Anchored Lentiviral Vector Episomes for Stem Cell Gene Therapy in Fanconi Anemia

Current Gene Therapy ◽

10.2174/1566523217666170113165407 ◽

2017 ◽

Vol 16 (5) ◽

pp. 329-337 ◽

Author(s):

Santhosh Verghese ◽

Peter Kurre

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Fanconi Anemia ◽

Lentiviral Vector ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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276. Immunological and Metabolic Correction After Lentiviral Vector Mediated Haematopoietic Stem Cell Gene Therapy for ADA Deficiency

Molecular Therapy ◽

10.1016/s1525-0016(16)35289-3 ◽

2014 ◽

Vol 22 ◽

pp. S106 ◽

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Lentiviral Vector ◽

Haematopoietic Stem Cell ◽

Metabolic Correction ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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330. Lentiviral Vector Mediated Hematopoietic Stem Cell Gene Therapy Combined with Non-Lethal Conditioning Restores T Cell Function in the Murine Model of Wiskott-Aldrich Syndrome

Molecular Therapy ◽

10.1016/j.ymthe.2005.06.333 ◽

2005 ◽

Vol 11 ◽

pp. S128

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

T Cell ◽

Murine Model ◽

Cell Function ◽

Lentiviral Vector ◽

Hematopoietic Stem ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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314. Hematopoietic Stem Cell Gene Therapy with Lentiviral Vector in 4 Patients with Cerebral X-Linked Adrenoleukodystrophy

Molecular Therapy ◽

10.1016/s1525-0016(16)36118-4 ◽

2012 ◽

Vol 20 ◽

pp. S124

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Hematopoietic Stem Cell ◽

Lentiviral Vector ◽

Hematopoietic Stem ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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Fanconi anemia and βc deficiency-associated pulmonary alveolar proteinosis as two hereditary diseases of childhood which are potentially curable by stem cell gene therapy but require different therapeutic approaches

Klinische Pädiatrie ◽

10.1055/s-2008-1043810 ◽

1999 ◽

Vol 211 (04) ◽

pp. 329-335 ◽

Author(s):

U. Dirksen ◽

T. Moritz ◽

S. Burdach ◽

M. Flasshove ◽

H. Hanenberg

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Fanconi Anemia ◽

Pulmonary Alveolar Proteinosis ◽

Hereditary Diseases ◽

Therapeutic Approaches ◽

Alveolar Proteinosis ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy

Science ◽

10.1126/science.1171242 ◽

2009 ◽

Vol 326 (5954) ◽

pp. 818-823 ◽

Cited By ~ 1010

Author(s):

N. Cartier ◽

S. Hacein-Bey-Abina ◽

C. C. Bartholomae ◽

G. Veres ◽

M. Schmidt ◽

...

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Hematopoietic Stem Cell ◽

Lentiviral Vector ◽

Hematopoietic Stem ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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Pre-clinical Safety and Efficacy of Lentiviral Vector-Mediated Ex Vivo Stem Cell Gene Therapy for the Treatment of Mucopolysaccharidosis IIIA

Molecular Therapy — Methods & Clinical Development ◽

10.1016/j.omtm.2019.04.001 ◽

2019 ◽

Vol 13 ◽

pp. 399-413 ◽

Author(s):

Stuart M. Ellison ◽

Aiyin Liao ◽

Shaun Wood ◽

Jessica Taylor ◽

Amir Saam Youshani ◽

...

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Lentiviral Vector ◽

Ex Vivo ◽

Clinical Safety ◽

Safety And Efficacy ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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279. Efficient and Safe Lentiviral Vector-Mediated Hematopoietic Stem Cell Gene Therapy in MNGIE Mice

Molecular Therapy ◽

10.1016/s1525-0016(16)33888-6 ◽

2015 ◽

Vol 23 ◽

pp. S111-S112

Author(s):

Rana Yadak ◽

Javier T. Torronteras ◽

Raquel C. Pérez ◽

Elly Bogaerts ◽

George de Ruijter ◽

...

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Hematopoietic Stem Cell ◽

Lentiviral Vector ◽

Hematopoietic Stem ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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336. Lentiviral Vector Enhanced Haematopoietic Stem Cell Gene Therapy Corrects the Neurological Phenotype of Mucopolysaccharidosis IIIA Mice

Molecular Therapy ◽

10.1016/s1525-0016(16)36908-8 ◽

2011 ◽

Vol 19 ◽

pp. S130

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Lentiviral Vector ◽

Haematopoietic Stem Cell ◽

Neurological Phenotype ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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712. Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector for the Multi-Systemic Lysosomal Storage Disorder Cystinosis

Molecular Therapy ◽

10.1016/s1525-0016(16)37285-9 ◽

2011 ◽

Vol 19 ◽

pp. S272

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Hematopoietic Stem Cell ◽

Lysosomal Storage Disorder ◽

Lentiviral Vector ◽

Lysosomal Storage ◽

Hematopoietic Stem ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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Gamma-globin lentiviral vector-mediated stem cell gene therapy of beta-thalassemia and sickle cell disease

Blood Cells Molecules and Diseases ◽

10.1016/j.bcmd.2007.10.070 ◽

2008 ◽

Vol 40 (2) ◽

pp. 281

Author(s):

Huifen Zhao ◽

Tamara Pestina ◽

Phillip Hargrove ◽

Yoon Sang Kim ◽

Robert Throm ◽

...

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Sickle Cell Disease ◽

Lentiviral Vector ◽

Beta Thalassemia ◽

Cell Disease ◽

Gamma Globin ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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