678. Update of Stem Cell Gene Therapy Clinical Trial for ADA-Deficiency in Japan

Molecular Therapy ◽

10.1016/s1525-0016(16)38119-9 ◽

2010 ◽

Vol 18 ◽

pp. S265

Keyword(s):

Clinical Trial ◽

Gene Therapy ◽

Stem Cell ◽

Cell Gene ◽

Stem Cell Gene ◽

Therapy Clinical Trial ◽

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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880. Hematopoietic Stem Cell Gene Therapy for the Treatment of Metachromatic Leukodystrophy: Clinical Trial Design

Molecular Therapy ◽

10.1016/s1525-0016(16)40283-2 ◽

2008 ◽

Vol 16 ◽

pp. S328-S329

Keyword(s):

Clinical Trial ◽

Gene Therapy ◽

Stem Cell ◽

Trial Design ◽

Metachromatic Leukodystrophy ◽

Clinical Trial Design ◽

Hematopoietic Stem ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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Hematopoietic stem cell gene therapy for cystinosis: Updated results from a phase I/II clinical trial

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2020.12.039 ◽

2021 ◽

Vol 132 (2) ◽

pp. S25

Author(s):

Stephanie Cherqui

Keyword(s):

Clinical Trial ◽

Gene Therapy ◽

Stem Cell ◽

Phase I ◽

Hematopoietic Stem Cell ◽

Hematopoietic Stem ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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Faculty Opinions recommendation of Repetitive busulfan administration after hematopoietic stem cell gene therapy associated with a dominant HDAC7 clone in a nonhuman primate.

Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature ◽

10.3410/f.4242977.4039078 ◽

2010 ◽

Author(s):

Karen E Pollok ◽

Shanbao Cai

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Hematopoietic Stem Cell ◽

Nonhuman Primate ◽

Hematopoietic Stem ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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Stem Cell Gene Therapy for Chronic Renal Failure

Current Gene Therapy ◽

10.2174/1566523034578221 ◽

2003 ◽

Vol 3 (5) ◽

pp. 387-394 ◽

Author(s):

T. Yokoo ◽

K. Sakurai ◽

T. Ohashi ◽

T. Kawamura

Keyword(s):

Gene Therapy ◽

Renal Failure ◽

Stem Cell ◽

Chronic Renal Failure ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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Long-term hematopoietic stem cell gene therapy corrects neuromuscular manifestations in preclinical study of Pompe mice

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2020.12.263 ◽

2021 ◽

Vol 132 (2) ◽

pp. S107

Author(s):

Niek P. van Til ◽

Yildirim Dogan ◽

Cecilia Barese ◽

Zeenath Unnisa ◽

Swaroopa Guda ◽

...

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Hematopoietic Stem Cell ◽

Preclinical Study ◽

Hematopoietic Stem ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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Update on safety and efficacy of lentiviral hematopoietic stem cell gene therapy (HSC-GT) for metachromatic leukodystrophy (MLD)

European Journal of Paediatric Neurology ◽

10.1016/j.ejpn.2017.04.986 ◽

2017 ◽

Vol 21 ◽

pp. e20

Author(s):

F. Fumagalli ◽

V. Calbi ◽

A. Zambon ◽

F. Ciotti ◽

L. Lorioli ◽

...

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Hematopoietic Stem Cell ◽

Metachromatic Leukodystrophy ◽

Hematopoietic Stem ◽

Safety And Efficacy ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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688. Towards a Stem Cell Gene Therapy for Breast Cancer

Molecular Therapy ◽

10.1016/s1525-0016(16)39046-3 ◽

2009 ◽

Vol 17 ◽

pp. S263

Keyword(s):

Breast Cancer ◽

Gene Therapy ◽

Stem Cell ◽

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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Historical Perspective on the Current Renaissance for Hematopoietic Stem Cell Gene Therapy

Hematology/Oncology Clinics of North America ◽

10.1016/j.hoc.2017.06.006 ◽

2017 ◽

Vol 31 (5) ◽

pp. 721-735 ◽

Author(s):

Donald B. Kohn

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Hematopoietic Stem Cell ◽

Historical Perspective ◽

Hematopoietic Stem ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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