromer et al. MOLECULAR THERAPY
- Repair of double-strand breaks induced by CRISPR–Cas9 leads to large deletions and complex rearrangements
- (2018) Michael Kosicki et al. NATURE BIOTECHNOLOGY
- Natural regulatory mutations elevate the fetal globin gene via disruption of BCL11A or ZBTB7A binding
- (2018) Gabriella E. Martyn et al. NATURE GENETICS
- p53 inhibits CRISPR–Cas9 engineering in human pluripotent stem cells
- (2018) Robert J. Ihry et al. NATURE MEDICINE
- CRISPR–Cas9 genome editing induces a p53-mediated DNA damage response
- (2018) Emma Haapaniemi et al. NATURE MEDICINE
- A Phase 3 Trial of l-Glutamine in Sickle Cell Disease
- (2018) Yutaka Niihara et al. NEW ENGLAND JOURNAL OF MEDICINE
- Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia
- (2018) Alexis A. Thompson et al. NEW ENGLAND JOURNAL OF MEDICINE
- Plerixafor enables safe, rapid, efficient mobilization of hematopoietic stem cells in sickle cell disease patients after exchange transfusion
- (2018) Chantal Lagresle-Peyrou et al. HAEMATOLOGICA
- A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells
- (2018) Christopher A. Vakulskas et al. NATURE MEDICINE
- Newborn screening for sickle cell disease in Europe: recommendations from a Pan-European Consensus Conference
- (2018) Stephan Lobitz et al. BRITISH JOURNAL OF HAEMATOLOGY
- Genetic Modifiers of Fetal Haemoglobin in Sickle Cell Disease
- (2018) Stephan Menzel et al. Molecular Diagnosis & Therapy
- Sickle cell disease
- (2017) Russell E Ware et al. LANCET
- Gene Therapy in a Patient with Sickle Cell Disease
- (2017) Jean-Antoine Ribeil et al. NEW ENGLAND JOURNAL OF MEDICINE
- Improving the DNA specificity and applicability of base editing through protein engineering and protein delivery
- (2017) Holly A. Rees et al. Nature Communications
- Indications and Results of HLA-Identical Sibling Hematopoietic Cell Transplantation for Sickle Cell Disease
- (2016) Mark C. Walters et al. BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION
- Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the βA(T87Q)-GlobinGene
- (2016) Olivier Negre et al. HUMAN GENE THERAPY
- CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells
- (2016) Daniel P. Dever et al. NATURE
- A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition
- (2016) Elizabeth A Traxler et al. NATURE MEDICINE
- Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells
- (2016) M. A. DeWitt et al. Science Translational Medicine
- Association of Variants at BCL11A and HBS1L-MYB with Hemoglobin F and Hospitalization Rates among Sickle Cell Patients in Cameroon
- (2014) Ambroise Wonkam et al. PLoS One
- The Co-Inheritance of Alpha-Thalassemia and Sickle Cell Anemia Is Associated with Better Hematological Indices and Lower Consultations Rate in Cameroonian Patients and Could Improve Their Survival
- (2014) Maryam Bibi Rumaney et al. PLoS One
- Unrelated Donor Allogeneic Hematopoietic Stem Cell Transplantation for Patients with Hemoglobinopathies Using a Reduced-Intensity Conditioning Regimen and Third-Party Mesenchymal Stromal Cells
- (2013) Sandhya Kharbanda et al. BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION
- After the Introduction into the National Newborn Screening Program: Who Is Receiving Genetic Counseling for Hemoglobinopathies in The Netherlands?
- (2013) J.O. Kaufmann et al. Public Health Genomics
- The BET Family of Proteins Targets Moloney Murine Leukemia Virus Integration near Transcription Start Sites
- (2013) Jan De Rijck et al. Cell Reports
- Global Burden of Sickle Cell Anaemia in Children under Five, 2010–2050: Modelling Based on Demographics, Excess Mortality, and Interventions
- (2013) Frédéric B. Piel et al. PLOS MEDICINE
- Oxidative stress in sickle cell disease; pathophysiology and potential implications for disease management
- (2011) Erfan Nur et al. AMERICAN JOURNAL OF HEMATOLOGY
- Fetal hemoglobin in sickle cell anemia
- (2011) I. Akinsheye et al. BLOOD
- High resource hospitalizations among children with vaso-occlusive crises in sickle cell disease
- (2011) Jean L. Raphael et al. PEDIATRIC BLOOD & CANCER
- Inhibition of cell adhesion by anti-P-selectin aptamer: a new potential therapeutic agent for sickle cell disease
- (2010) D. R. Gutsaeva et al. BLOOD
- Global distribution of the sickle cell gene and geographical confirmation of the malaria hypothesis
- (2010) Frédéric B. Piel et al. Nature Communications
- Maternal knowledge and attitudes about newborn screening for sickle cell disease and cystic fibrosis
- (2009) Colleen Walsh Lang et al. AMERICAN JOURNAL OF MEDICAL GENETICS PART A
- Identification of genetic polymorphisms associated with risk for pulmonary hypertension in sickle cell disease
- (2008) A. E. Ashley-Koch et al. BLOOD
- Sickle Cell Anemia, the First Molecular Disease: Overview of Molecular Etiology, Pathophysiology, and Therapeutic Approaches
- (2008) Martin H. Steinberg TheScientificWorldJOURNAL
Discover Peeref hubs
Discuss science. Find collaborators. Network.
Join a conversationPublish scientific posters with Peeref
Peeref publishes scientific posters from all research disciplines. Our Diamond Open Access policy means free access to content and no publication fees for authors.
Learn More