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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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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383. Zinc Finger Nuclease-Boosted Gene Targeting and Synergistic Transient Regenerative Stem Cell Gene Therapy: Toward Clinical Gene Repair/Alteration and Custom Site-Specific Integrative Gene Therapy

Molecular Therapy ◽

10.1016/j.ymthe.2005.06.386 ◽

2005 ◽

Vol 11 ◽

pp. S148-S149 ◽

Cited By ~ 2

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Gene Targeting ◽

Zinc Finger ◽

Zinc Finger Nuclease ◽

Gene Repair ◽

Site Specific ◽

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 ◽

Cited By ~ 32

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 ◽

Cited By ~ 21

Author(s):

B Jamieson

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

In Vivo Model ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy ◽

Hiv 1

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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 ◽

Cited By ~ 1

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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Zinc finger nuclease-boosted gene targeting and synergistic transient regenerative gene therapy for long-term stem cell gene therapy

Biogenic Amines ◽

10.1163/1569391041501915 ◽

2004 ◽

Vol 18 (3) ◽

pp. 503-538 ◽

Cited By ~ 2

Author(s):

Roger Bertolotti

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Gene Targeting ◽

Zinc Finger ◽

Zinc Finger Nuclease ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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1018. Stem Cell Gene Therapy: Toward a Universal Platform Relying on Custom Endonuclease-Boosted Gene Targeting, Cybridization, Transient Regenerative/Epigenetic Gene Therapy and Synergistic Combinations

Molecular Therapy ◽

10.1016/j.ymthe.2006.08.1112 ◽

2006 ◽

Vol 13 ◽

pp. S392 ◽

Cited By ~ 2

Author(s):

Roger Bertolotti

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Gene Targeting ◽

Cell Gene ◽

Synergistic Combinations ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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