Literature DB >> 17173507

Therapeutic options for patients with severe beta-thalassemia: the need for globin gene therapy.

Michel Sadelain1, Farid Boulad, Renzo Galanello, Patricia Giardina, Franco Locatelli, Aurelio Maggio, Stefano Rivella, Isabelle Riviere, John Tisdale.   

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Year:  2007        PMID： 17173507     DOI： 10.1089/hum.2006.151

Source DB:  PubMed          Journal:  Hum Gene Ther        ISSN： 1043-0342            Impact factor:   5.695


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  18 in total

1.  Future alternative therapies for β-thalassemia.

Authors:  Stefano Rivella; Eliezer Rachmilewitz
Journal:  Expert Rev Hematol       Date:  2009-12-01       Impact factor: 2.929

2.  Correction of β-thalassemia by CRISPR/Cas9 editing of the α-globin locus in human hematopoietic stem cells.

Authors:  Giulia Pavani; Anna Fabiano; Marine Laurent; Fatima Amor; Erika Cantelli; Anne Chalumeau; Giulia Maule; Alexandra Tachtsidi; Jean-Paul Concordet; Anna Cereseto; Fulvio Mavilio; Giuliana Ferrari; Annarita Miccio; Mario Amendola
Journal:  Blood Adv       Date:  2021-03-09

3.  Plerixafor+G-CSF-mobilized CD34+ cells represent an optimal graft source for thalassemia gene therapy.

Authors:  Garyfalia Karponi; Nikoletta Psatha; Carsten Werner Lederer; Jennifer Eileen Adair; Fani Zervou; Nikolaos Zogas; Marina Kleanthous; Constantinos Tsatalas; Achilles Anagnostopoulos; Michel Sadelain; Isabelle Rivière; George Stamatoyannopoulos; Evangelia Yannaki
Journal:  Blood       Date:  2015-06-18       Impact factor: 22.113

Review 4.  Beta-thalassemia.

Authors:  Renzo Galanello; Raffaella Origa
Journal:  Orphanet J Rare Dis       Date:  2010-05-21       Impact factor: 4.123

Review 5.  Gene therapy for hemoglobinopathies: progress and challenges.

Authors:  Alisa Dong; Stefano Rivella; Laura Breda
Journal:  Transl Res       Date:  2013-01-19       Impact factor: 7.012

6.  A cis-proline in alpha-hemoglobin stabilizing protein directs the structural reorganization of alpha-hemoglobin.

Authors:  David A Gell; Liang Feng; Suiping Zhou; Philip D Jeffrey; Katerina Bendak; Andrew Gow; Mitchell J Weiss; Yigong Shi; Joel P Mackay
Journal:  J Biol Chem       Date:  2009-08-25       Impact factor: 5.157

7.  Locus control region elements HS1 and HS4 enhance the therapeutic efficacy of globin gene transfer in beta-thalassemic mice.

Authors:  Leszek Lisowski; Michel Sadelain
Journal:  Blood       Date:  2007-10-05       Impact factor: 22.113

Review 8.  Cell and Gene Therapy for the Beta-Thalassemias: Advances and Prospects.

Authors:  Jorge Mansilla-Soto; Isabelle Riviere; Farid Boulad; Michel Sadelain
Journal:  Hum Gene Ther       Date:  2016-04       Impact factor: 5.695

9.  CRISPR/Cas9 gene correction of HbH-CS thalassemia-induced pluripotent stem cells.

Authors:  Xie Yingjun; Xie Yuhuan; Chen Yuchang; Li Dongzhi; Wang Ding; Song Bing; Yang Yi; Lu Dian; Xue Yanting; Xiong Zeyu; Liu Nengqing; Chen Diyu; Sun Xiaofang
Journal:  Ann Hematol       Date:  2019-09-09       Impact factor: 3.673

10.  Low-dose parenteral busulfan provides an extended window for the infusion of hematopoietic stem cells in murine hosts.

Authors:  Matthew M Hsieh; Saskia Langemeijer; Aisha Wynter; Oswald A Phang; Elizabeth M Kang; John F Tisdale
Journal:  Exp Hematol       Date:  2007-07-09       Impact factor: 3.084
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