Literature DB >> 27081721

Gene Therapy for Hemoglobinopathies: Tremendous Successes and Remaining Caveats.

Punam Malik1.   

Abstract

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Year:  2016        PMID： 27081721      PMCID： PMC4886952          DOI： 10.1038/mt.2016.57

Source DB:  PubMed          Journal:  Mol Ther        ISSN： 1525-0016            Impact factor:   11.454


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  25 in total

1.  High-level beta-globin expression and preferred intragenic integration after lentiviral transduction of human cord blood stem cells.

Authors:  Suzan Imren; Mary E Fabry; Karen A Westerman; Robert Pawliuk; Patrick Tang; Patricia M Rosten; Ronald L Nagel; Philippe Leboulch; Connie J Eaves; R Keith Humphries
Journal:  J Clin Invest       Date:  2004-10       Impact factor: 14.808

2.  A novel human gamma-globin gene vector for genetic correction of sickle cell anemia in a humanized sickle mouse model: critical determinants for successful correction.

Authors:  Ajay Perumbeti; Tomoyasu Higashimoto; Fabrizia Urbinati; Robert Franco; Herbert J Meiselman; David Witte; Punam Malik
Journal:  Blood       Date:  2009-05-27       Impact factor: 22.113

3.  Mixed chimerism after bone marrow transplantation in thalassemia.

Authors:  S Nesci; M Manna; G Lucarelli; P Tonucci; M Donati; O Buffi; F Agostinelli; M Andreani
Journal:  Ann N Y Acad Sci       Date:  1998-06-30       Impact factor: 5.691

4.  A novel murine model of Cooley anemia and its rescue by lentiviral-mediated human beta-globin gene transfer.

Authors:  Stefano Rivella; Chad May; Amy Chadburn; Isabelle Rivière; Michel Sadelain
Journal:  Blood       Date:  2002-12-12       Impact factor: 22.113

5.  The degree of phenotypic correction of murine beta -thalassemia intermedia following lentiviral-mediated transfer of a human gamma-globin gene is influenced by chromosomal position effects and vector copy number.

Authors:  Derek A Persons; Phillip W Hargrove; Esther R Allay; Hideki Hanawa; Arthur W Nienhuis
Journal:  Blood       Date:  2002-10-31       Impact factor: 22.113

6.  A recombinant human hemoglobin with anti-sickling properties greater than fetal hemoglobin.

Authors:  Dana N Levasseur; Thomas M Ryan; Michael P Reilly; Steven L McCune; Toshio Asakura; Tim M Townes
Journal:  J Biol Chem       Date:  2004-04-14       Impact factor: 5.157

Review 7.  Management of sickle cell disease: summary of the 2014 evidence-based report by expert panel members.

Authors:  Barbara P Yawn; George R Buchanan; Araba N Afenyi-Annan; Samir K Ballas; Kathryn L Hassell; Andra H James; Lanetta Jordan; Sophie M Lanzkron; Richard Lottenberg; William J Savage; Paula J Tanabe; Russell E Ware; M Hassan Murad; Jonathan C Goldsmith; Eduardo Ortiz; Robinson Fulwood; Ann Horton; Joylene John-Sowah
Journal:  JAMA       Date:  2014-09-10       Impact factor: 56.272

8.  Quantitatively different red cell/nucleated cell chimerism in patients with long-term, persistent hematopoietic mixed chimerism after bone marrow transplantation for thalassemia major or sickle cell disease.

Authors:  Marco Andreani; Manuela Testi; Javid Gaziev; Rossella Condello; Andrea Bontadini; Pier Luigi Tazzari; Francesca Ricci; Lidia De Felice; Francesca Agostini; Daniela Fraboni; Giuliana Ferrari; Mariarosa Battarra; Maria Troiano; Pietro Sodani; Guido Lucarelli
Journal:  Haematologica       Date:  2010-10-07       Impact factor: 9.941

9.  Preclinical evaluation of efficacy and safety of an improved lentiviral vector for the treatment of β-thalassemia and sickle cell disease.

