Literature DB >> 24012366

Broadening the indications for hematopoietic stem cell genetic therapies.

David A Williams1.   

Abstract

The use of recombinant retroviral vectors to effect corrective genetic therapies in hematopoietic stem cells (HSCs) has long been predicted to revolutionize medicine. Two recent papers in Science now show that this technology could be considered as effective as, and perhaps superior to, allogeneic HSC transplants in some rare diseases.
Copyright © 2013 Elsevier Inc. All rights reserved.

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Year:  2013        PMID： 24012366     DOI： 10.1016/j.stem.2013.08.006

Source DB:  PubMed          Journal:  Cell Stem Cell        ISSN： 1875-9777            Impact factor:   24.633


  7 in total

1.  Unexpected help: mTOR meets lentiviral vectors.

Authors:  Christian Brendel; David A Williams
Journal:  Blood       Date:  2014-08-07       Impact factor: 22.113

Review 2.  Concise review: lessons learned from clinical trials of gene therapy in monogenic immunodeficiency diseases.

Authors:  David A Williams; Adrian J Thrasher
Journal:  Stem Cells Transl Med       Date:  2014-03-28       Impact factor: 6.940

Review 3.  Evolving Gene Therapy in Primary Immunodeficiency.

Authors:  Adrian J Thrasher; David A Williams
Journal:  Mol Ther       Date:  2017-03-31       Impact factor: 11.454

4.  Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype.

Authors:  Christian Brendel; Swaroopa Guda; Raffaele Renella; Daniel E Bauer; Matthew C Canver; Young-Jo Kim; Matthew M Heeney; Denise Klatt; Jonathan Fogel; Michael D Milsom; Stuart H Orkin; Richard I Gregory; David A Williams
Journal:  J Clin Invest       Date:  2016-09-06       Impact factor: 14.808

5.  Platelets are efficient and protective depots for storage, distribution, and delivery of lysosomal enzyme in mice with Hurler syndrome.

Authors:  Mei Dai; Jingfen Han; Salim S El-Amouri; Roscoe O Brady; Dao Pan
Journal:  Proc Natl Acad Sci U S A       Date:  2014-02-03       Impact factor: 11.205

6.  Getting the Most: Enhancing Efficacy by Promoting Erythropoiesis and Thrombopoiesis after Gene Therapy in Mice with Hurler Syndrome.

Authors:  Jing-Fen Han; Salim S El-Amouri; Mei Dai; Phuong Cao; Dao Pan
Journal:  Mol Ther Methods Clin Dev       Date:  2018-10-10       Impact factor: 6.698

7.  In Vivo Tracking of Human Hematopoiesis Reveals Patterns of Clonal Dynamics during Early and Steady-State Reconstitution Phases.

Authors:  Luca Biasco; Danilo Pellin; Serena Scala; Francesca Dionisio; Luca Basso-Ricci; Lorena Leonardelli; Samantha Scaramuzza; Cristina Baricordi; Francesca Ferrua; Maria Pia Cicalese; Stefania Giannelli; Victor Neduva; David J Dow; Manfred Schmidt; Christof Von Kalle; Maria Grazia Roncarolo; Fabio Ciceri; Paola Vicard; Ernst Wit; Clelia Di Serio; Luigi Naldini; Alessandro Aiuti
Journal:  Cell Stem Cell       Date:  2016-05-26       Impact factor: 24.633
  7 in total

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