Literature DB >> 25184354

Lentiviral hematopoietic stem cell gene therapy in inherited metabolic disorders.

Gerard Wagemaker1.   

Abstract

After more than 20 years of development, lentiviral hematopoietic stem cell gene therapy has entered the stage of initial clinical implementation for immune deficiencies and storage disorders. This brief review summarizes the development and applications, focusing on the lysosomal enzyme deficiencies, especially Pompe disease.

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Year:  2014        PMID： 25184354      PMCID： PMC4179920          DOI： 10.1089/hum.2014.102

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


  35 in total

Review 1.  Chance or necessity? Insertional mutagenesis in gene therapy and its consequences.

Authors:  Christopher Baum; Christof von Kalle; Frank J T Staal; Zhixiong Li; Boris Fehse; Manfred Schmidt; Floor Weerkamp; Stefan Karlsson; Gerard Wagemaker; David A Williams
Journal:  Mol Ther       Date:  2004-01       Impact factor: 11.454

2.  Mobilization as a preparative regimen for hematopoietic stem cell transplantation.

Authors:  Jing Chen; André Larochelle; Simon Fricker; Gary Bridger; Cynthia E Dunbar; Janis L Abkowitz
Journal:  Blood       Date:  2006-01-26       Impact factor: 22.113

3.  Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector.

Authors:  H Bobby Gaspar; Kathryn L Parsley; Steven Howe; Doug King; Kimberly C Gilmour; Joanna Sinclair; Gaby Brouns; Manfred Schmidt; Christof Von Kalle; Torben Barington; Marianne A Jakobsen; Hans O Christensen; Abdulaziz Al Ghonaium; Harry N White; John L Smith; Roland J Levinsky; Robin R Ali; Christine Kinnon; Adrian J Thrasher
Journal:  Lancet       Date:  2004 Dec 18-31       Impact factor: 79.321

4.  Bone marrow transplantation for Pompé's disease.

Authors:  P M Hoogerbrugge; G Wagemaker; D W van Bekkum; A J Reuser; A T vd Ploeg
Journal:  N Engl J Med       Date:  1986-07-03       Impact factor: 91.245

5.  Donor-derived cells in the central nervous system of twitcher mice after bone marrow transplantation.

Authors:  P M Hoogerbrugge; K Suzuki; K Suzuki; B J Poorthuis; T Kobayashi; G Wagemaker; D W van Bekkum
Journal:  Science       Date:  1988-02-26       Impact factor: 47.728

6.  Woodchuck hepatitis virus post-transcriptional regulatory element deleted from X protein and promoter sequences enhances retroviral vector titer and expression.

Authors:  A Schambach; J Bohne; C Baum; F G Hermann; L Egerer; D von Laer; T Giroglou
Journal:  Gene Ther       Date:  2006-04       Impact factor: 5.250

7.  Graft-versus-host disease in children who have received a cord-blood or bone marrow transplant from an HLA-identical sibling. Eurocord and International Bone Marrow Transplant Registry Working Committee on Alternative Donor and Stem Cell Sources.

Authors:  V Rocha; J E Wagner; K A Sobocinski; J P Klein; M J Zhang; M M Horowitz; E Gluckman
Journal:  N Engl J Med       Date:  2000-06-22       Impact factor: 91.245

8.  Isolation and characterisation of a recombinant, precursor form of lysosomal acid alpha-glucosidase.

Authors:  M Fuller; A Van der Ploeg; A J Reuser; D S Anson; J J Hopwood
Journal:  Eur J Biochem       Date:  1995-12-15

9.  In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector.

Authors:  L Naldini; U Blömer; P Gallay; D Ory; R Mulligan; F H Gage; I M Verma; D Trono
Journal:  Science       Date:  1996-04-12       Impact factor: 47.728

10.  Enzyme therapy and immune response in relation to CRIM status: the Dutch experience in classic infantile Pompe disease.

Authors:  Carin M van Gelder; Marianne Hoogeveen-Westerveld; Marian A Kroos; Iris Plug; Ans T van der Ploeg; Arnold J J Reuser
Journal:  J Inherit Metab Dis       Date:  2014-04-09       Impact factor: 4.982
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  6 in total

1.  Pseudotyping of lentiviral vector with novel vesiculovirus envelope glycoproteins derived from Chandipura and Piry viruses.

Authors:  Shuang Hu; Dipu Mohan Kumar; Chelsea Sax; Clayton Schuler; Ramesh Akkina
Journal:  Virology       Date:  2015-11-30       Impact factor: 3.616

Review 2.  THE JEREMIAH METZGER LECTURE NOVEL THERAPEUTIC STRATEGIES OF ALLERGIC AND IMMUNOLOGIC DISORDERS.

Authors:  Ronald G Crystal; Odelya E Pagovich
Journal:  Trans Am Clin Climatol Assoc       Date:  2018

Review 3.  Phenylketonuria.

Authors:  Francjan J van Spronsen; Nenad Blau; Cary Harding; Alberto Burlina; Nicola Longo; Annet M Bosch
Journal:  Nat Rev Dis Primers       Date:  2021-05-20       Impact factor: 52.329

Review 4.  Targeted approaches to induce immune tolerance for Pompe disease therapy.

Authors:  Phillip A Doerfler; Sushrusha Nayak; Manuela Corti; Laurence Morel; Roland W Herzog; Barry J Byrne
Journal:  Mol Ther Methods Clin Dev       Date:  2016-01-27       Impact factor: 6.698

Review 5.  Mitochondrial Neurogastrointestinal Encephalomyopathy Caused by Thymidine Phosphorylase Enzyme Deficiency: From Pathogenesis to Emerging Therapeutic Options.

Authors:  Rana Yadak; Peter Sillevis Smitt; Marike W van Gisbergen; Niek P van Til; Irenaeus F M de Coo
Journal:  Front Cell Neurosci       Date:  2017-02-15       Impact factor: 5.505

6.  Preclinical Efficacy and Safety Evaluation of Hematopoietic Stem Cell Gene Therapy in a Mouse Model of MNGIE.

Authors:  Rana Yadak; Raquel Cabrera-Pérez; Javier Torres-Torronteras; Marianna Bugiani; Joost C Haeck; Marshall W Huston; Elly Bogaerts; Steffi Goffart; Edwin H Jacobs; Merel Stok; Lorena Leonardelli; Luca Biasco; Robert M Verdijk; Monique R Bernsen; George Ruijter; Ramon Martí; Gerard Wagemaker; Niek P van Til; Irenaeus F M de Coo
Journal:  Mol Ther Methods Clin Dev       Date:  2018-01-08       Impact factor: 6.698
  6 in total

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