Authors:  Olivier Negre; Cynthia Bartholomae; Yves Beuzard; Marina Cavazzana; Lauryn Christiansen; Céline Courne; Annette Deichmann; Maria Denaro; Edouard de Dreuzy; Mitchell Finer; Raffaele Fronza; Béatrix Gillet-Legrand; Christophe Joubert; Robert Kutner; Philippe Leboulch; Leïla Maouche; Anaïs Paulard; Francis J Pierciey; Michael Rothe; Byoung Ryu; Manfred Schmidt; Christof von Kalle; Emmanuel Payen; Gabor Veres
Journal:  Curr Gene Ther       Date:  2015       Impact factor: 4.391

10.  Pigtailed macaques as a model to study long-term safety of lentivirus vector-mediated gene therapy for hemoglobinopathies.

Authors:  Hans-Peter Kiem; Paritha I Arumugam; Christopher R Burtner; Catherine F Fox; Brian C Beard; Phillip Dexheimer; Jennifer E Adair; Punam Malik
Journal:  Mol Ther Methods Clin Dev       Date:  2014-12-17       Impact factor: 6.698
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  6 in total

1.  Lentiviral Transfer of γ-Globin with Fusion Gene NUP98-HOXA10HD Expands Hematopoietic Stem Cells and Ameliorates Murine β-Thalassemia.

Authors:  Hui Fen Zhao; Allistair Abraham; Yoon-Sang Kim; Yong-Dong Wang; Tamara Pestina; Jun Zhan; Keith Humphries; Arthur W Nienhuis; Derek A Persons
Journal:  Mol Ther       Date:  2017-02-09       Impact factor: 11.454

2.  Family cord blood banking for sickle cell disease: a twenty-year experience in two dedicated public cord blood banks.

Authors:  Hanadi Rafii; Françoise Bernaudin; Helene Rouard; Valérie Vanneaux; Annalisa Ruggeri; Marina Cavazzana; Valerie Gauthereau; Aurélie Stanislas; Malika Benkerrou; Mariane De Montalembert; Christele Ferry; Robert Girot; Cecile Arnaud; Annie Kamdem; Joelle Gour; Claudine Touboul; Audrey Cras; Mathieu Kuentz; Claire Rieux; Fernanda Volt; Barbara Cappelli; Karina T Maio; Annalisa Paviglianiti; Chantal Kenzey; Jerome Larghero; Eliane Gluckman
Journal:  Haematologica       Date:  2017-03-16       Impact factor: 9.941

Review 3.  Gene therapy in PIDs, hemoglobin, ocular, neurodegenerative, and hemophilia B disorders.

Authors:  Arome Solomon Odiba; Nkwachukwu Oziamara Okoro; Olanrewaju Ayodeji Durojaye; Yanjun Wu
Journal:  Open Life Sci       Date:  2021-05-03       Impact factor: 0.938

Review 4.  Clinical development of gene therapy: results and lessons from recent successes.

Authors:  Sandeep Rp Kumar; David M Markusic; Moanaro Biswas; Katherine A High; Roland W Herzog
Journal:  Mol Ther Methods Clin Dev       Date:  2016-05-25       Impact factor: 6.698

5.  A comparison of intrauterine hemopoietic cell transplantation and lentiviral gene transfer for the correction of severe β-thalassemia in a HbbTh3/+ murine model.

Authors:  Niraja M Dighe; Kang Wei Tan; Lay Geok Tan; Steven S W Shaw; Suzanne M K Buckley; Dedy Sandikin; Nuryanti Johana; Yi-Wan Tan; Arijit Biswas; Mahesh Choolani; Simon N Waddington; Michael N Antoniou; Jerry K Y Chan; Citra N Z Mattar
Journal:  Exp Hematol       Date:  2018-03-29       Impact factor: 3.084

6.  Allogeneic hematopoietic stem cell transplantation in congenital hemoglobinopathies with myeloablative conditioning and rabbit anti-thymocyte globulin.

Authors:  Bo-Kyoung Park; Hyo-Sup Kim; Seongkoo Kim; Jae-Wook Lee; Young Shil Park; Pil-Sang Jang; Nack-Gyun Chung; Dae-Chul Jeong; Bin Cho
Journal:  Blood Res       Date:  2018-06-25
  6 in total

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