Literature DB >> 31738187

Are genetic approaches still needed to cure sickle cell disease?

Robert A Brodsky1, Michael R DeBaun2.   

Abstract

Mesh：

Year:  2020        PMID： 31738187      PMCID： PMC6934198          DOI： 10.1172/JCI133856

Source DB:  PubMed          Journal:  J Clin Invest        ISSN： 0021-9738            Impact factor:   14.808


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  16 in total

1.  Haploidentical Bone Marrow Transplantation with Post-Transplantation Cyclophosphamide Plus Thiotepa Improves Donor Engraftment in Patients with Sickle Cell Anemia: Results of an International Learning Collaborative.

Authors:  Josu de la Fuente; Nathalie Dhedin; Tatsuki Koyama; Francoise Bernaudin; Mathieu Kuentz; Leena Karnik; Gérard Socié; Kathryn A Culos; Robert A Brodsky; Michael R DeBaun; Adetola A Kassim
Journal:  Biol Blood Marrow Transplant       Date:  2018-11-28       Impact factor: 5.742

2.  Allogeneic donor availability for hematopoietic stem cell transplantation in children with sickle cell disease.

Authors:  David Justus; Evelio Perez-Albuerne; Jacqueline Dioguardi; David Jacobsohn; Allistair Abraham
Journal:  Pediatr Blood Cancer       Date:  2015-02-07       Impact factor: 3.167

3.  Decreased median survival of adults with sickle cell disease after adjusting for left truncation bias: a pooled analysis.

Authors:  Michael R DeBaun; Djamila L Ghafuri; Mark Rodeghier; Poulami Maitra; Shruti Chaturvedi; Adetola Kassim; Kenneth I Ataga
Journal:  Blood       Date:  2018-12-10       Impact factor: 22.113

4.  Successful hematopoietic stem cell mobilization and apheresis collection using plerixafor alone in sickle cell patients.

Authors:  Erica B Esrick; John P Manis; Heather Daley; Cristina Baricordi; Hélène Trébéden-Negre; Francis J Pierciey; Myriam Armant; Sarah Nikiforow; Matthew M Heeney; Wendy B London; Luca Biasco; Mohammed Asmal; David A Williams; Alessandra Biffi
Journal:  Blood Adv       Date:  2018-10-09

Review 5.  Sickle cell disease.

Authors:  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
Journal:  Nat Rev Dis Primers       Date:  2018-03-15       Impact factor: 52.329

6.  Cyclophosphamide improves engraftment in patients with SCD and severe organ damage who undergo haploidentical PBSCT.

Authors:  Courtney D Fitzhugh; Matthew M Hsieh; Tiffani Taylor; Wynona Coles; Katherine Roskom; Delon Wilson; Elizabeth Wright; Neal Jeffries; Christopher J Gamper; Jonathan Powell; Leo Luznik; John F Tisdale
Journal:  Blood Adv       Date:  2017-04-19

7.  Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia.

Authors:  Alexis A Thompson; Mark C Walters; Janet Kwiatkowski; John E J Rasko; Jean-Antoine Ribeil; Suradej Hongeng; Elisa Magrin; Gary J Schiller; Emmanuel Payen; Michaela Semeraro; Despina Moshous; Francois Lefrere; Hervé Puy; Philippe Bourget; Alessandra Magnani; Laure Caccavelli; Jean-Sébastien Diana; Felipe Suarez; Fabrice Monpoux; Valentine Brousse; Catherine Poirot; Chantal Brouzes; Jean-François Meritet; Corinne Pondarré; Yves Beuzard; Stany Chrétien; Thibaud Lefebvre; David T Teachey; Usanarat Anurathapan; P Joy Ho; Christof von Kalle; Morris Kletzel; Elliott Vichinsky; Sandeep Soni; Gabor Veres; Olivier Negre; Robert W Ross; David Davidson; Alexandria Petrusich; Laura Sandler; Mohammed Asmal; Olivier Hermine; Mariane De Montalembert; Salima Hacein-Bey-Abina; Stéphane Blanche; Philippe Leboulch; Marina Cavazzana
Journal:  N Engl J Med       Date:  2018-04-19       Impact factor: 91.245

Review 8.  Blood and marrow transplantation for sickle cell disease: is less more?

Authors:  Javier Bolaños-Meade; Robert A Brodsky
Journal:  Blood Rev       Date:  2014-08-27       Impact factor: 8.250

9.  CRISPR-Cas9 genome editing induces a p53-mediated DNA damage response.

Authors:  Emma Haapaniemi; Sandeep Botla; Jenna Persson; Bernhard Schmierer; Jussi Taipale
Journal:  Nat Med       Date:  2018-06-11       Impact factor: 53.440

10.  HLA-haploidentical bone marrow transplantation with posttransplant cyclophosphamide expands the donor pool for patients with sickle cell disease.

Authors:  Javier Bolaños-Meade; Ephraim J Fuchs; Leo Luznik; Sophie M Lanzkron; Christopher J Gamper; Richard J Jones; Robert A Brodsky
Journal:  Blood       Date:  2012-09-06       Impact factor: 22.113
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  4 in total

1.  The Meaning of Informed Consent: Genome Editing Clinical Trials for Sickle Cell Disease.

Authors:  Stacy Desine; Brittany M Hollister; Khadijah E Abdallah; Anitra Persaud; Sara Chandros Hull; Vence L Bonham
Journal:  AJOB Empir Bioeth       Date:  2020-10-12

Review 2.  Genetic therapies for the first molecular disease.

Authors:  Phillip A Doerfler; Akshay Sharma; Jerlym S Porter; Yan Zheng; John F Tisdale; Mitchell J Weiss
Journal:  J Clin Invest       Date:  2021-04-15       Impact factor: 14.808

Review 3.  Complement in Sickle Cell Disease: Are We Ready for Prime Time?

Authors:  Eleni Gavriilaki; Efthymia Vlachaki; Christos Varelas; Athina Tampaki; Ioanna Sakellari; Αchilles Anagnostopoulos
Journal:  J Blood Med       Date:  2021-03-23

Review 4.  Research in Sickle Cell Disease: From Bedside to Bench to Bedside.

Authors:  Gabriel Salinas Cisneros; Swee Lay Thein
Journal:  Hemasphere       Date:  2021-06-01
  4 in total

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