404. Gene Repair/Modification and Targeted Integration of Minigenes: Toward Improved Ex Vivo/In Vivo Stem-Cell Gene-Therapy

Molecular Therapy ◽

10.1016/s1525-0016(16)43234-x ◽

2002 ◽

Vol 5 (5) ◽

pp. S133

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Ex Vivo ◽

Gene Repair ◽

Targeted Integration ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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710. Stem Cell Gene Therapy: Synergizing Improved Gene Repair/Modification & Targeted Integration of Transgenes to Transient In Vivo Topical Minigene Expression

Molecular Therapy ◽

10.1016/j.ymthe.2004.06.617 ◽

2004 ◽

Vol 9 ◽

pp. S271

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Gene Repair ◽

Targeted Integration ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy ◽

Minigene Expression

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350. Toward Genosafe Endonuclease-Boosted Gene Targeting Using Breakthrough CRISPR/Cas9 for Next Generation Stem Cell Gene Therapy Culminating in Efficient Ex Vivo/In Vivo Gene Repair/Genomic Editing

Molecular Therapy ◽

10.1016/s1525-0016(16)33959-4 ◽

2015 ◽

Vol 23 ◽

pp. S139

Author(s):

Roger Bertolotti

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Gene Targeting ◽

Ex Vivo ◽

Gene Repair ◽

Genomic Editing ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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244. Transient Epigenetic Gene Therapy as a Rising Arm of a Universal Stem Cell Gene Therapy Platform both for Ex Vivo and In Vivo Applications

Molecular Therapy ◽

10.1016/s1525-0016(16)37685-7 ◽

2010 ◽

Vol 18 ◽

pp. S93

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Ex Vivo ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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Hematopoietic stem cell gene therapy of murine protoporphyria by methylguanine-DNA-methyltransferase-mediated in vivo drug selection

Gene Therapy ◽

10.1038/sj.gt.3302335 ◽

2004 ◽

Vol 11 (22) ◽

pp. 1638-1647 ◽

Author(s):

E Richard ◽

E Robert ◽

M Cario-André ◽

C Ged ◽

F Géronimi ◽

...

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Dna Methyltransferase ◽

Drug Selection ◽

Hematopoietic Stem ◽

Methylguanine Dna Methyltransferase ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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The SCID-hu mouse: an in-vivo model for HIV-1 pathogenesis and stem cell gene therapy for AIDS

Seminars in Immunology ◽

10.1006/smim.1996.0027 ◽

1996 ◽

Vol 8 (4) ◽

pp. 215-221 ◽

Author(s):

B Jamieson

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

In Vivo Model ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy ◽

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Case report of the first patient treated with ex-vivo autologous haematopoietic stem cell gene therapy transplant in mucopolysaccharidosis type IIIA

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2019.11.225 ◽

2020 ◽

Vol 129 (2) ◽

pp. S90 ◽

Author(s):

Jane Louise Kinsella ◽

Heather Church ◽

Wendy Ogden ◽

Ceri Jones ◽

Karen Buckland ◽

...

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Case Report ◽

Ex Vivo ◽

Haematopoietic Stem Cell ◽

Mucopolysaccharidosis Type ◽

Cell Gene ◽

Mucopolysaccharidosis Type Iiia ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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442. LV Expressing MR Reporter Genes Allows In Vivo Monitoring of Stem Cell Gene Therapy

Molecular Therapy ◽

10.1016/j.ymthe.2006.08.509 ◽

2006 ◽

Vol 13 ◽

pp. S170

Author(s):

Mario Amendola ◽

Letterio S. Politi ◽

Marcello Cadioli ◽

Rossella Galli ◽

Elena Binda ◽

...

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Reporter Genes ◽

In Vivo Monitoring ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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Curative therapy for hemoglobinopathies: an International Society for Cell & Gene Therapy Stem Cell Engineering Committee review comparing outcomes, accessibility and cost of ex vivo stem cell gene therapy versus allogeneic hematopoietic stem cell transplantation

Cytotherapy ◽

10.1016/j.jcyt.2021.09.003 ◽

2021 ◽

Author(s):

Alexis Leonard ◽

Alice Bertaina ◽

Carmem Bonfim ◽

Sandra Cohen ◽

Susan Prockop ◽

...

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Ex Vivo ◽

Cell Engineering ◽

Hematopoietic Stem ◽

Curative Therapy ◽

Stem Cell Engineering ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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In Vivo Hematopoietic Stem Cell Gene Therapy Might Become a Reality

Human Gene Therapy ◽

10.1089/hum.2020.29143.agg ◽

2021 ◽

Vol 32 (1-2) ◽

pp. 14-16

Author(s):

Alejandra Gutierrez-Guerrero ◽

Els Verhoeyen

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Hematopoietic Stem Cell ◽

Hematopoietic Stem ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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Ex-vivo autologous stem cell gene therapy clinical trial for mucopolysaccharidosis type IIIA: Update on phase I/II clinical trial

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2020.12.126 ◽

2021 ◽

Vol 132 (2) ◽

pp. S56-S57

Author(s):

Jane Louise Kinsella ◽

Simon Jones ◽

Adrian J. Thrasher ◽

Claire Booth ◽

Karen F. Buckland ◽

...

Keyword(s):

Clinical Trial ◽

Gene Therapy ◽

Stem Cell ◽

Ex Vivo ◽

Mucopolysaccharidosis Type ◽

Cell Gene ◽

Mucopolysaccharidosis Type Iiia ◽

Stem Cell Gene ◽

Therapy Clinical Trial ◽

Stem Cell Gene Therapy

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